These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
- MediciNova (MNOV), 3,933% surge in interest
- Rapt Therapeutics (RAPT), 625% surge in interest
- Syros Pharmaceuticals (SYRS), 452% surge in interest
- Fulcrum Therapeutics (FULC), 432% surge in interest
- Heron Therapeutics (HRTX), 419% surge in interest
- Citius Pharmaceuticals (CTXR), 410% surge in interest
- Phathom Pharmaceuticals (PHAT), 335% surge in interest
- Repligen (RGEN), 315% surge in interest
- Altimmune (ALT), 303% surge in interest
- Axsome Therapeutics (AXSM), 268% surge in interest
Pipeline and key clinical candidates for these companies:
MediciNova is a clinical-stage biopharmaceutical company developing a broad late-stage pipeline of novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases. Based on two compounds, MN-166 and MN-001, with multiple mechanisms of action and strong safety profiles, MediciNova has 11 programs in clinical development. MediciNova’s lead asset, MN-166, is currently in Phase 3 for amyotrophic lateral sclerosis and degenerative cervical myelopathy and is Phase 3-ready for progressive multiple sclerosis. MN-166 is also being evaluated in Phase 2 trials in Long COVID and substance dependence. MN-001 was evaluated in a Phase 2 trial in idiopathic pulmonary fibrosis and a second Phase 2 trial in non-alcoholic fatty liver disease is ongoing.
Rapt Therapeutics is focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology. Rapt has discovered and advanced two unique drug candidates, RPT193 and FLX475, each targeting C-C motif chemokine receptor 4, for the treatment of inflammation and cancer, respectively. The company is also pursuing a range of targets that are in the discovery stage of development.
Syros says it 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.
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum’s two lead programs in clinical development are losmapimod, a small molecule for the treatment of facioscapulohumeral muscular dystrophy and FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and other hemoglobinopathies, including beta-thalassemia. The company’s proprietary product engine, FulcrumSeek, identifies drug targets that can modulate gene expression to treat the known root cause of gene mis-expression.
Heron Therapeutics is a commercial-stage biotechnology company that says it is developing “novel, patient-focused solutions that apply its innovative science and technologies to already-approved pharmacological agents for patients suffering from pain or cancer.”
Citius Pharmaceuticals is a late-stage biopharmaceutical company focused on the development and commercialization of first-in-class critical care products, with a pipeline of anti-infectives in oncology, adjunct cancer care, stem cell therapy and unique prescription products. Its diversified pipeline of therapies targeting unmet medical needs includes three potential first-and-only prescription treatments in their indications, and a next-generation single-donor, allogeneic, clonal and scalable stem cell therapy program.
Phathom Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing novel treatments for gastrointestinal diseases. The company has licensed the exclusive rights in the U.S., Europe, and Canada to vonoprazan, a first-in-class potassium-competitive acid blocker. Phathom has assembled a team of seasoned gastrointestinal and pharmaceutical industry experts with deep expertise in developing GI therapeutics, including anti-secretory agents.
Repligen Corporation develops and commercializes bioprocessing technologies and systems that enable efficiencies in the process of manufacturing biological drugs. The customers served are primarily biopharmaceutical drug developers and contract development and manufacturing organizations, or CDMOs.
Altimmune is a clinical-stage biopharmaceutical company that says it is focused on developing “innovative next-generation therapeutics for the treatment of patients with liver diseases and obesity.” The company’s lead product candidate, pemvidutide, is a GLP-1/glucagon dual receptor agonist that is being developed for the treatment of obesity and NASH. In addition, Altimmune is developing HepTcell, an immunotherapeutic designed to achieve a functional cure for chronic hepatitis B.
Axsome Therapeutics is developing and delivering novel therapies for central nervous system, or CNS, conditions that have limited treatment options. AXS-05 is a novel, oral, patent protected, investigational N-methyl-D-aspartate receptor antagonist with multimodal activity under development for the treatment of Alzheimer’s disease agitation and other central nervous system disorders.
Recent news on these stocks:
November 13
Fulcrum Therapeutics reported Q3 EPS of (35c) against a consensus of (41c). “In the third quarter, we made progress enrolling patients and activating new sites in the Phase 1b PIONEER trial of pociredir in sickle cell disease and advancing our preclinical pipeline,” said CEO Alex Sapir. “The need for effective therapeutic options for patients with sickle cell disease has become even more urgent due to the recent withdrawal of OXBRYTA globally. Based on the initial data generated in the Phase 1b trial, we believe that pociredir has the potential to increase fetal hemoglobin to levels that could ameliorate symptoms of sickle cell disease. While we were disappointed with the results of the Phase 3 REACH trial announced in September, we remain committed to improving the lives of patients with genetically defined diseases in areas of high unmet medical need like sickle cell disease. We are focused on progressing the development of pociredir as expeditiously as possible and remain on track to provide data from the PIONEER trial in 2025.”
November 12
Heron Therapeutics reported Q3 EPS of (3c) against a consensus of (3c), and reported Q3 revenue of $32.81M against a consensus of $36.93M. “We are pleased to report that with our prudent financial management and continued revenue growth we were able to achieve positive adjusted EBITDA for the quarter,” said CEO Craig Collard. “The future looks bright as we continue to grow all product revenue. The CrossLink partnership roll out, FDA approval of the VAN in September, and inclusion in the CMS Final Rule Non-Opioid Policy for Pain Relief, positions ZYNRELEF for significant growth within the surgical setting. We continue to deliver on our commitment to financial efficiency while growing revenue. The team has made great strides in the transformation of Heron over the past year. We are looking forward to a strong Q4 which is off to a great start.” Heron Therapeutics narrowed its FY24 revenue view to $140M-$146M from $138M-$158M, against a consensus of $148.4M.
Repligen reported Q3 adjusted EPS of 43c against a consensus of 33c, and reported Q3 revenue of $154.871M against a consensus of $153.23M. CEO Olivier Loeillot said, “I’m pleased to report strong third quarter results, made possible by the excellent execution by our team and improving market conditions. We are encouraged to see strengthening CDMO and equipment sales in the quarter, with each delivering year-over-year and sequential growth. In new modalities, we achieved record sales in the quarter and our differentiated technology continues to serve us well. We are further encouraged by continued strength in Filtration, consumables, and Pharma, as well as order recovery in Chromatography. With clear line of sight, we are narrowing the 2024 revenue guidance shared in our September update, maintaining the midpoint, and we are optimistic about our opportunity funnel as we move into 2025.”
Altimmune reported Q3 EPS of (32c) against a consensus of (35c), and reported Q3 revenue of $5,000 vs $362,000 last year. “In the third quarter, we reached several important milestones, most notably the completion of enrollment in the Phase 2b IMPACT trial of pemvidutide in MASH, positioning us to report top-line efficacy data in the second quarter of 2025,” said CEO Vipin K. Garg, Ph.D. “Further, we successfully completed our End-of-Phase 2 meeting with the FDA for the pemvidutide Phase 3 obesity program, gaining agreement on the design of the pivotal studies as well as the measures of efficacy and safety.”
Axsome Therapeutics reported Q3 EPS of ($1.34) against a consensus of ($1.41), and reported Q3 revenue of $104.8M against a consensus of $98.1M. “In the third quarter, we continued our strong commercial performance and advanced our innovative, industry-leading, late-stage development pipeline towards important near-term milestones,” said CEO Herriot Tabuteau, MD. “In response to continued strong demand growth, a second expansion of the Auvelity sales force is planned for the first quarter of 2025. In addition, with the January 31, 2025, PDUFA date for our AXS-07 product candidate for migraine fast approaching, commercial preparations are underway for a timely and successful launch, if approved. We expect a busy end to the year with several clinical catalysts anticipated, including a planned simultaneous release of topline results from the ongoing Phase 3 ADVANCE-2 and ACCORD-2 trials of AXS-05 in Alzheimer’s disease agitation in the fourth quarter. Our growth as an organization positions us well to potentially deliver multiple innovative new medicines to the millions of individuals living with central nervous system disorders in the U.S. Importantly, we have the resources in hand to execute our operating plans and create substantial value for shareholders.”
November 11
MediciNova was notified by Sanofi (SNY) that the Sanofi/Novartis (NVS) litigation was settled. Accordingly, MediciNova is entitled to receive a certain amount of monetary damages through this settlement. Further details will be provided as they become available.
Rapt Therapeutics announced it is terminating its zelnecirnon program. Zelnecirnon was being evaluated in two randomized, placebo-controlled Phase 2 clinical trials in asthma and atopic dermatitis, and both trials were placed on clinical hold by the U.S. Food and Drug Administration in February 2024 due to a serious adverse event of liver injury requiring transplant in one patient in the AD trial. No liver toxicity nor other treatment-related SAEs were reported in any other trial participant. The company subsequently closed both studies before completing the planned enrollment. Following feedback recently received from the FDA, the company has stopped its zelnecirnon program. “In light of the agency’s feedback, we do not see a viable path forward for zelnecirnon, although we continue to believe that CCR4 remains an exciting target with the potential to provide a safe, oral therapeutic option across a number of inflammatory diseases,” commented Brian Wong, M.D., Ph.D., President and CEO of RAPT. “We plan to continue advancing our next generation CCR4 compounds with improved safety margins for inflammatory disease and expect to identify a new candidate in the first half of 2025. Additionally, we continue to actively pursue in-licensing opportunities for clinical-stage assets.”
Syros Pharmaceuticals announced that the SELECT-MDS-1 Phase 3 trial evaluating tamibarotene in combination with azacitidine in newly diagnosed higher-risk myelodysplastic syndrome patients with RARA gene overexpression did not meet its primary endpoint of complete response rate. Tamibarotene is Syros’ proprietary oral, selective, retinoic acid receptor alpha agonist. In the first 190 enrolled patients, the CR rate by intent-to-treat in the tamibarotene/azacitidine treatment arm was 23.8% compared to a CR rate of 18.8% in the placebo/azacitidine control arm and was not statistically significant. In the safety analysis of all enrolled patients, tamibarotene in combination with azacitidine appeared to be generally well-tolerated, with an adverse event profile that was similar to that seen in earlier Syros-sponsored studies. Syros also reported that, as previously disclosed in its filings with the SEC, the failure of the SELECT-MDS-1 trial to achieve its primary endpoint constitutes an event of default under its secured loan facility with Oxford Finance LLC. “We are deeply disappointed by this outcome, particularly for the HR-MDS patients who are seeking a new treatment option for this challenging disease,” said Conley Chee, Chief Executive Officer of Syros. “We plan to stop the study, review the clinical data more thoroughly, and evaluate the next steps. We want to express our sincere appreciation for the patients, caregivers and healthcare professionals who took part in the SELECT-MDS-1 trial and to all the employees of Syros for their exceptional work on the tamibarotene program.”
Citius Pharmaceuticals and Citius Oncology (CTOR) announced “promising” preliminary results from an ongoing investigator-initiated Phase I clinical trial evaluating the safety and efficacy of a combined regimen of pembrolizumab and lymphir in patients with recurrent solid tumors. The trial aims to identify an optimal dose for future trials and explore the impact of a treatment regimen combining pembrolizumab and lymphir on the tumor immune microenvironment. Pembrolizumab, developed by Merck (MRK) and sold under the brand name keytruda, is the leading PD-1 inhibitor. The results of this chemotherapy-free regimen combining two immuno-modulator agents, pembrolizumab and lymphir demonstrated: an overall response rate of 27% – 4/15 – and a clinical benefit rate of 33% among evaluable patients; and median progression-free survival for patients achieving clinical benefit of 57 weeks. Two of the four patients who achieved partial remission had received prior checkpoint inhibitors. The trial enrolled 21 patients with recurrent or metastatic solid tumors. Among the evaluable participants, four patients achieved a partial response, and one patient demonstrated durable stable disease lasting over six months. The combination regimen was generally well tolerated, with most adverse events related to the patients’ underlying disease. No significant immune-related adverse events were observed.
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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.