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:
Moleculin Biotech is focused on the development of a portfolio of drug candidates for the treatment of highly resistant tumors and viruses. The company's lead program, Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms with little to no cardiotoxicity. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia, or AML, and soft tissue sarcoma lung metastases.
Elevai Labs specializes in medical aesthetics and biopharmaceutical drug development, focusing on innovations for skin aesthetics and treatments tied to obesity and metabolic health. Driven by a commitment to scientific research, Elevai aims to transform personal health and beauty, the company states.
Wave Life Sciences is a biotechnology company "focused on unlocking the broad potential of RNA medicines to transform human health" whose RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders, the company says. Wave's pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington's disease, as well as a preclinical program in obesity.
Seelos Therapeutics is focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system, or CNS, disorders and other rare diseases. The company's portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior in Major Depressive Disorder, amyotrophic lateral sclerosis, or ALS, and spinocerebellar ataxia, or SCA, as well as early-stage programs in Huntington's disease, Alzheimer's disease, and Parkinson's disease.
Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. Biohaven's clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; and antibody recruiting, bispecific molecules and antibody drug conjugates for cancer.
Capricor Therapeutics is a biotechnology company that says it is "dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases." Lead product candidate, deramiocel, is an allogeneic cardiac-derived cell therapy that is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy.
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.
Marinus is a commercial stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The company's commercial product, Ztalmy oral suspension CV, has been approved by the FDA for the treatment of seizures associated with CDKL5 deficiency disorder in patients two years of age and older. The potential of ganaxolone is also being studied in other rare seizure disorders, including in Phase 3 trials in tuberous sclerosis complex and refractory status epilepticus.
Regeneron is a biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. The company's medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.
Genmab has invented next-generation antibody technology platforms and leveraged translational research and data sciences, which has resulted in a proprietary pipeline including bispecific T-cell engagers, next-generation immune checkpoint modulators, effector function enhanced antibodies and antibody-drug conjugates. To help develop and deliver novel antibody therapies to patients, Genmab has formed 20+ strategic partnerships with biotechnology and pharmaceutical companies.
Recent news on these stocks:
September 25
A Wave Life Sciences 23.125M share Secondary priced at $8.00. The deal size was increased to $200M from $175M and the deal priced below its last closing price of $9.01. JPMorgan and Leerink acted as joint book running managers for the offering.
Seelos Therapeutics announced that its board approved a 1-for-16 reverse stock split of its outstanding shares of common stock, to be effective on Friday, September 27. The company's common stock will begin trading on a reverse stock split-adjusted basis at the opening of the market on Friday, September 27. Following the reverse stock split, the company's common stock will continue to trade on the Nasdaq Capital Market under the symbol "SEEL" with the new CUSIP number, 81577F 406. The reverse stock split is intended for the company to regain compliance with the minimum bid price requirement of $1.00 per share of common stock for continued listing on the Nasdaq Capital Market. The reverse stock split was approved by the company's board.
September 24
Capricor Therapeutics announced, following recent meetings with the FDA, its intent to file a Biologics License Application, BLA, based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy cardiomyopathy. Following the FDA meetings: Capricor plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024. The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children's Hospital Medical Center. In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in Q4 of 2024.
September 23
Moleculin Biotech announced the recent presentation of positive in vivo efficacy data of Annamycin in orthotopic and experimental lung metastatic models of sarcoma. Key Highlights: Annamycin demonstrated high uptake and retention in lung parenchyma of mice and rats. The therapeutic effects of doxorubicin, DOX, are diminished due to low lung DOX uptake as demonstrated in the tested in vivo models. In contrast, Annamycin exhibits consistent efficacy in vivo in orthotopic and experimental lung metastatic models of sarcoma, breast, and colon cancer. This correlated with high Annamycin concentration in lungs, which exceeded DOX levels by 10- to 30-fold. Preclinical tests clearly demonstrate a better cardiac safety profile of Annamycin when compared to DOX and no cardiotoxicity of Annamycin in the in vivo models. No cardiotoxicity of Annamycin has been noted in ongoing clinical studies. The observed organotropic properties of Annamycin, its efficacy in vivo, and its promising safety profile warrant further translational studies to evaluate Annamycin in patients with primary or metastatic lung cancers, as a single agent and in combination with currently used therapeutics.
Elevai Labs has priced a public offering with gross proceeds to the company expected to be approximately $8M, before deducting placement agent fees and other estimated expenses payable by the company. The offering is a best-efforts offering, with no minimum amount of securities required to be sold. The offering is comprised of 28.57M shares of the company's common stock. This offering is expected to close on or about September 24, subject to satisfaction of customary closing conditions. Univest Securities is acting as sole placement agent for the offering.
Biohaven announced positive topline results from pivotal Study BHV4157-206-RWE demonstrating the efficacy of troriluzole on the mean change from baseline in the f-SARA after 3 years of treatment. The study achieved the primary endpoint and showed statistically significant improvements on the f-SARA at years 1 and 2. Collectively, data across multiple analyses demonstrate a robust and clinically meaningful slowing of disease progression in SCA patients. These treatment benefits translate into a 50-70% slower rate of decline compared to untreated patients, representing 1.5-2.2 years delay in disease progression over the 3-year study period. Additionally, in a responder sensitivity analysis, disease progression when defined by a 2 point or greater worsening on the f-SARA at 3 years showed an odds ratio of 4.1 for the untreated external control arm versus troriluzole treated subjects.
Oppenheimer upgraded Marinus Pharmaceuticals to Outperform from Perform with a $6 price target. The firm views the risk/reward for the shares as attractive ahead of the Phase 3 TrustTSC topline results expected in early Q4. Key opinion leaders are expressing confidence in the trial design and likelihood for clinically meaningful efficacy, and a slower titration schedule compared to Phase 2 should improve both tolerability and efficacy, the analyst tells investors in a research note. Opco's scenario analysis forecasts 75% probability adjusted upside for the shares.
September 20
Regeneron Pharmaceuticals and Sanofi (SNY) announced that the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion recommending the expanded approval of Dupixent in the European Union for eosinophilic esophagitis n children down to 1 year of age. The recommendation is for children aged 1 to 11 years who weigh at least 15 kg and who are inadequately controlled by, intolerant to, or who are not candidates for conventional medicinal therapy. The European Commission is expected to announce a final decision in the coming months. Dupixent is already approved in the EU for certain adults and adolescents aged 12 years and older with EoE. The positive CHMP opinion is supported by a two-part EoE KIDS Phase 3 trial in children aged 1 to 11 years. In Part A, a significantly greater proportion of children receiving weight-based doses of Dupixent achieved histological disease remission at week 16, compared to placebo, with results sustained for up to one year in Part B. At week 16, caregivers of children treated with Dupixent also observed improvements in the frequency and severity of EoE signs, and fewer days with at least one sign of EoE, compared to placebo. These data established a bridge showing the response to Dupixent in children with EoE is similar to that of the approved adult and adolescent EoE populations. The safety results in the EoE KIDS trial were generally consistent with the known safety profile of Dupixent in adolescents and adults with EoE. AEs more commonly observed with Dupixent in either weight-based dosing regimen compared to placebo during Part A were COVID-19, nausea, injection site pain and headache. The long-term safety profile of Dupixent evaluated in Part B was similar to that observed during Part A. Results from the trial were recently published in The New England Journal of Medicine. The use of Dupixent in children aged 1 to 11 years with EoE is investigational in the EU and is not yet approved.
September 19
Genmab announced that it launched a partnership with OpenAI for 'AI Everywhere,' which looks to pioneer next-generation antibody therapies to treat cancer and other serious diseases. "Genmab's ambition is to integrate AI into everything we do," said Tahi Ahmadi, executive VP and Chief Medical Officer, Head of Experimental Medicines. "We anticipated AI to contribute significantly to the quality of our science, decision making, and efficiency in bringing medicines to patients." As a company that has recently tripled in size, Genmab wanted to use AI to address operational challenges-and develop new ways of working with vast amounts of complex scientific data, the company said. Just one month after ChatGPT Enterprise launched, Genmab rolled it out to 1,000 employees. Now, less than a year later, the company announced that it has launched "AI Everywhere" to give ChatGPT access to almost everyone at the company-more than 2,000 licenses. Genmab said it now has more than 100 custom GPTs that help with everything from drafting documents, summarizing scientific literature to simple and advanced analytics of data. "Our partnership with OpenAI not only highlights Genmab's commitment to innovation, but also aims to unleash every colleague's inner algorithmic leader to truly unlock large language model's potential and discover increasingly novel and progressive ways of leveraging AI to improve, and at times, re-imagine their tasks," said Hisham Hamadeh, SVP and Global Head of Data Sciences and AI at Genmab.
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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.
