Biotech Alert: Searches spiking for these stocks today

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include: 

• RedHill Biopharma (RDHL), 35,346% surge in interest
• Avidity Biosciences (RNA), 3,487% surge in interest
• Springworks Therapeutics (SWTX), 1,007% surge in interest
• Xenon Pharmaceuticals (XENE), 842% surge in interest
• Argenx (ARGX), 795% surge in interest
• Abeona Therapeutics (ABEO), 557% surge in interest
• Journey Medical (DERM), 467% surge in interest
• Cabaletta Bio (CABA), 461% surge in interest
• Elevai Labs (ELAB), 459% surge in interest
• Axcella Health (AXLA), 208% surge in interest

Pipeline and key clinical candidates for these companies:

RedHill Biopharma is primarily focused on gastrointestinal and infectious diseases. RedHill promotes the gastrointestinal drugs, Movantik for opioid-induced constipation in adults, Talicia for the treatment of Helicobacter pylori infection in adults, and Aemcolo for the treatment of travelers’ diarrhea in adults.

Avidity Bioscience’s proprietary AOCs – Antibody Oligonucleotide Conjugates – are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics, the company says. Avidity’s advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 and is currently in Phase 1/2 development with the ongoing MARINA and MARINA-OLE trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy and is currently in Phase 1/2 development with the FORTITUDE trial. AOC 1044 is designed for people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44 trial.

SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. Ogsiveo, approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is SpringWorks’ first FDA-approved therapy. 

Xenon Pharmaceuticals is a clinical stage biopharmaceutical company committed to developing innovative therapeutics to improve the lives of patients with neurological disorders. The company is advancing a novel product pipeline of neurology therapies to address areas of high unmet medical need, with a focus on epilepsy.

Argenx is an immunology company that says it is “committed to improving the lives of people suffering from severe autoimmune diseases” and “aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines.” Argenx developed and is commercializing what it calls “the first-and-only approved neonatal Fc receptor blocker in the U.S., the EU and UK, and Japan.” The company is evaluating efgartigimod in multiple autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises.

Abeona Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona’s lead clinical program is pz-cel, its investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona’s fully integrated cell and gene therapy cGMP manufacturing facility produced pz-cel for the pivotal Phase 3 VIITAL study and is capable of clinical and potential commercial production of AAV-based gene therapies.

Journey Medical says it is focused on identifying, acquiring, developing and strategically commercializing “innovative, differentiated dermatology products through its efficient sales and marketing model.” The company currently markets eight branded and three generic products that help treat and heal common skin conditions.

Caballeta Bio is focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA platform encompasses chimeric antigen receptor T cells for autoimmunity and Cabaletta Bio’s proprietary chimeric autoantibody receptor T cells.

Elevai Labs is a medical aesthetics company developing cutting-edge physician-dispensed skin care applications. The company solves unmet needs in the medical aesthetics space through a combination of cutting-edge science-driven and next-generation consumer applications. Elevai Labs develops topical aesthetic skin care cosmetic products for the physician-dispensed market, with a focus on leveraging a proprietary stem cell exosome technology.

Axcella is “pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators,” or EMMs, the company states. The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID, NASH, and the reduction in risk of OHE recurrence.

Recent news on these stocks:

November 29

Elevai Labs announced that they have signed a licensing agreement with Yuva Biosciences to research and develop means to incorporate Yuva Bioscience’s mitochondrial research with ELEVAI’s PREx to potentially develop topical skincare formulations that combine mitochondrial biology with Elevai’s human umbilical mesenchymal stem cell derived exosomes. Elevai currently markets its proprietary ELEVAI E-Series which are peri-procedure topical exosome skincare products. Yuva Biosciences utilizes mitochondrial science to address signs of aging of hair, scalp, and skin. “We are excited to partner with Yuva Biosciences in order to begin research and development of products that may incorporate the power of mitochondrial technology to further advance the potential of Elevai exosomes to address cosmetic skin and hair concerns associated with signs of aging,” said Jordan R. Plews PhD and CEO of ELEVAI Labs.

November 28

Avidity Biosciences announced a global licensing and research collaboration with Bristol Myers Squibb (BMY) focused on the discovery, development and commercialization of multiple cardiovascular targets with potential cumulative payments of up to $2.3B. Antibody Oligonucleotide Conjugates, or AOCs, are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. This strategic collaboration broadens the reach of AOCs through the expansion of the existing relationship with Bristol Myers Squibb. Avidity continues to advance its internal research and development programs in rare cardiac indications. Under the terms of the agreement, Avidity will receive $100M upfront, which includes a $60M cash payment as well as the purchase of approximately $40M of Avidity common stock at a purchase price of $7.88 per share. Avidity is also eligible to receive up to approximately $1.35B in research and development milestone payments, up to approximately $825M in commercial milestone payments, and tiered royalties up to low double-digits on net sales. Bristol Myers Squibb will fund all future clinical development, regulatory and commercialization activities coming from this collaboration.

Argenx announced topline results from the ADVANCE-SC study evaluating Vyvgart Hytrulo in adults with primary immune thrombocytopenia, or ITP. The study did not meet the primary endpoint of a sustained platelet count response in chronic ITP patients. Additional analyses of the dataset are ongoing and the full results will be presented at an upcoming medical meeting and in a peer-reviewed publication. ADVANCE-SC is the second of two registrational trials conducted as part of the ongoing ITP development program for Vyvgart and enrolled 207 adult patients with chronic and persistent ITP. Patients were heavily pre-treated and 75% of patients had received three or more prior ITP therapies. Primary endpoint was not met; 13.7% of treated patients demonstrated a sustained platelet count response compared to 16.2% of placebo patients. Secondary endpoints were not met, including additional endpoints on International Working Group, or IWG, responder status and mean platelet count change from baseline. Vyvgart Hytrulo was well-tolerated in ADVANCE-SC; the observed safety and tolerability profile was consistent with ADVANCE-IV and the confirmed safety profile of Vyvgart and Vyvgart Hytrulo. Results from the first study in the ITP registrational program, ADVANCE-IV, were reported in May 2022. The study met its primary and key platelet-derived secondary endpoints. ADVANCE-IV formed the basis of the regulatory submission for approval of Vyvgart IV for ITP in Japan, where a decision is expected in the first quarter of 2024. Vyvgart is currently being evaluated in 13 severe autoimmune diseases, including the registrational ADDRESS study for pemphigus from which topline results are expected around year-end 2023.

Cantor Fitzgerald wrote in a research note that Cabaletta Bio and other stocks were down following news that the FDA is investigating the link between CAR-T and T-cell malignancies with approved products in oncology. While the report might slow down progress at some centers, this is “likely a storm in a tea cup,” as INDs are still being approved, trials are still proceeding, unmet need remains high, the analyst tells investors in a research note. The firm, which made no change to its Overweight rating or $50 price target on Cabaletta Bio, is not going to be spooked by headlines that say the “FDA is looking into this,” as it has already accepted many development risks to its thinking.

November 27

RedHill Biopharma announced that the FDA has granted five years’ market exclusivity for Talicia under the Generating Antibiotic Incentives Now Act Qualified Infectious Disease Product designation, as recommended by the FDA Exclusivity Board. This grant is on top of three years’ exclusivity granted for the approval of Talicia under section 505(b)(2). Talicia is protected by its broad intellectual property suite to 2034. “Talicia is the only FDA-approved rifabutin-based therapy for the eradication of H. pylori. Its components and formulation are optimized to provide patients with the necessary medications for successful H. pylori eradication, an innovation rewarded by this market exclusivity and other IP, providing protection to 2034,” said Patricia Anderson, RedHill’s senior VP of Regulatory Affairs. “With significant concerns around maintaining effective therapeutic options for infectious diseases in the face of growing microbial resistance, it is vital that the most effective options are utilized first line. Talicia’s favorable efficacy, tolerability, and resistance profile, in the only all-in-one formulation available, provides potential advantages over clarithromycin-based regimens for most patients.” The FDA recently approved a supplemental new drug application for Talicia, allowing a change to a more flexible three times daily, taken at least 4 hours apart with food, dosing regimen for H. pylori eradication enabling patients to follow a convenient “breakfast, lunch and dinner” dosing routine, which may support increased patient adherence and optimize the potential for successful H. pylori eradication.

Xenon Pharmaceuticals reported topline results from the randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept X-NOVA clinical trial, which evaluated the clinical efficacy, safety, and tolerability of 10 mg and 20 mg of XEN1101 in 168 patients with moderate to severe major depressive disorder, or MDD. Summary of Efficacy Data: The primary endpoint of the study was a change in the Montgomery-Asberg Depression Rating Scale, or MADRS, at week 6. The mean reduction was 13.90 in the placebo group, 15.61 in the XEN1101 10 mg group and 16.94 in the XEN1101 20 mg group. A clear dose response and a clinically meaningful, but not statistically significant, 3.04 difference between placebo and the XEN1101 20 mg group was observed. Statistical significance was achieved on the pre-specified endpoint of the Hamilton Depression Rating Scale, or HAM-D17, at week 6 with a mean reduction of 10.18 in the placebo group and 13.26 in the XEN1101 20 mg group. Statistical significance was achieved on the key secondary endpoint of a change in the Snaith-Hamilton Pleasure Scale, or SHAPS, measuring anhedonia at week 6 with a reduction of 5.30 in the placebo group and 7.77 in the XEN1101 20 mg group. Statistical significance was achieved in MADRS at week 1 with a mean reduction of 4.88 in the placebo group and 7.54 in the XEN1101 20 mg group demonstrating early onset of efficacy. Statistical significance was achieved in reporting of at least minimally improved symptoms of depression as assessed by physicians using the Clinical Global Impression of Improvement in the XEN1101 20 mg group compared to placebo. Summary of Safety and Tolerability Data: XEN1101 was well tolerated with similar rates of adverse events reported across all treatment arms. The most commonly reported treatment-emergent adverse events in the XEN1101 20 mg group included dizziness, somnolence, headache and disturbance in attention, as compared to the placebo group which reported dizziness, somnolence, headache and disturbance in attention. Rates of discontinuation were similar across all treatment arms and rates of discontinuation due to TEAEs were low with three patients in the XEN1101 20 mg group, as compared to two patients in the placebo group. No serious adverse events were reported in the two XEN1101 treatment groups and there were two patients in the placebo group who experienced a treatment-emergent SAE. XEN1101 was not associated with clinically meaningful weight gain or sexual dysfunction.

Abeona Therapeutics announced that the FDA has accepted and granted Priority Review for the Biologics License Application, BLA for pz-cel, Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa. Under the Prescription Drug User Fee Act, PDUFA, the FDA has set a target action date of May 25, 2024. The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application. “The FDA’s acceptance of our BLA for priority review underscores the high unmet need in RDEB and the potential for pz-cel to provide meaningful benefit to these patients,” said Vish Seshadri, CEO of Abeona. “We thank the FDA for their commitment and look forward to working with them through the BLA review, with the goal of bringing this therapy to patients as soon as possible.”

SpringWorks Therapeutics announced that the FDA has approved OGSIVEO, an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. The FDA previously granted breakthrough therapy, fast track and orphan drug designations to nirogacestat for the treatment of desmoid tumors.

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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.