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: 

• Ayala Pharmaceuticals (ADXS), 3,386% surge in interest
• Tenax Therapeutics (TENX), 3,213% surge in interest
• InVivo Therapeutics (NVIV), 2,577% surge in interest
• Equillium (EQ), 2,171% surge in interest
• Madrigal Pharmaceuticals (MDGL), 492% surge in interest
• Akero Therapeutics (AKRO), 403% surge in interest
• Exicure (XCUR), 388% surge in interest
• REGENXBIO (RGNX), 250% surge in interest
• Exelixis (EXEL), 249% surge in interest
• 89bio (ETNB), 228% surge in interest

Pipeline and key clinical candidates for these companies:

Ayala Pharmaceuticals is a clinical-stage oncology company primarily focused on developing and commercializing small molecule therapeutics for people living with rare tumors and aggressive cancers. Ayala is also developing proprietary Lm-based antigen delivery products for patients suffering from more common cancers. The company’s lead candidates under development are the oral gamma secretase inhibitor, AL102, for desmoid tumors; ADXS-504, a Lm-based therapy for early-stage prostate cancer; and the intravenous gamma secretase inhibitor, AL101, for adenoid cystic carcinoma. AL102 has received Fast Track Designation from the U.S. FDA and is currently in the Phase 3 segment of a pivotal study for patients with desmoid tumors.

Tenax Therapeutics is a specialty pharmaceutical company focused on identifying, developing, and commercializing products that address cardiovascular and pulmonary diseases with high unmet medical need. Tenax Therapeutics is developing a unique oral formulation of imatinib. The company also owns North American rights to develop and commercialize subcutaneous and oral formulations of levosimendan.

InVivo is focused on redefining the treatment of spinal cord injury. By modulating the healing environment to facilitate cell survival and growth, the company has the opportunity to change the treatment of spinal cord injury. “InVivo has steadily advanced its scaffold technology through years of research and development with encouraging clinical data. The team is united in its commitment to bring the Neuro-Spinal Scaffold to patients and to deliver meaningful improvements to their daily lives,” the company states.

Equillium is a clinical-stage biotechnology company working to develop novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need. The company’s pipeline consists of the following novel first-in-class immunomodulatory assets and product platform targeting immuno-inflammatory pathways. EQ101: a selective tri-specific cytokine inhibitor targeting IL-2, IL-9, and IL-15; currently under evaluation in a Phase 2 proof-of-concept clinical study of patients with alopecia areata being conducted in Australia and New Zealand by Equillium’s Australian subsidiary as the trial sponsor. EQ302: an orally delivered, selective bi-specific cytokine inhibitor targeting IL-15 and IL-21; currently in pre-clinical development. 

Madrigal Pharmaceuticals is a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis, or NASH, a liver disease with high unmet medical need. Madrigal’s lead candidate, resmetirom, is a once daily, oral, thyroid hormone receptor-beta selective agonist designed to target key underlying causes of NASH in the liver.

Akero Therapeutics is developing “transformational treatments for patients with serious metabolic diseases marked by high unmet medical need,” including NASH, a disease without any approved therapies. Akero’s lead product candidate, EFX, is a differentiated Fc-FGF21 fusion protein that has been engineered to mimic the balanced biological activity profile of native FGF21, an endogenous hormone that alleviates cellular stress and regulates metabolism throughout the body. EFX is currently being evaluated in two Phase 2b clinical trials: the HARMONY study in patients with pre-cirrhotic NASH and the SYMMETRY study in patients with cirrhotic NASH. EFX is also being evaluated in an expansion cohort of the SYMMETRY study, comparing the safety and tolerability of EFX to placebo when added to an existing GLP-1 receptor agonist in patients with pre-cirrhotic NASH and Type 2 diabetes.

Exicure is an early-stage biotechnology company historically focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. The company says it continues to actively pursue out-licensing opportunities for its clinical asset, cavrotolimod, as well as for its preclinical candidates, including the SCN9A program for neuropathic pain, and to pursue all strategic alternatives with the goal of maximizing stockholder value.

REGENXBIO is a clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a “5x’25” strategy to progress five AAV Therapeutics from its internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.

Exelixis is an oncology company that says it is “innovating next-generation medicines and regimens at the forefront of cancer care.” The company says it is rapidly evolving its product portfolio to target an expanding range of tumor types and indications with its clinically differentiated pipeline of small molecules, antibody-drug conjugates and other biotherapeutics.

89bio is a clinical-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases. The company’s lead product candidate, pegozafermin, is currently being developed for the treatment of NASH and SHTG.

Recent news on these stocks:

February 8

Madrigal Pharmaceuticals announced the publication of the pivotal Phase 3 MAESTRO-NASH trial of resmetirom in the New England Journal of Medicine. NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally. Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis. The FDA granted resmetirom Priority Review and assigned a Prescription Drug User Fee Act date of March 14, 2024, the target date by which FDA intends to complete its review. The MAESTRO-NASH trial evaluates resmetirom treatment vs. placebo in patients with NASH with significant fibrosis, a population at elevated risk of progressing to cirrhosis and other adverse liver outcomes. The study includes a 52-week biopsy assessment to support accelerated approval and an ongoing 54-month outcomes study designed to generate confirmatory data that, if positive, will help verify resmetirom’s clinical benefit and support full approval. Based on the results of the 52-week biopsy portion of the trial, MAESTRO-NASH is the only Phase 3 study in NASH to achieve both primary endpoints that FDA proposed as reasonably likely to predict clinical benefit: NASH resolution with no worsening of fibrosis and fibrosis reduction with no worsening of NAFLD activity score. Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement. More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis. In addition to the two primary endpoints, multiple secondary endpoints were achieved in the MAESTRO-NASH study, including statistically significant reduction from baseline in liver enzymes. Reductions in atherogenic lipids and lipoproteins, fibrosis biomarkers and imaging tests were observed in resmetirom treatment arms as compared with placebo. MAESTRO-NASH also included many biomarker and imaging assessments that may be used in real world clinical practice to identify appropriate patients for treatment and monitor response to resmetirom, if approved. The incidence of serious adverse events was similar across the treatment groups, 10.9%, 12.7%, and 11.5% in 80 mg, 100 mg and placebo groups respectively. Transient diarrhea and nausea were more frequent with resmetirom at the beginning of therapy. No increase in the incidence of diarrhea and nausea was noted among resmetirom-treated patients relative to placebo-treated patients after the first few weeks of treatment. There was no incidence of drug-induced liver injury. There were no increases in bone fractures, or fracture risk score with resmetirom or increase in adverse events related to thyroid hormone effects outside the liver such as heart rate changes or sex hormone abnormalities.

February 7

Tenax Therapeutics announced that the first patient has enrolled in the company’s Phase 3 LEVEL Study. As previously disclosed, the FDA does not require Tenax conduct a long-term, cardiovascular outcomes trial in this population, significantly reducing the costs and time for the registration of TNX-103. The extensive levosimendan patent estate includes protections through at least 2040 of all therapeutic doses of the proprietary oral formulation being evaluated in LEVEL, as well as I.V. and subcutaneous formulations, in patients with PH-HFpEF. We believe this positions Tenax, and potential strategic partners, to maximize the commercial potential of this unique, first-in-class therapy. The LEVEL study is the first of two planned Phase 3 studies that will evaluate TNX-103 for the treatment of pulmonary hypertension in heart failure with preserved ejection fraction. The LEVEL study will evaluate six-minute walking distance as the primary endpoint, and will enroll a total of 152 patients. The Phase 3 program for TNX-103 has been designed to exceed the minimal clinically important difference, and satisfy the FDA’s request for subject drug exposure of 300 patients for 6 months and 100 patients for 1 year.

REGENXBIO announced topline results from the Phase I/II/III CAMPSIITE trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II, also known as Hunter syndrome, demonstrating that the pivotal phase of the trial met its primary endpoint with statistical significance. The results were presented at the 20th Annual WORLDSymposium by Paul Harmatz, M.D., UCSF Benioff Children’s Hospital and trial investigator. In the pivotal phase, MPS II patients treated with RGX-121 achieved decreased cerebrospinal fluid levels of D2S6, a key biomarker of brain disease activity, below maximum attenuated disease levels at 16 weeks. Patients receiving RGX-121 demonstrated an 86% median reduction in D2S6, approaching normal levels. Pivotal results were consistent with data from the dose-finding phase of CAMPSIITE. In the dose-finding phase, the majority of patients are exceeding expectations in neurodevelopmental function compared to natural history data up to four years. New long-term follow-up of patients treated with RGX-121 in the dose-finding phase also showed there was a high rate of patients for whom trial investigators chose to discontinue standard-of-care intravenous enzyme replacement therapy or were allowed to remain ERT-naive. At the pivotal dose level, 80% of patients were ERT-free at last time point. As of January 3, 2024, RGX-121 continues to be well tolerated in 25 patients dosed across all phases of the CAMPSIITE trial. Following an RMAT meeting held with FDA at the end of 2023, REGENXBIO continues with plans to use CSF levels of D2S6 as a surrogate endpoint for accelerated approval and is completing remaining activities in order to file a BLA in the second half of 2024. Based on an expected priority review, potential approval of the planned BLA could result in receipt of a Rare Pediatric Disease Priority Review Voucher in 2025.

February 6

Immunome (IMNM) entered into a definitive asset purchase agreement with Ayala Pharmaceuticals, a clinical-stage oncology company, to acquire AL102 and related drug candidate AL101 from Ayala. Based on the terms of the agreement, Immunome will pay Ayala $20M in cash and $30M in Immunome common stock at the closing and will pay up to an additional $37.5M in development and commercial milestone payments. Completion of the transaction is subject to customary conditions including Ayala obtaining the requisite stockholder approval. AL102 is an investigational small molecule gamma secretase inhibitor currently being evaluated in the randomized Phase 3 RINGSIDE international trial for the treatment of desmoid tumors – a debilitating soft tissue malignancy. AL102 is a potential once-daily oral treatment for desmoid tumors. Data from clinical trials have shown AL102 may be more effective in treating desmoid tumors than OGSIVEO, which recently became the first treatment approved for desmoid tumors by the U.S. Food and Drug Administration in November 2023. The Phase 2 portion of the RINGSIDE study demonstrated clinically meaningful anti-tumor activity across multiple parameters. The data showed high objective response rates, including 75% of evaluable patients in the 1.2 mg once daily arm, the dose being evaluated in the current Phase 3 trial. Furthermore, patients receiving the 1.2 mg daily dose experienced a median best reduction in tumor volume of 88% as measured by serial MRI exams, as of the previously reported July 5, 2023 data cut-off, and also demonstrated improvement in other radiographic parameters.

Exelixis reported Q4 adjusted EPS 33c against a consensus of 21c, and reported Q4 revenue of $479.65M against a consensus of $480.17M. “Exelixis entered 2024 with significant momentum on the research, development, commercial and financial fronts,” said CEO Michael Morrissey. “This year, we plan to advance our regulatory strategies for cabozantinib label expansions in neuroendocrine tumors and metastatic castration-resistant prostate cancer, both indications with high unmet medical need and the potential to drive revenue growth for the franchise for years to come. Positive data from the CABINET and CONTACT-02 studies give us confidence that cabozantinib has the potential to become an important option for clinicians treating patients with these forms of cancer. As we pursue these additional growth opportunities, we remain steadfast in our defense of cabozantinib’s intellectual property and anticipate a ruling on the second bench trial for our ongoing litigation with MSN Pharmaceuticals this spring.”

February 5

Exicure and Bluejay Therapeutics announced they entered into a patent license agreement to develop cavrotolimod for potential treatment for hepatitis. Under the terms of the agreement, Bluejay will receive an exclusive license in the field of hepatitis to all of Exicure’s relevant patents. Bluejay paid Exicure an initial small, one-time payment after the execution of this Agreement. Exicure will also be entitled to modest royalties on future net sales on all licensed technology by Bluejay during the term of the licensed patents. Exicure will be responsible for, and make all decisions concerning, the preparation, filing, prosecution, and maintenance for each patent and patent application included within the licensed patents.

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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.