Stay ahead of biotech stocks seeing a surge in interest from retail and financial professional investors with this exclusive recap from The Fly These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
Pipeline and key clinical candidates for these companies:
Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARalpha agonist in frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression.
Genprex is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex's technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options, the company says.
Revance Therapeutics is a biotechnology company whose aesthetics portfolio includes Daxxify for injection, the RHA Collection of dermal fillers, and OPUL, the "first-of-its-kind Relational Commerce platform for aesthetic practices." Revance has also partnered with Viatris to develop a biosimilar to Botox, which will compete in the existing short-acting neuromodulator marketplace. Revance's therapeutics pipeline is currently focused on muscle movement disorders including evaluating Daxxify in two debilitating conditions, cervical dystonia and upper limb spasticity.
Trevena is a biopharmaceutical company focused on the development and commercialization of innovative medicines for patients with CNS disorders. The company has one approved product in the United States, OLINVYK injection, indicated in adults for the management of acute pain severe enough to require an intravenous opioid analgesic and for whom alternative treatments are inadequate. The company's novel pipeline is based on Nobel Prize winning research and includes three differentiated investigational drug candidates: TRV045 for diabetic neuropathic pain and epilepsy, TRV250 for the acute treatment of migraine and TRV734 for maintenance treatment of opioid use disorder.
Daré Bioscience is advancing products for women's health, stating that the company's mission is "to identify, develop and bring to market a diverse portfolio of differentiated therapies that prioritize women's health and well-being, expand treatment options, and improve outcomes, primarily in the areas of contraception, fertility, and vaginal and sexual health." Daré's first FDA-approved product, XACIATO vaginal gel, is a lincosamide antibacterial indicated for the treatment of bacterial vaginosis in female patients 12 years of age and older, which is under a global license agreement with Organon. Daré's portfolio also includes potential first-in-category candidates in clinical development: Ovaprene, a novel, hormone-free monthly intravaginal contraceptive whose U.S. commercial rights are under a license agreement with Bayer; Sildenafil Cream, 3.6%, a novel cream formulation of sildenafil to treat female sexual arousal disorder utilizing the active ingredient in Viagra; and DARE-HRT1, a combination bio-identical estradiol and progesterone intravaginal ring for hormone therapy following menopause.
Orgenesis is a global biotech company that has been committed to unlocking the potential of cell and gene therapies since 2012 as well as a paradigm-shifting decentralized approach to processing since 2020. This new model allows Orgenesis to bring academia, hospitals, and industry together to make these essential therapies a reality sooner rather than later. Orgenesis is focusing on advancing its CGTs toward eventual commercialization, while partnering with key industry stakeholders to provide a rapid, globally harmonized pathway for these therapies to reach and treat a larger number of patients more cost effectively and with better outcomes through great science and decentralized production.
Ascendis Pharma is applying its platform technology to "build a leading, fully integrated, global biopharmaceutical company focused on making a meaningful difference in patients' lives," the company states. The company uses its TransCon technologies to "create new and potentially best-in-class therapies," Ascendis says.
Mirum Pharmaceuticals is focused on the treatment of rare liver diseases. Mirum's approved medication is Livmarli , or maralixibat oral solution, which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. In Europe, the European Committee for Medicinal Products for Human Use has issued a positive opinion for Livmarli for the treatment of cholestatic pruritus in patients with Alagille syndrome two months of age and older. A decision by the European Commission is expected by year-end 2022. Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults.
Organigram is focused on producing high-quality, indoor-grown cannabis for patients and adult recreational consumers in Canada, as well as developing international business partnerships to extend the company's global footprint. Organigram has also developed a portfolio of legal adult-use recreational cannabis brands, including Edison, Holy Mountain, Big Bag O' Buds, SHRED, Monjour and Trailblazer.
Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for the treatment of chronic respiratory diseases with significant unmet medical needs. If successfully developed and approved, Verona's product candidate, ensifentrine, has the potential to become the first non-steroidal therapy for the treatment of respiratory diseases that combines bronchodilator and anti-inflammatory activities in one molecule, the company said.
Recent news on these stocks:
August 14
Genprex announced positive clinical study updates for its Acclaim-1 and Acclaim-3 clinical trials for the treatment of non-small cell lung cancer, or NSCLC, and small cell lung cancer, or SCLC, respectively, and plans to re-focus its oncology clinical development program. Patients in the company's lung cancer clinical trials are being treated with the Company's lead drug candidate, Reqorsa, or quaratusugene ozeplasmid, Gene Therapy. Two patients in the Acclaim-1 study have had prolonged Progression Free Survival, or PFS, and importantly, the first treated patient in the Acclaim-3 study attained a Partial Remission, or PR, from the start of maintenance therapy. Ryan Confer, President and CEO, commented on the update: "We are excited by these early and promising patient responses to REQORSA treatment, particularly as these patients represent some of the most difficult to treat lung cancer patient populations. There is significant unmet medical need for patients afflicted with lung cancer, as nearly all patients' disease progresses following treatment, even when treated with today's most advanced targeted therapies and immunotherapies. This leaves patients with limited therapeutic options. We are thrilled our novel gene therapy treatment for lung cancer, REQORSA, is demonstrating early evidence of efficacy with a favorable safety profile. We look forward to continuing to evaluate REQORSA in our lung cancer clinical trials while we advance our efforts to bring new therapies to those battling cancer." The Acclaim-1 clinical trial is evaluating the combination of REQORSA and AstraZeneca's (AZN) Tagrisso to treat patients with late-stage NSCLC who have activating EGFR mutations and disease progression after treatment with Tagrisso. The Acclaim-3 clinical trial is evaluating the combination of REQORSA and Genentech's Tecentriq as a maintenance therapy to treat patients with extensive stage small cell lung cancer who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. Confer and the executive team have evaluated resource allocations to ensure streamlined, focused strategies to support expeditious regulatory submissions for REQORSA and will implement the following changes to the Company's oncology clinical development plans in order to prioritize resources and focus on the most promising aspects of the Acclaim-1 and Acclaim-3 lung cancer clinical trials.
Maxim downgraded Dare Bioscience to Hold from Buy without a price target. While Dare continues to make some progress in its Phase 3 Ovaprene study, the halfway mark of enrollment won't complete the six months of Ovaprene use until the end of 2Q25, and there is no clarity on timing for top-line data, the analyst tells investors in a research note. In addition, the topical sildenafil program for female sexual arousal disorder is planning to move to a Phase 3 program, but Dare is still waiting for additional feedback from the FDA, Maxim says. With runway into 1Q25 and a lack of meaningful catalysts, Maxim believes Dare will likely need to raise additional capital.
Orgenesis announced a strategic partnership agreement with Harley Street Healthcare Group. This partnership aims to accelerate the clinical development and commercialization of advanced wellness and longevity therapies and products to a global customer base, aimed at significantly reducing the impact of lifestyle-related illnesses on people's health. Under the terms of the agreement, Orgenesis and HSHG will establish a joint venture, with Orgenesis owning 49% and HSHG owning 51% of the entity. The JV will focus on launching innovative health and wellness services, including personalized preventative care and regenerative therapies. The goal is to establish a comprehensive "Health-Wellness-as-a-Service" model, leveraging HSHG's established healthcare network and Orgenesis' cutting-edge biotech innovations. The JV intends to introduce a suite of wellness and longevity products by the end of 2024 that will include immune cell banking, aging and longevity therapies, preventative illness screening, and regenerative therapies using stem cells. The initial rollout will target key global regions, including the United Kingdom, UAE, MENA, Canada, ASEAN, the Balkans, Africa, Latam, and the Indian subcontinent. In connection with the agreement, HSHG has agreed to invest up to $10 million over three years into Orgenesis and the joint venture.
August 13
Dare Bioscience announced that additional data from the exploratory Phase 2b RESPOND clinical study of Sildenafil Cream, 3.6%, an investigational topical cream formulation of sildenafil being developed as an on-demand treatment for female sexual arousal disorder, has been published by The Journal of Sexual Medicine. "In this first-of-its-kind study in FSAD patients, we are very pleased to be able to share these data," said Sabrina Martucci Johnson, President and CEO of Dare Bioscience. "Being able to show consistent results from patient reported outcome measures, regardless of whether she is recalling her experiences over the last month or the last day, provides important optionality as we consider how to clinically measure the impact of Sildenafil Cream on patients with FSAD."
Organigram reported Q3 revenue of C$41.1M vs. C$32.8M last year, and reported Q3 adjusted EBITDA C$3.5M vs. (C$2.9M) last year. "We are pleased to report a strong third quarter, highlighted by a 25% year-over-year increase in net revenue, and a significant improvement in adjusted EBITDA", said CEO Beena Goldenberg. "Our strategic investments and partnerships, both domestically and internationally, have positioned us for growth and diversification, particularly in the European market with our investment in Sanity Group. Furthermore, the preliminary results from our landmark PK study on our latest patent pending nanoemulsion technology demonstrate our ongoing commitment to innovation and expanding our product offerings. I continue to be very proud of our dedicated team for their hard work and contributions to these achievements." CFO Greg Guyatt commented, "We are pleased with the results of our focus on operating efficiencies and resulting margin improvements and positive adjusted EBITDA in the quarter. With $173M in pro-forma cash, we believe we have one of the strongest balance sheets in the industry and are well positioned to capitalize on future growth opportunities."
August 12
Syros Pharmaceuticals announced that it will discontinue enrollment in the SELECT-AML-1 Phase 2 clinical trial evaluating the triplet regimen of tamibarotene in combination with venetoclax and azacitidine compared to the doublet regimen of venetoclax and azacitidine in newly diagnosed, unfit patients with acute myeloid leukemia and RARA gene overexpression. This decision is based on the results of a prespecified interim analysis of the trial. Data from 51 patients enrolled in SELECT-AML-1 were reviewed on August 9, 2024. This review included a prespecified non-binding futility analysis conducted on the first 40 randomized patients after the fortieth randomized patient received approximately three months of study drug or discontinued treatment. A similar complete response/complete response with incomplete hematologic recovery rate was observed in the triplet arm and the doublet arm. As a result, the probability for success of the SELECT-AML-1 study to demonstrate superiority at the final analysis in 80 randomized patients was considered low, and Syros made the decision to discontinue further enrollment. There were no new safety signals associated with the use of tamibarotene in combination with venetoclax and azacitidine. Patients currently enrolled in SELECT-AML-1 will have the opportunity to remain on study at the discretion of study investigators. Syros plans to present data from SELECT-AML-1 at the 12th Annual Meeting of the Society of Hematologic Oncology in September 2024. "We are disappointed by this unexpected outcome, especially for people living with AML," said David A. Roth, M.D., Chief Medical Officer of Syros. "In our prior Phase 2 clinical trial, the doublet combination of tamibarotene and azacitidine delivered a 61% CR/CRi rate in newly diagnosed AML patients with RARA overexpression. This supports our conviction in pursuing a doublet strategy in higher-risk MDS, where we are comparing tamibarotene and azacitidine to azacitidine alone. We remain steadfast in our commitment to delivering tamibarotene for the treatment of HR-MDS and look forward to sharing pivotal data from SELECT-MDS-1 by mid-fourth quarter."
Crown Laboratories and Revance Therapeutics announced that they have entered into a merger agreement pursuant to which the companies seek to merge the two complementary organizations. Under the terms of the agreement, which has been unanimously approved by Revance's board, Crown will commence a tender offer to acquire all outstanding shares of Revance's common stock for $6.66 per share in cash, representing a total enterprise value of $924M. The purchase price represents a premium of 89% over Revance's closing market price on August 9 and a 111% premium to Revance's 60-day volume-weighted average price. Upon completion of the transaction, Crown Laboratories expects to be one of the leading global aesthetics and skincare companies in an attractive, high-growth market, with an industry-leading portfolio of 10+ cutting-edge skin health and aesthetic brands, and one of the largest distribution footprints in skincare across medical, retail and e-commerce channels. The transaction is expected to close by year end. Following completion of the merger, Revance will be wholly owned by Crown and Revance's stock will no longer be publicly traded on Nasdaq. The transaction is subject to stockholders validly tendering shares representing at least a majority of the voting power of Revance, required regulatory approvals and other customary closing conditions.
Ascendis Pharma announced that the FDA has approved YORVIPATH for the treatment of hypoparathyroidism in adults. YORVIPATH is a prodrug of parathyroid hormone, administered once daily, designed to provide continuous exposure to released PTH over the 24-hour dosing period. Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone that impact multiple organs and affects an estimated 70,000 to 90,000 people in the United States.
August 8
Trevena announced that it will effect a reverse stock split of its common stock at a ratio of 1-for-25. The reverse stock split will become effective on Tuesday, August 13. Trevena's common stock will continue to be traded on the Nasdaq Capital Market under the symbol "TRVN" and will begin trading on a split-adjusted basis when the market opens on Tuesday, August 13. The reverse stock split is intended to enable Trevena to regain compliance with the $1.00 minimum bid price required for continued listing on the Nasdaq Capital Market.
Cantor Fitzgerald raised the firm's price target on Mirum Pharmaceuticals to $60 from $50 and kept an Overweight rating on the shares. Mirum reported Q2 earnings results with a slight top-line beat for both Livmarli and total revenue, the analyst told investors in a research note. The company announced a new Livmarli basket trial addressing a group of ultra-rare pruritic cholestatic diseases, which the company conservatively estimates affects more than 500 patients in the U.S., Cantor added.
Baird raised the firm's price target on Mirum Pharmaceuticals to $44 from $39 and kept an Outperform rating on the shares. The firm said Q2 results were broadly in line with expectations as livmarli performed as expected, and noted that management reaffirmed net product sales guidance for the year.
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About "Biotech Alert"
The Fly will report on a selection of biotech stocks seeing a surge in interest from retail and financial professional investors, based on data from InvestingChannel.
This Fly exclusive recap reveals the biotech stocks that are seeing a spike in searches among the 20-plus million retail and financial professional investors through InvestingChannel's online financial news media ecosystem.
This increased attention from the investors may be in response to, or advance of, outsized moves for stocks in the biotech sector, which tend to be volatile and prone to sharp swings in share price around binary events such as clinical study results and FDA approvals.
