Archive for Healthcare – Page 2

Heathcare and Biotech Updates

Biopharma Co. Trades to New 52-Week High

Source: Streetwise Reports  (11/28/22)

Shares of Axsome Therapeutics Inc. traded to a new 52-week high after the company reported its AXS-05 successfully met both the primary and key secondary endpoints in the Phase 3 ACCORD Alzheimer’s disease agitation trial.

Biopharmaceutical company Axsome Therapeutics Inc. (AXSM:NASDAQ), which is focused on developing new medicines for use in the treatment of central nervous system (CNS) disorders, today announced that “AXS-05, a novel, oral, investigational NMDA receptor antagonist with multimodal activity, met the primary and key secondary endpoints in the ACCORD (Assessing Clinical Outcomes in Alzheimer’s Disease Agitation) Phase 3 trial, by substantially and statistically significantly delaying the time to relapse and preventing relapse of agitation in patients with Alzheimer’s disease, as compared to placebo.”

The company advised that the multi-center, randomized Phase 3 ACCORD study included a total of 178 patients in the U.S. who were diagnosed with Alzheimer’s disease agitation. The firm indicated that those who had received open-label treatment with AXS-05 demonstrated “rapid, substantial, and statistically significant improvement compared to baseline in agitation symptoms.” These patients who had experienced a sustained clinical response after open-label treatment with AXS-05 were then randomized to receive continued treatment with AXS-05 or a placebo.

Axsome Therapeutics noted that in the ACCORD study, AXS-05 met the primary objective defined as a substantial and statistically significant delay in the time elapsed to relapse of agitation symptoms compared to placebo. In the study, AXS-05 also met a key secondary endpoint which was listed as the prevention of relapses.

The company highlighted using the modified Alzheimer’s Disease Cooperative Study-CGIC (clinicians) scale, AXS-05 was shown to lessen Alzheimer’s disease agitation in 66% of patients after two weeks and by 86% of patients after five weeks. Similarly, utilizing the PGI-C assessment (caregivers) scale, 68% of patients showed improvement in agitation at two weeks, and 89% registered improvement at five weeks.

Dr. Cummings continued, “The results of the ACCORD trial demonstrate convincing clinical activity for AXS-05 on agitation associated with Alzheimer’s disease based on both a significant delay in symptom relapse as well as a reduction of relapse compared to placebo.”

Jeffrey Cummings, M.D., D.Sc., Director Emeritus of the Cleveland Clinic Lou Ruvo Center for Brain Health, and Chambers Professor of Brain Science at the University of Nevada Las Vegas, noted that “Agitation is one of the most troubling and consequential aspects of Alzheimer’s disease for patients and their caregivers as it is associated with early nursing home placement, accelerated cognitive decline, and increased mortality.”

Dr. Cummings continued, “The results of the ACCORD trial demonstrate convincing clinical activity for AXS-05 on agitation associated with Alzheimer’s disease based on both a significant delay in symptom relapse as well as a reduction of relapse compared to placebo. Treatment with AXS-05 during the open-label period in a large cohort of patients resulted in rapid and clinically meaningful improvements in Alzheimer’s disease agitation.”

The company’s CEO, Herriot Tabuteau, M.D., stated, “With the positive results from ACCORD, AXS-05 has now demonstrated efficacy in the treatment of Alzheimer’s disease agitation in two well-controlled trials. In addition to the strong results versus placebo in the double-blind period, results from the open-label period evidenced rapid, substantial, and significant improvements in Alzheimer’s disease agitation versus baseline with AXS-05 treatment.”

“We intend to discuss these findings with the FDA in the context of the ongoing clinical development of AXS-05 in this indication, with the goal of providing a much-needed treatment to the millions of patients living with Alzheimer’s disease agitation and their caregivers,” Dr. Tabuteau added.

The company stated that “Alzheimer’s disease (AD) is a progressive neurodegenerative disorder characterized by cognitive decline and behavioral and psychological symptoms including agitation.” AD affects around six million people in the U.S. and is the most frequently occurring type of dementia. Agitation, which includes aggressive behavior, disinhibition, disruptive irritability, and emotional distress, is reported in about 70% of patients diagnosed with AD. Currently, there are no U.S. Food and Drug Administration (FDA) approved therapies to treat agitation in AD patients.

The firm explained that “AXS-05 (dextromethorphan-bupropion) is a novel, oral, patent protected, investigational N-methyl-D-aspartate (NMDA) receptor antagonist with multimodal activity under development for the treatment of Alzheimer’s disease (AD) agitation and other central nervous system (CNS) disorders.” The company uses its metabolic inhibition technology to modulate the delivery of AXS-05’s patented formulation of dextromethorphan and bupropion. The report listed that supported by the positive results collected during the ADVANCE-1 trial, AXS-05 was awarded Breakthrough Therapy designation by the FDA in June 2020 for the treatment of Alzheimer’s disease agitation.

Axsome Therapeutics is a biopharmaceutical firm headquartered in New York, NY that is working to develop novel therapies for treating central nervous system (CNS) conditions. Axsome’s ongoing product development pipeline includes potential treatments for agitation associated with Alzheimer’s disease, acute migraine, fibromyalgia, smoking cessation, cataplexy in narcolepsy, and attention deficit hyperactivity disorder.

Axsome Therapeutics started the day with a market cap of around US$2.47 billion, with approximately 43.43 million shares outstanding and a short interest of about 15.6%. AXSM shares opened 25% higher today at US$71.035 (+US$14.215, +25.02%) over Friday’s US$56.82 closing price and reached a new 52-week high price this morning of US$79.68. The stock traded today between US$68.12 and US$79.68 per share and closed for trading at US$74.68 (+US$17.92, +31.54%).

Disclosures:

1) Stephen Hytha wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

US Biopharma Co. Is Attractive, Derisked Investment, Analyst Says

Source: Streetwise Reports  (11/11/22)

With late-stage drug candidates and major near-term catalysts, undervalued Aldeyra Therapeutics warrants a Buy to Outperform rating and consideration by potential investors, according to various analysts.

For a biopharma with one new drug candidate on the verge of potential approval in the U.S. and a second close behind, Aldeyra Therapeutics Inc.(ALDX:NASDAQ) is currently undervalued and represents an attractive, derisked investment opportunity, experts said.

The Massachusetts-based firm develops treatments for immune-mediated diseases, which regulate entire immunological systems rather than alter a single protein. The therapeutic candidates are designed to optimize numerous pathways while limiting toxicity, and the biopharma is currently advancing three such products.

A Trio of Potential New Therapies

1) Reproxalap: This RASP, or reactive aldehyde species, inhibiting 0.25% ophthalmic solution for dry eye disease is Aldeyra’s lead drug candidate, for which the company is on schedule to file a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) by year-end.

Approval “could lead to a meaningful lift for the shares,” purported Oppenheimer analyst Justin Kim.

In clinical trials, reproxalap was shown to be efficacious and safe. It “demonstrated robust and consistent dry eye disease benefit,” wrote H.C. Wainwright & Co. analyst Matthew Caufield in a July 13 research note. “We view RASP inhibition as presenting a viable novel pathway in addressing current dry eye disease therapeutic limitations.”

Caulfield noted that approved dry eye disease treatments on the market could take months to have an appreciable effect, have inconsistent responses among patients, and can be uncomfortable, often causing patients to stop using them. RASP is different as it is said to provide immediate relief, unlike previous therapies.

 “From an FDA perspective, reproxalap is very safe, passes the Schirmer test with high significance, and has a novel mechanism of action in a field with underserved patients. We think that will be enough for approval,” said BTIG’s Thomas Shrader.

Laidlaw & Co. analyst Dr. Yale Jen stated, “Although ALDX could launch reproxalap by themselves, we believe this is a highly desirable product for large pharma companies, especially those that could leverage their existing or start an ophthalmology sales force.”

BTIG’s Thomas Shrader is one of several analysts who remain bullish on reproxalap’s chances of approval for dry eye disease. In a June 8, 2022 research report, he wrote, “From an FDA perspective, reproxalap is very safe, passes the Schirmer test with high significance, and has a novel mechanism of action in a field with underserved patients. We think that will be enough for approval.”

Newsletter writer Clive Maund said, in a November 1st posting, “Action since this candle looks like a tiny bull Flag suggesting renewed advance soon. Buyers here should place a stop below US$5.00.”

Reproxalap is also being evaluated for allergic conjunctivitis and is now in Phase 3.

2) ADX-2191: This intravitreal methotrexate injection is a Phase 2 developmental treatment for retinitis pigmentosa and primary vitreoretinal lymphoma. For the latter, Aldeyra has a pre-NDA meeting scheduled with the FDA this quarter.

Aldeyra intends for ADX-2191 to also prevent proliferative vitreoretinopathy (in Phase 3). Topline Phase 3 GUARD trial results suggest ADX-2191 treatment could be safer and more effective in this indication than compounded methotrexate, wrote Dr. Yale Jen, a Laidlaw & Co. analyst, in an Oct. 6, 2022 research note.

Jen also noted the current clinical package for ADX-2191 in proliferative vitreoretinopathy is “strong” and likely to support an NDA. To delineate the regulatory pathway forward for this, Aldeyra is scheduling a Type C meeting with the FDA for H1/23.

3) ADX-629. This orally administered RASP modulator is in Phase 2 clinical testing for the treatment of four immune-mediated diseases: ethanol toxicity, chronic cough, Sjögren-Larsson Syndrome, and minimal change disease.

Implications of Near-Term Catalysts

Because dry eye disease is a large, currently underserved market, FDA approval of reproxalap as a treatment for it would be a significant development for Aldeyra, BTIG analyst Shrader wrote.

Approval “could lead to a meaningful lift for the shares,” purported analyst Justin Kim in a Sept. 15 research report. His firm Oppenheimer rates Aldeyra Outperform.

Were reproxalap approved, Aldeyra could reach commercialization in 2023.

As for Aldeyra’s shares, they are currently “underexposed and undervalued,” according to Laidlaw‘s Dr. Yale Jen.

With respect to ADX-2191, positive GUARD trial results, and the pre-NDA meeting on primary vitreoretinal lymphoma, Kim noted, “could catalyze a nontrivial revenue opportunity relative to current share levels.”

As for Aldeyra’s shares, they are currently “underexposed and undervalued,” according to analyst Jen. Today Aldeyra’s share price is US$5.32, and it has been trading in the US$5 range since Sept. 20, 2022.

In comparison, Jen’s firm, Laidlaw & Co., has a US$30 per share target price on the biopharma; this represents a significant jump and return on investment from its share price today.

While Aldeyra’s cash position declined in the latest quarter, Jen noted in a November 11 research note that “ALDX ended 3Q22 with ~US$185M cash, enough to support its operations throughout 2023.” In the report, Laidlaw & Co. reiterated its Buy rating and said, “ALDX shares remain underexposed and under-valued.”

Institutions Dominate Ownership

Institutions held 68.43% of Aldeyra’s shares, the Top 3 being Perceptive Advisors LLC (16.98%), The Vanguard Group Inc. (4.18%), and Citadel Advisors LLC (3.97%). The No. 1 mutual fund holder was the Vanguard Total Stock Market Index Fund at 2.74%.

Aldeyra insiders, including Chief Executive Officer Dr. Todd Brady, Chief Development Officer Dr. Stephen Machatha, Chairman of the Board Dr. Richard Douglas, and several directors, together owned 2.36% of the company’s shares.

The Aldeyra investment opportunity is one that numerous biotech research analysts view favorably. As of them, Kim wrote, “Aldeyra’s late-stage ophthalmology pipeline in allergic conjunctivitis and dry eye diseases offers a favorable risk-reward to current share levels, coupled with long-term pipeline optionality from ADX-2191 in proliferative vitreoretinopathy and systemic RASP applications.”

Coverage and Share Structure

Aldeyra is followed by numerous analysts, including Wainwright & Co. analyst Matthew Caufield, BTIG’s Thomas Shrader, Dr. Yale Jen of Laidlaw & Co., and Justin Kim of Oppenheimer. Newsletter writer Clive Maund also follows the stock. Click “See More Live Data” in the data box above to read their reports.

Aldeyra Therapeutics’ market cap is US$320.78M. The company has 58.32 million shares outstanding, and it trades in a 52-week range of  US$2.36 and US$9.06. 

Disclosures:
1) Doresa Banning compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. She or members of her household own securities of the following companies mentioned in the article: None. She or members of her household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees. As of the date of this article, an affiliate of Streetwise Reports has a consulting relationship with None. Please click here for more information.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases. As of the date of this article, officers and/or employees of Streetwise Reports LLC (including members of their household) own securities of Aldeyra Therapeutics Inc., a company mentioned in this article.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Expert Says Buy Pharma Co. Stock ‘as Soon as Possible’

Source: Clive Maund  (10/25/22)

Algernon Pharmaceuticals has entered into a clinical trial agreement with Yale and caught expert Clive Maund’s attention. In light of this, Maund reviews the company’s chart to tell you when he believes you should buy its stock.

Algernon Pharmaceuticals Inc. (AGN:CSE; AGNPF:OTCQB; AGN0:XFRA) got sold down further than we expected, making an unexpected further drop last week that took it down close to a trendline connecting a series of lows.

However, it now looks set to reverse to the upside, especially as this morning it came out with the news that it has entered into a clinical trial agreement with Yale University for a DMT Phase 2 Depression Study.

The fact that it has only 2 million shares in issue improves its potential for rapid recovery.

Anyone holding should therefore stay long, and it is rated a Buy again here. Those interested should aim to buy it as soon as possible after the open this morning.

Algernon Pharmaceuticals’s website.

Algernon Pharmaceuticals Inc. closed at CA$2.90, $2.09 on October 21, 2022.

CliveMaund.com Disclosures

The above represents the opinion and analysis of Mr. Maund, based on data available to him, at the time of writing. Mr. Maund’s opinions are his own, and are not a recommendation or an offer to buy or sell securities. Mr. Maund is an independent analyst who receives no compensation of any kind from any groups, individuals or corporations mentioned in his reports. As trading and investing in any financial markets may involve serious risk of loss, Mr. Maund recommends that you consult with a qualified investment advisor, one licensed by appropriate regulatory agencies in your legal jurisdiction and do your own due diligence and research when making any kind of a transaction with financial ramifications. Although a qualified and experienced stock market analyst, Clive Maund is not a Registered Securities Advisor. Therefore Mr. Maund’s opinions on the market and stocks can only be construed as a solicitation to buy and sell securities when they are subject to the prior approval and endorsement of a Registered Securities Advisor operating in accordance with the appropriate regulations in your area of jurisdiction.

Disclosures:
1) Clive Maund: I, or members of my immediate household or family, own securities of the following companies mentioned in this article: None. I personally am, or members of my immediate household or family are, paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: Algernon Pharmaceuticals Inc. Click here for important disclosures about sponsor fees. As of the date of this article, an affiliate of Streetwise Reports has a consulting relationship with Algernon Pharmaceuticals Inc. Please click here for more information.

3) Statements and opinions expressed are the opinions of the author and not of Streetwise Reports or its officers. The author is wholly responsible for the validity of the statements. The author was not paid by Streetwise Reports for this article. Streetwise Reports was not paid by the author to publish or syndicate this article. Streetwise Reports requires contributing authors to disclose any shareholdings in, or economic relationships with, companies that they write about. Streetwise Reports relies upon the authors to accurately provide this information and Streetwise Reports has no means of verifying its accuracy.

4) This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases. As of the date of this article, officers and/or employees of Streetwise Reports LLC (including members of their household) own securities of Algernon Pharmaceuticals Inc., a company mentioned in this article.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Biopharma Co.’s Shares Roll to New 52-Week High Price

Source: Streetwise Reports  (10/12/22)

Shares of DICE Therapeutics Inc. traded 62% higher yesterday and established a new 52-week intraday high after the company reported positive topline data from its Phase 1 DC-806 Psoriasis study.

Biopharmaceutical company DICE Therapeutics Inc. (DICE:NASDAQ), which utilizes its proprietary technology platform to create and develop novel oral therapeutic candidates for use in the treatment of chronic immune and other diseases, yesterday announced “positive topline data from its Phase 1 clinical trial of DC-806, an oral small molecule antagonist of the pro-inflammatory cytokine IL-17.”

The company advised that DC-806 is its leading interleukin-17 (IL-17) antagonist that is being evaluated as a potential treatment for psoriasis. The firm noted that DC-806 has been well tolerated in the Phase 1 trial across all dose groups in healthy volunteers and psoriasis patients with a robust PK profile.

DICE Therapeutics indicated that both high and low doses of DC-806 administered to participants in the study provided clear beneficial pharmacodynamic results on two distinct biomarkers.

DICE shares opened 79% higher yesterday at $44.18 (+$14.265, +57.87%) over last Friday’s $24.65 closing price and reached a new 52-week high price yesterday morning of $45.99.

The company explained that the randomized, double-blind Phase 1 trial was designed to evaluate the safety and pharmacokinetics of DC-806 in healthy volunteers and to serve as the basis for the potential use treatment of psoriasis patients.

The study included overlapping modules that included a Phase 1a single ascending dose, Phase 1b multiple ascending doses, and Phase 1c, which was structured under a proof-of-concept context for use in treating patients with psoriasis.

The firm emphasized that in the clinical proof-of-concept portion of the trial (Phase 1c), “psoriasis patients achieved with a mean percentage reduction in PASI from baseline at four weeks of 43.7% in the high dose group compared to 13.3% in the placebo group.”

DICE Therapeutics advised that the data gathered in the trials provide staunch support for advancing DC-806 as a potential oral agent for treating psoriasis. The firm added that it intends to file an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) during H1/23 and stated that it plans to move forward with a dose-ranging Phase 2b clinical trial in patients with moderate-to-severe psoriasis in H1/23.

DICE Therapeutics’ CEO Kevin Judice, Ph.D. stated, “We are extremely excited by the overall clinical profile of DC-806 and clear proof-of-concept in psoriasis in this Phase 1 clinical trial, which we believe is the first-ever clinical demonstration of direct inhibition of a cytokine with a small molecule.”

Judice continued, “We believe these data not only support further development of DC-806 as a potential best-in-class oral therapy for psoriasis, but also may unlock additional IL-17-mediated disease indications given DC-806’s excellent safety profile, strong PK data, and robust dose-dependent target engagement.”

The company’s Chief Medical Officer, Tim Lu, M.D., Ph.D., remarked, “Based on the Phase 1 clinical trial, we believe DC-806 has the potential to be the best-in-class oral therapeutic agent for patients with psoriasis…Data from this Phase 1 clinical trial provides early evidence for the potential differentiation of DC-806 on efficacy, safety, and ease of use. We look forward to advancing DC-806 into a Phase 2b clinical trial to optimize dosing and further explore peak efficacy with a longer duration of treatment.”

DICE listed that “it is developing orally-available, small molecule antagonists of the pro-inflammatory signaling molecule IL-17, an immune cell-derived cytokine that is produced in response to infection by certain microorganisms.”

The company stated in the report that there is no cure for psoriasis, which “manifests as erythematous plaques with thick scaling that can occur anywhere on the body.” The disease causes symptoms that typically include itchiness, pain, and bleeding from scratching, which often results in scarring and disfiguration. The firm stated that according to the National Psoriasis Association, psoriasis affects about 125 million people globally.

DICE Therapeutics is a clinical-stage biopharma company based in South San Francisco, Calif., that focuses on developing and advancing new medicines for treating chronic autoimmune and inflammatory diseases.

The firm uses its proprietary DELSCAPE platform to build a pipeline of novel oral therapeutics to address well-validated targets in immunology that match or exceed the potency of their systemic biologic counterparts. The DELSCAPE platform can identify and discover selective oral small molecules that demonstrate potential for modulating protein-protein interactions (PPIs) that are equal to or better than systemic biologics.

DICE Therapeutics started off yesterday with a market cap of around $941.9 million, with approximately 38.21 million shares outstanding and a short interest of about 12.9%. DICE shares opened 79% higher yesterday at $44.18 (+$14.265, +57.87%) over last Friday’s $24.65 closing price and reached a new 52-week high price yesterday morning of $45.99. The stock traded between $36.56 and $45.99 per share and closed for trading at $40.00 (+$15.35, +62.27%).

Disclosures:
1) Stephen Hytha wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Biopharma Co. Partners With Merck for Combo Drug Trial

Source: Dr. Joseph Pantginis  (10/12/22)

With this and other clinical studies, the U.S. company has a busy and, thus, catalyst-rich Q4/22 ahead, noted an H.C. Wainwright & Co. report.

Compass Therapeutics Inc. (CMPX:NASDAQ) entered a partnership with Merck & Co. Inc. (MRK:NYSE) to conduct a clinical trial evaluating the combination treatment of Compass’ CTX-471 plus Merck’s Keytruda in a subpopulation of patients with certain metastatic or locally advanced cancers, reported H.C. Wainwright & Co. analyst Dr. Joseph Pantginis in an October 11, 2022 research note. CTX-471 is a monoclonal antibody targeting CD137.

“Phase 1b is an important signal-seeking study,” Pantginis wrote.

Also of note, the Massachusetts-based biopharma offers investors significant potential return, as indicated by its current share price of US$3.11 and H.C. Wainwright’s target price on the company of US$12 per share.

Participants in the Phase 1b collaboration study will be patients with non-small cell lung cancer, small cell lung cancer, melanoma, or squamous cell carcinoma of the head and neck who progressed after receiving checkpoint inhibitor therapy. In the trial, after a patient progresses, they will be given CTX-471 plus Keytruda. Part one of the study will test escalating doses of CTX-471 and a fixed Keytruda dose. Part two will test expanding doses.

Also of note, the Massachusetts-based biopharma offers investors significant potential return, as indicated by its current share price of US$3.11 and H.C. Wainwright’s target price on the company of US$12 per share.

Pantginis pointed out that Merck and other big pharmaceutical firms are now highly selective when collaborating regarding their checkpoint inhibitors. Merck having agreed to join forces with Compass, suggests “the combination approach must make mechanistic sense, and we believe combining CTX-471 and Keytruda fits the bill.”

Other Plans for Pipeline

In addition to this Phase 1b, Compass intends to conduct two additional CTX-471 studies. One is a post-cyclin-dependent kinase inhibitor salvage study, Pantginis relayed. The other will test induction therapy of carboplatin/etoposide/atezolizumab followed by maintenance atezolizumab plus/minus CTX-471 as a first-line treatment for small cell lung cancer patients.

Near-Term Catalysts

Compass is busy this quarter with several “meaningful milestones lined up, noted Pantginis. The “leading value driver” for the biopharma, according to Pantginis, is the start of a Phase 2/3 study in the U.S. of CTX-009 in biliary tract cancer.

Other potential stock-moving events are the start of a Phase 2 study of CTX-009 in advanced colorectal cancer and the completion of Phase 1b CTX-471 monotherapy study in various cancers. Finally, Compass is targeting Q4/22 for submitting an investigational new drug application for CTX-8371.

H.C. Wainwright has a Buy rating on Compass.

“Our focus is squarely on Compass’ clinical data delivered by CTX-009, to date, as well as its strong cash balance to weather these depressing markets,” Pantginis wrote.

Disclosures:
1) Doresa Banning wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. She or members of her household own securities of the following companies mentioned in the article: None. She or members of her household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security. As of the date of this article, an affiliate of Streetwise Reports has a consulting relationship with: None. Please click here for more information.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal  disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Disclosures For H.C.Wainwright & Co., Compass Therapeutics Inc., October 11, 2022

H.C. Wainwright & Co, LLC (the “Firm”) is a member of FINRA and SIPC and a registered U.S. Broker-Dealer.

I, Joseph Pantginis, Ph.D. , certify that 1) all of the views expressed in this report accurately reflect my personal views about any and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectly related to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members of my household is an officer, director or advisory board member of these companies.

None of the research analysts or the research analyst’s household has a financial interest in the securities of Compass Therapeutics, Inc. (including, without limitation, any option, right, warrant, future, long or short position). As of September 30, 2022 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities of Compass Therapeutics, Inc..

Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the time of publication of this research report. The research analyst principally responsible for preparation of the report does not receive compensation that is based upon any specific investment banking services or transaction but is compensated based on factors including total revenue and profitability of the Firm, a substantial portion of which is derived from investment banking services.

The firm or its affiliates received compensation from Compass Therapeutics, Inc. for non-investment banking services in the previous 12 months. The Firm or its affiliates did receive compensation from Compass Therapeutics, Inc. for investment banking services within twelve months before, and will seek compensation from the companies mentioned in this report for investment banking services within three months following publication of the research report.

H.C. Wainwright & Co., LLC managed or co-managed a public offering of securities for Compass Therapeutics, Inc. during the past 12 months. The Firm does not make a market in Compass Therapeutics, Inc. as of the date of this research report. The securities of the company discussed in this report may be unsuitable for investors depending on their specific investment objectives and financial position. Past performance is no guarantee of future results. This report is offered for informational purposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdiction where such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice to any person. Electronic versions of H.C. Wainwright & Co., LLC research reports are made available to all clients simultaneously.

No part of this report may be reproduced in any form without the expressed permission of H.C. Wainwright & Co., LLC. Additional information available upon request. H.C. Wainwright & Co., LLC does not provide individually tailored investment advice in research reports. This research report is not intended to provide personal – investment advice and it does not take into account the specific investment objectives, financial situation and the particular needs of any specific person. Investors should seek financial advice regarding the appropriateness of investing in financial instruments and implementing investment strategies discussed or recommended in this research report.

H.C. Wainwright & Co., LLC’s and its affiliates’ salespeople, traders, and other professionals may provide oral or written market commentary or trading strategies that reflect opinions that are contrary to the opinions expressed in this research report. H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to time have long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants) thereof of covered companies referred to in this research report.

The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as being accurate and does not purport to be a complete statement or summary of the available data on the company, industry or security discussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date of this report and are subject to change without notice.

Securities and other financial instruments discussed in this research report: may lose value; are not insured by the Federal Deposit Insurance Corporation; and are subject to investment risks, including possible loss of the principal amount invested.

 

 

Pantginis pointed out that Merck and other big pharmaceutical firms are now highly selective when collaborating regarding their checkpoint inhibitors. Merck having agreed to join forces with Compass, suggests “the combination approach must make mechanistic sense, and we believe combining CTX-471 and Keytruda fits the bill.”

Trial Results Show New Drug Improves Dry Eye Symptoms

These study data from Aldeyra Therapeutics Inc. solidified the therapeutic’s path to filing a new drug application, potentially in Q3/22, noted an H.C. Wainwright & Co. report.

Aldeyra Therapeutics Inc.’s (ALDX:NASDAQ) reproxalap was shown, via recent clinical trial results, to be effective for dry eye disease, reported H.C. Wainwright & Co. analyst Matthew Caufield in a July 13 research note. These data bode well for the new drug application (NDA) the biotech intends to submit to the U.S. Food and Drug Administration (FDA) for reproxalap, likely in Q3/22.

Reproxalap is a RASP, or reactive aldehyde species, inhibiting 0.25% ophthalmic solution to be administered topically.

“Reproxalap demonstrated robust and consistent dry eye disease benefit,” added Caufield. “We view RASP inhibition as presenting a viable novel pathway in addressing current dry eye disease therapeutic limitations.”

The approved dry eye disease treatments on the market, noted Caufield, can take months to have an appreciable effect, have inconsistent responses among patients, and can be uncomfortable, often causing patients to stop using them.

Caufield highlighted that the newly released positive data, from the vehicle-controlled crossover reproxalap trial, show the drug met primary and secondary endpoints with statistical significance.

With reproxalap, study participants exhibited improvement in:

1) Dry eye chamber ocular redness, primary endpoint, (p=0.0004). The benefit was observed initially at 10 minutes and then through the final 90 minutes after chamber entry.

2) Schirmer test tear production assessment after one day of dosing, primary endpoint, (p=0.0005). This result supports prior dosing activity and fourth dose activity versus single-day dosing.

3) Schirmer test greater than or equal to 10-millimeter responder analysis, secondary endpoint, (p=0.0361).

Other secondary endpoints reproxalap showed a benefit in were ocular dryness (p=0.0068), discomfort (p<0.0001), grittiness (p=0.0001), stinging (p=0.0001), burning (p<0.0001) and itching (p=0.0003).

These “reflect a broad representation of prospective symptom benefit within a characteristically heterogeneous indication population,” wrote Caufield.

Reproxalap also demonstrated a positive safety and tolerability profile. The most common drug-related adverse event was mild, fleeting discomfort at the administration site.

“This most recent data set provides direct RASP inhibition support for the prior TRANQUILITY-2 and TRANQUILITY trials,” Caufield commented.

In the next step for its reproxalap program, Aldeyra will meet with the FDA for feedback before submitting the NDA, the most imminent near-term catalyst for the drug developer, according to Caufield.

The analyst also pointed out the FDA does not require all benefits of a prospective dry eye disease drug to be exhibited in one clinical trial. Rather, each benefit must be demonstrated in more than one clinical trial. This is the case with reproxalap, simplifying the approval path for it.

H.C. Wainwright has a Buy rating and a $15 per share price target on Aldeyra, the current share price of which is around $5.06.

 

Disclosures

1) Doresa Banning wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. They or members of their household own securities of the following companies mentioned in the article: None. They or members of their household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.

6) This article does not constitute medical advice. Officers, employees, and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Disclosures for H.C.Wainwright & Co., Aldeyra Therapeutics, July 13, 2022

H.C. Wainwright & Co, LLC (the “Firm”) is a member of FINRA and SIPC and a registered U.S. Broker-Dealer.

I, Matthew Caufield and Andrew S. Fein , certify that 1) all of the views expressed in this report accurately reflect my personal views about any and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectly related to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members of my household is an officer, director or advisory board member of these companies. None of the research analysts or the research analyst’s household has a financial interest in the securities of Aldeyra Therapeutics (including, without limitation, any option, right, warrant, future, long or short position).

As of June 30, 2022 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities of Aldeyra Therapeutics. Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the time of publication of this research report.

The research analyst principally responsible for preparation of the report does not receive compensation that is based upon any specific investment banking services or transaction but is compensated based on factors including total revenue and profitability of the Firm, a substantial portion of which is derived from investment banking services. The firm or its affiliates received compensation from Aldeyra Therapeutics for non-investment banking services in the previous 12 months.

The Firm or its affiliates did not receive compensation from Aldeyra Therapeutics for investment banking services within twelve months before, but will seek compensation from the companies mentioned in this report for investment banking services within three months following publication of the research report.

The Firm does not make a market in Aldeyra Therapeutics as of the date of this research report.

The securities of the company discussed in this report may be unsuitable for investors depending on their specific investment objectives and financial position. Past performance is no guarantee of future results. This report is offered for informational purposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdiction where such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice to any person. Electronic versions of H.C. Wainwright & Co., LLC research reports are made available to all clients simultaneously.

H.C. Wainwright & Co., LLC does not provide individually tailored investment advice in research reports. This research report is not intended to provide personal investment advice and it does not take into account the specific investment objectives, financial situation and the particular needs of any specific person. Investors should seek financial advice regarding the appropriateness of investing in financial instruments and implementing investment strategies discussed or recommended in this research report.

H.C. Wainwright & Co., LLC’s and its affiliates’ salespeople, traders, and other professionals may provide oral or written market commentary or trading strategies that reflect opinions that are contrary to the opinions expressed in this research report.

H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to time have long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants) thereof of covered companies referred to in this research report.

The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as being accurate and does not purport to be a complete statement or summary of the available data on the company, industry or security discussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date of this report and are subject to change without notice.

Securities and other financial instruments discussed in this research report: may lose value; are not insured by the Federal Deposit Insurance Corporation; and are subject to investment risks, including possible loss of the principal amount invested.

Biopharma Co. Looks to Expand New Drug Label Down the Road

ScPharmaceuticals Inc. just released topline results from a Phase 2 pilot study supporting the drug developer that is pursuing the expansion of its new drug label, noted an H.C. Wainwright & Co. report.

ScPharmaceuticals Inc. (SCPH:NASDAQ) garnered support for the “value proposition” and future “addition to the label” of its drug Furoscix, from the topline results of AT-HOME, a Phase 2 pilot study in heart failure, reported H.C. Wainwright & Co. analyst Douglas Tsao in a July 13 research note.

Furoscix is under U.S. Food and Drug Administration (FDA) review at present as a treatment of congestion due to fluid overload in adult patients with chronic heart failure, who do not need hospitalizing and who respond suboptimally to oral diuretics.

“We expect approval on the PDUFA date of October 8” (of this year), Tsao commented.

The purpose of the AT-HOME trial was twofold. It was to assess the clinical outcomes and safety of Furoscix against those of “treatment as usual.” This approach typically involves increasing a patient’s oral furosemide dose or adding metolazone to their treatment regimen, Tsao wrote.

The second goal of AT-HOME was to inform the design of a potentially larger study to be used as support for future expansion of Furoscix’s approved uses, noted Tsao. The biopharma plans to meet with the FDA to discuss such a trial.

AT-HOME was not powered to evaluate Furoscix for statistically significant efficacy in chronic heart failure patients, yet its topline results showed just that, versus “treatment as usual,” Tsao pointed out.

Specifically, of note, the analyst relayed that patients treated with Furoscix versus those treated “as usual” exhibited a positive trend in the primary endpoint. It was a 30-day hierarchal composite of cardiovascular death, heart failure hospitalizations, emergency department visits for heart failure, and the percent change in the N terminal-pro hormone BNP from baseline at day seven, as determined using the Finkelstein-Schoenfeld win ratio.

Also, Tsao reported that at day 30 the Furoscix-treated patients had a 37% reduction in the risk of a heart failure-related hospitalization relative to patients who received “treatment as usual.” The Furoscix group also fared better in terms of secondary endpoints, including improvements in congestion, quality of life, and functional status.

In light of these new data, H.C. Wainwright maintained its Buy rating and $15 per share price target on scPharmaceuticals. Its stock today, in comparison, is trading at around $5.08 per share.

Disclosures
1) Doresa Banning wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. They or members of their household own securities of the following companies mentioned in the article: None. They or members of their household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.

6) This article does not constitute medical advice. Officers, employees, and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Disclosures for H.C.Wainwright & Co., scPharmaceuticals Inc., July 13, 2022

H.C. Wainwright & Co, LLC (the “Firm”) is a member of FINRA and SIPC and a registered U.S. Broker-Dealer.

I, Douglas Tsao , certify that 1) all of the views expressed in this report accurately reflect my personal views about any and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectly related to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members of my household is an officer, director or advisory board member of these companies.

None of the research analysts or the research analyst’s household has a financial interest in the securities of scPharmaceuticals Inc. (including, without limitation, any option, right, warrant, future, long or short position).

As of June 30, 2022 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities of scPharmaceuticals Inc.. Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the time of publication of this research report.

The research analyst principally responsible for preparation of the report does not receive compensation that is based upon any specific investment banking services or transaction but is compensated based on factors including total revenue and profitability of the Firm, a substantial portion of which is derived from investment banking services. The firm or its affiliates received compensation from scPharmaceuticals Inc. for non-investment banking services in the previous 12 months.

The Firm or its affiliates did not receive compensation from scPharmaceuticals Inc. for investment banking services within twelve months before, but will seek compensation from the companies mentioned in this report for investment banking services within three months following publication of the research report.

The Firm does not make a market in scPharmaceuticals Inc. as of the date of this research report.

The securities of the company discussed in this report may be unsuitable for investors depending on their specific investment objectives and financial position. Past performance is no guarantee of future results. This report is offered for informational purposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdiction where such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice to any person. Electronic versions of H.C. Wainwright & Co., LLC research reports are made available to all clients simultaneously.

H.C. Wainwright & Co., LLC does not provide individually tailored investment advice in research reports. This research report is not intended to provide personal investment advice and it does not take into account the specific investment objectives, financial situation and the particular needs of any specific person. Investors should seek financial advice regarding the appropriateness of investing in financial instruments and implementing investment strategies discussed or recommended in this research report.

H.C. Wainwright & Co., LLC’s and its affiliates’ salespeople, traders, and other professionals may provide oral or written market commentary or trading strategies that reflect opinions that are contrary to the opinions expressed in this research report.

H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to time have long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants) thereof of covered companies referred to in this research report.

The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as being accurate and does not purport to be a complete statement or summary of the available data on the company, industry or security discussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date of this report and are subject to change without notice.

Securities and other financial instruments discussed in this research report: may lose value; are not insured by the Federal Deposit Insurance Corporation; and are subject to investment risks, including possible loss of the principal amount invested.

Patients in Algernon’s Phase 2a Study Show Improved Lung Capacity

Source: Streetwise Reports   07/18/2022

One junior biotech company that attempts to treat current health problems with older previously approved drugs, recently completed a Phase 2 study that showed promise in the treatment of idiopathic pulmonary fibrosis (IPF) and chronic cough. It moved the small company a step closer to bringing the treatment to market.

A 12-week, Stage 2a proof of concept study using NP-120 or Ifenprodil to treat idiopathic pulmonary fibrosis (IPF) and chronic cough was recently completed by Vancouver-based Algernon Pharmaceuticals Inc. (AGN:CSE; AGNPF:OTCQB; AGN0:XFRA) — and it demonstrated that 13 out of 20 IPF patients who enrolled had stable or better lung capacity after 12 weeks.

The Phase 2 study’s positive topline data moved the company one step closer to bringing a new IPF and cough treatment to market.

“The IPF data look quite good,” said Dr. Martin Kolb, professor of respirology at McMaster University and global expert on IPF. Kolb is also a member of Algernon’s Medical and Scientific Advisory Board.

Toronto-based AlphaNorth Asset Management is Algernon’s largest shareholder with about a 7% stake. Co-founder Steve Palmer says he continues to accumulate Algernon shares as the company trades near 52-week lows. He told Streetwise Reports that it’s difficult to determine how the market will react to the study news.

AGN is incredibly undervalued having a market cap of only $6 million. AGN’s Ifenprodil has a well established safety profile and now promising initial IPF Phase 2a data — for an Orphan indication that is targeting an unmet medical need for a very large market opportunity… We recommend investors with a higher risk tolerance to aggressively purchase the stock.”

— Research Capital Analyst Andre Uddin

“It’s great news. The study was not designed for statistical significance, it was designed to see if there was any signal with this drug. To have achieved statistical significance with such a small number of patients is excellent,” Palmer told Streetwise Reports. “However, the market has been pretty poor across the board…it’s hard to predict in this kind of environment, how the stock will react. If we had a normal or good market, then I think the stock would react very positively.”

Current IPF treatments cost as much US$90,000 annually in a market expected to be worth more than US$4.2 billion ($4.2B) by 2030. It’s currently dominated by two pharmaceutical behemoths: Germany’s Boehringer Ingelheim, and Roche, based in Basel, Switzerland.

IPF is a chronic lung disorder that results in patients suffering shortness of breath after stiffening, thickening and ultimately scarring (fibrosis) of their lung tissue. The disease is usually terminal less than six years after diagnosis.

The cough data showed that while Ifenprodil didn’t achieve the more stringent primary endpoint on cough, 30% of patients saw a 50% reduction in their 24 hour cough count and overall, 75% of subjects saw improvements in their cough over 12 weeks. This may lead Algernon to focus on IPF patients who have associated cough instead of pursuing a separate Phase 2b study on cough alone.

“IPF is a $4B market while cough is a $1B market and so we have some thinking to do about next steps,” Algernon CEO Christopher J. Moreau told Streetwise Reports.

Based on the positive data, Algernon plans to file a Pre-Investigational New Drug Application with the U.S. Food and Drug Administration (FDA) for a Phase 2b IPF study.

If the study gets the green light, the company could switch to a new once-a-day dose of Ifenprodil. Patients who were in the 2a study took the drug three times daily.

Moreau said that and more have yet to be determined.

“We will communicate with the FDA and determine what our Phase 2b plan is and settle on the number of patients, the number of study arms, primary and secondary endpoints and likely a multiple dose treatment regime,” Moreau told Streetwise Reports. “There is a lot of work involved in planning a Phase 2 study and so it will be a number of months before we would be ready to start.”

He added that he believes some of the some big players in the pharmaceutical space are keeping an eye on Algernon and some have already signed non-disclosure agreements with the company.

How the Study Worked

IPF patients in the Stage 2a study in study groups in Australia and New Zealand had their lung function measured by forced vital capacity (FVC) — where a patient blows into a device measuring the force of their blowing — at the start of the trial, and then again after 12 weeks.

Patients whose FVC declined were classified as non-responders, while those whose FVC improved or remained stable were classified as responders.

Of the IPF 20 patients who enrolled, 13 (65%) had stable or improved FVC over the 12-week treatment period, whereas 40% of those treated with a placebo demonstrated similar or higher FVC over 12 weeks.

The drug essentially targets the body’s N-methyl-D-aspartate (NMDA) receptors and interupts glutamate signalling.

Algernon also reported that many of the serum markers — proC3, C3M, C6M, reC1M, proC8 and ELP-3 — trended lower during testing but the data did not reach statistical significance. Previous studies have shown that as these markers go up, so does the risk of disease progression and increased mortality.

Old Drugs, New Purpose

Drug studies typically start with a pre-clinical research phase where drugs are kept in the lab and never touch a human being. This phase has the highest failure rate at about 90%. If the drug moves forward, it goes into a Phase 1 study, which determines if a drug is safe for humans. Historically speaking, about 35% of drugs will fail in this phase.

Once a drug is considered safe, it can go into a Phase 2 clinical trial, which is the first time the drug is tested in the patient population the drug was intended to treat. If successful, the drug moves into a Phase 3 trial, which in essence is a much larger Phase 2. For new chemical entities, known as NCEs, the whole process usually takes about 13 or 14 years.

Algernon uses a different strategy. Its scientists examine research papers on drugs that have already been approved but are off patent and attempt to apply those same drugs to different diseases. One might say it’s akin to teaching an old drug a new “trick.”

By using a drug like Ifenprodil, which has already been approved, it shaves years off the development timeline.

Ifenprodil is still being distributed by Sanofi, but only in Japan where it is used to treat vertigo. This limited market exposure is part of Algernon’s drug strategy where the company only investigates drugs that have never before been approved in the U.S. or Europe.

“The benefit of this strategy is that it shortens the timeframe of the trial process because they can generally skip the Phase 1 because they already know that the drug is safe, and they can move right into Phase 2 studies to identify whether it works or not,” AlphaNorth Asset Management co-founder Steve Palmer told Streetwise Reports in a story originally published on July 11.

IPF is also classified as an orphan disease indication — that means it’s considered a significant health problem and any new approved drugs to treat IPF in the U.S. get at least seven years of market exclusivity. It’s 10 years in Europe.

Algernon plans to file for an orphan designation with the U.S FDA for Ifenprodil and IPF shortly, as well as an application with the FDA for a Breakthrough Therapy designation.

Everybody’s Business

In addition to finding a new treatment for IPF, Algernon is also seeking ways to patent N, N-dimethyltryptamine (DMT), a psychedelic compound to treat strokes — a global market estimated at US$40 billion annually.

Another drug known as Repirinast, which was initially developed to treat asthma, is being repurposed by Algernon to treat kidney disease.

Repirinast was originally developed by Mitsubishi Tanabe Pharma for the Japanese market.

Following the news of the successful Phase 2a study, Research Capital Corp. Analyst Andre Uddin maintained his Speculative Buy rating on Algernon with a CA$25 target price.

Uddin wrote: “AGN is incredibly undervalued having a market cap of only $6 million. AGN’s ifenprodil has a well established safety profile and now promising initial IPF Phase 2a data — for an Orphan indication that is targeting an unmet medical need for a very large market opportunity… We recommend investors with a higher risk tolerance to aggressively purchase the stock.”

Algernon recently completed a CA$1.2 million financing, basically replenishing the CA$1.2 million it had at the end of Q2. The company’s current burn rate is about $100,000 per month.

The company has 1.979 million shares issued, a small sum for any publicly traded company.

Toronto-based AlphaNorth Asset Management owns about 7% of Algernon, while Moreau owns slightly more than 1%.

Algernon trades in a 52-week range of CA$13 and CA$3.06. It closed at CA$3.14 on July 15.

Disclosures:
1) Brian Sylvester wrote this article with files from Steve Sobek for Streetwise Reports LLC. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: Algernon Pharmaceuticals Inc. Click here for important disclosures about sponsor fees. As of the date of this article, an affiliate of Streetwise Reports has a consulting relationship with Algernon Pharmaceuticals Inc. Please click here for more information.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports’ terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases. As of the date of this article, officers and/or employees of Streetwise Reports LLC (including members of their household) own securities of Algernon Pharmaceuticals Inc., a company mentioned in this article.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

Company Disclosures

This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional. Any action taken as a result of reading information here is the reader’s sole responsibility.

Why are drug names so long and complicated? A pharmacist explains the logic behind the nomenclature

By Jasmine Cutler, University of South Florida 

At some point in your life, you’ll likely find yourself with a prescription from your doctor to fill. While it’s important to keep track of all the medications you’re taking, that can be hard to do when the names of so many of these drugs are difficult to pronounce and even harder to remember.

In my role as a pharmacist, I’ve helped countless patients figure out exactly which medication they were taking for what ailment. Some wonder why they were prescribed the medication in the first place, or need help differentiating between drugs with names that seem like complete gibberish.

But there is a rhyme and a reason to drug names. All prescribed medications follow a standard nomenclature that describes what the drug is made of and how it functions.

Who names drugs?

Drugs get both a brand, or proprietary, name and a generic name that is nonproprietary. Each is assigned in a slightly different process.

As long as a drug compound isn’t trademarked, drug companies decide on a proprietary brand name for the medications they sell. Usually the brand name relates to the conditions the drug is intended to treat and is easy for both providers and patients to remember but doesn’t follow a standardized naming guideline. For example, the drug Lopressor helps lower blood pressure.

On the other hand, generic drug names all follow a standard nomenclature that helps medical providers and researchers more easily recognize and classify the drug. Lopressor, for example, has a generic name of metoprolol tartrate. The U.S. Adopted Names Council, composed of representatives from the Food and Drug Administration, American Medical Association, U.S. Pharmacopeia and American Pharmacists Association, works with the World Health Organization to assign international nonproprietary names, or INNs, to drug compounds. Similar organizations exist internationally.

A globally recognized naming process makes an otherwise confusing name game more manageable. It helps the medical community easily learn and categorize newly approved medications and reduce prescribing errors by providing a unique, standard name that reflects each active ingredient in the drug.

For example, several Type 2 diabetes medications fall under one class called glucagon-like peptide-1 (GLP-1) receptor agonists. Although all medications in this class have different brand names, each of the generic versions ends in the suffix “-tide.” This helps health providers identify all the drugs that belong to this medication class. A few examples include Byetta (exenatide), Trulicity (dulaglutide) and Victoza (liraglutide).

How are generic drug names assigned?

The naming process starts when a drug company submits an application to the U.S. Adopted Names Council with a proposed generic name. USAN considers a number of factors when evaluating a name, such as whether it relates to how the drug works, how translatable it is to other languages and whether it is easy to say. In general, the name should be simple – fewer than four syllables long – and should not be easily confused with other existing generic drugs.

Once a name is agreed upon by USAN and the drug company, it is then proposed to the INN Expert Group. Sponsored by the World Health Organization, the INN Expert Group is composed of global specialists who represent the pharmaceutical, chemical, pharmacological and biochemical sciences. They may either accept the proposed name or suggest an alternative. Once the drug company, USAN and the INN Expert Group come to an agreement about a name, it is placed in the WHO Drug Information journal for four months for public comments or objections before final adoption.

What’s in a generic drug name?

Generic names follow a prefix-infix-stem system. The prefix helps distinguish a drug from other drugs in the same class. The infix, used more occasionally, further subclassifies the drug. The stem at the very end of the name indicates the drug’s function and marks its place within the name game.

Stems are composed of one or two syllables that describe a drug’s biological effects as well as its physical and chemical qualities and structure. Drugs with the same stem share features like the conditions they treat and how they work in the body. The WHO publishes a regularly updated stem book to keep everything in line.

For example, the stem “-prazole” indicates that the drug is chemically related to a class of compounds called benzimidazoles that have similar functions. As a result, drugs such as lansoprazole (Prevacid), esomeprazole (Nexium) and omeprazole (Prilosec) all treat acid reflux, ulcers and heartburn. The “e” prefix of esomeprazole differentiates it from omeprazole, which has a slightly different chemical structure.

Another common example is drugs that use the stem “stat,” which means enzyme inhibitors. Atorvastatin (Lipitor), rosuvastatin (Crestor) and simvastatin (Zocor) all belong to the same class of inhibitors that block a key enzyme in the body’s cholesterol production process. As a result, these cholesterol-reducing “statins” are used to prevent cardiovascular conditions like heart attack and stroke.

Are there exceptions to the name game?

Although generic names stay consistent, there have been multiple changes to brand names over the past couple of decades after increases in prescribing and dispensing errors. Some examples include the acid reflux and stomach ulcer drug omeprazole, which was rebranded from Losec to Prilosec because it was frequently confused with the diuretic Lasix. Another example is when the antidepressant Brintellix was changed to Trintellix because it was commonly confused with the blood thinner Brilinta.

Some generic medications may work at multiple targets in the body and be used for multiple conditions. For example, drugs with the stem “-afil,” such as tadalafil (Cialis), sidenafil (Viagra) and vardenafil (Levitra), belong to a class of drugs that relax smooth muscle and widen the blood vessels. Although commonly prescribed for erectile dysfunction, they can also be used to treat pulmonary arterial hypertension, a specific type of elevated blood pressure that affects the arteries in the heart and lungs.

In addition, nomenclature guidelines aren’t set in stone, and the U.S. Adopted Names Council anticipates that they will continue to change as newer, more complex substances are discovered, developed and marketed.

For example, a rise in the number of drugs developed with different salts and esters has led to the use of a modified naming process to incorporate the inactive parts of the compound.

As you can guess, it takes health care providers countless months and years to learn and understand this naming process. We are taught the science behind each chemical structure and how it works, which makes it easier to know the rules of the name game. But for those without a background in chemistry and biology, it can be like reading a foreign language.

There are several resources that can help you navigate the drug name game, however. Ask your health care provider or pharmacist if you have questions about how your medication works or what it is used for. They are generally a phone call or visit away.The Conversation

About the Author:

Jasmine Cutler, Assistant Professor of Pharmacotherapeutics, University of South Florida

This article is republished from The Conversation under a Creative Commons license. Read the original article.

 

Healthcare Co.’s Psychedelic-Assisted Treatments Have ‘Blockbuster Drug Potential’

Source: Patrick Trucchio   07/15/2022

Psychedelic-assisted therapy has shown in a clinical trial that it can help patients with alcohol use disorder (AUD) or alcoholism. One company hopes to bring its treatments to clinics throughout Europe and become the go-to standard of care for AUD.

H.C. Wainright & Co. Analyst Patrick Trucchio initiated coverage on Awakn Life Sciences Corp. (AWKN:NEO; AWKNF:OTCQB), a biotechnology company that is advancing methylenedioxymethamphetamine (MDMA)-assisted therapy for alcohol use disorder (AUD) in Europe.

“We estimate (that MDMA-assisted therapy) could have blockbuster drug potential based on the significant unmet medical need and evidence generated to-date pointing to the potential of MDMA-assisted therapy in a variety of mood disorders,” Trucchio stated.

“Moreover, Awakn’s MDMA-assisted therapy has generated promising Phase 2a data in AUD, which follows the validation of the approach in PTSD in a late-stage program being conducted by MAPS, a non-profit organization based in the U.S.,” Trucchio added.

Awakn’s R&D efforts are focused on continued research related to ketamine-assisted and MDMA-assisted psychotherapy, at least initially.

“We estimate (that MDMA-assisted therapy) could have blockbuster drug potential based on the significant unmet medical need.”

— H.C. Wainwright & Co. Analyst Patrick Trucchio

In March 2021, Awakn acquired an exclusive license to the Phase 2 trial — the Ketamine for Reduction of Alcohol Relapse (KARE) study — from the University of Exeter in the U.K.

The KARE treatment is to be delivered in Awakn clinics in the U.K. Awakn has signed a Memorandum of Understanding with the National Healthcare Service (NHS), the U.K. healthcare regulator, and the University of Exeter, to assess options for bringing the KARE therapy into a Phase 3 program.

Separately, Trucchio notes that in March 2021 “Awakn acquired five years of know-how and research data from Equasy Enterprises, which is led by long-time psychedelic researcher David Nutt, (the data) has provided Awakn with access to details of potentially newly discovered modes of action for MDMA as well as details of potential faster acting entactogen-like compounds.”

Six months after that Awakn acquired the exclusive rights to MDMA-assisted therapies from Imperial College London which conducted a “successful Phase 2a. trial evaluating MDMA-assisted therapy in AUD.”

MDMA, also known as “ecstasy” and classified as a Schedule 1 drug (meaning it is illegal in the U.S.) since 1985, is a phenethylamine that raises levels of monoamine neurotransmitters in the brain.

Trucchio noted that “MDMA elevates mood, increases sociability and feelings of closeness to others, and can facilitate imagination and memory; evidence from neuroimaging studies shows a decrease in amygdala/hippocampus activity and an association between reduced amygdala activity and improved ability to process negative memories. Together with changes in social cognition, interpersonal closeness and communication, these data support the proposition that MDMA could be of benefit as an adjunctive psychotherapeutic treatment for alcohol addiction and co-morbid psychological disorders.”

In MDMA-assisted therapy studies conducted in post-traumatic stress disorder, on average, participants were drinking 130.6 units of alcohol per week in the month before detoxification, no units at the point of detox, and 18.7 units per week at nine months, implying a reduction of around 86% at nine months as compared to the average consumption one month prior to detox.

Awakn is building out a clinics network capable of delivering evidence-backed, psychedelic drug-assisted therapies for addiction and other mental health conditions in clinics in the U.K., and other European countries. As part of this build out, Awakn is also pursuing licensing partnerships of addiction treatments beyond the U.K. and Europe.

H.C. Wainwright & Co. has a Buy rating and a CA$10 per share price target on Awakn. The current share price is around CA$0.78.

 

Disclosures

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Disclosures for H.C. Wainwright & Co., Awakn Life Sciences Corp., February 1, 2022

None of the research analysts or the research analyst’s household has a financial interest in the securities of AWAKN Life Sciences Corp, Atai Life Sciences N.V., Field Trip Health Ltd. and Mind Medicine (MindMed) Inc. (including, without limitation, any option, right, warrant, future, long or short position). As of January 31, 2022 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities of AWAKN Life Sciences Corp, Atai Life Sciences N.V., Field Trip Health Ltd. and Mind Medicine (MindMed) Inc.. Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the time of publication of this research report.

I, Patrick R. Trucchio, CFA , certify that 1) all of the views expressed in this report accurately reflect my personal views about any and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectly related to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members of my household is an officer, director or advisory board member of these companies.

The research analyst principally responsible for preparation of the report does not receive compensation that is based upon any specific investment banking services or transaction but is compensated based on factors including total revenue and profitability of the Firm, a substantial portion of which is derived from investment banking services. The firm or its affiliates received compensation from Atai Life Sciences N.V., Field Trip Health Ltd. and Mind Medicine (MindMed) Inc. for non-investment banking services in the previous 12 months.

The Firm or its affiliates did not receive compensation from AWAKN Life Sciences Corp, Atai Life Sciences N.V., Field Trip Health Ltd. and Mind Medicine (MindMed) Inc. for investment banking services within 12 months before, but will seek compensation from the companies mentioned in this report for investment banking services within three months following publication of the research report. The Firm does not make a market in AWAKN Life Sciences Corp, Atai Life Sciences N.V., Field Trip Health Ltd. and Mind Medicine (MindMed) Inc. as of the date of this research report.

Past performance is no guarantee of future results. This report is offered for informational purposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdiction where such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice to any person.

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H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to time have long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants) thereof of covered companies referred to in this research report. The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as being accurate and does not purport to be a complete statement or summary of the available data on the company, industry or security discussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date of this report and are subject to change without notice.