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: 

• NGM Biopharmaceuticals (NGM), 4,414% surge in interest
• Voyager Therapeutics (VYGR), 2,626% surge in interest
• Soligenix (SNGX), 2,600% surge in interest
• Oncocyte (OCX), 1,329% surge in interest
• Corcept Therapeutics (CORT), 897% surge in interest
• Agios Pharmaceuticals (AGIO), 867% surge in interest
• Anavex Life Sciences (AVXL), 551% surge in interest
• Applied Therapeutics (APLT), 322% surge in interest
• Enlivex Therapeutics (ENLV), 291% surge in interest
• Moderna (MRNA), 247% surge in interest

Pipeline and key clinical candidates for these companies:

NGM Biopharmaceuticals says its biology-centric drug discovery approach “aims to seamlessly integrate interrogation of complex disease-associated biology and protein engineering expertise to unlock proprietary insights that are leveraged to generate promising product candidates and enable their rapid advancement into proof-of-concept studies.” NGM Bio “aspires to operate one of the most productive research and development engines in the biopharmaceutical industry,” the company adds.

Voyager Therapeutics says its TRACER AAV capsid discovery platform has “generated novel capsids with high target delivery and blood-brain barrier penetration at low doses, potentially addressing the narrow therapeutic window associated with conventional gene therapy delivery vectors.” The platform is fueling alliances with Pfizer (PFE), Novartis (NVS) and Neurocrine Biosciences (NBIX) as well as multiple programs in Voyager’s own pipeline, which includes wholly-owned and collaborative preclinical programs in Alzheimer’s disease, amyotrophic lateral sclerosis, Parkinson’s disease, and Friedreich’s Ataxia, the company has stated.

Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases. Their Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma. Development programs in this business segment also include expansion of synthetic hypericin into psoriasis, their first-in-class innate defense regulator technology, dusquetide for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate for the prevention/treatment of gastrointestinal disorders characterized by severe inflammation including pediatric Crohn’s disease.

Oncocyte is a precision diagnostics company. The company’s tests are designed to help provide clarity and confidence to physicians and their patients. VitaGraft is a blood-based solid organ transplantation monitoring test. DetermaIO is a gene expression test that assesses the tumor microenvironment to predict response to immunotherapies, and the pipeline test DetermaCNI is blood-based monitoring tool for assessing therapeutic efficacy.

Corcept’s focus on cortisol modulation and its potential to treat patients across a wide variety of serious disorders has led to the discovery of more than 1,000 proprietary selective cortisol modulators. Corcept’s advanced clinical trials are being conducted in patients with hypercortisolism, solid tumors, amyotrophic lateral sclerosis and liver disease. In February 2012, the company introduced Korlym, the first medication approved by the FDA for the treatment of patients with Cushing’s syndrome.

Agios says it is “the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases.” In the U.S., Agios markets a first-in-class pyruvate kinase, PK, activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera, and a preclinical PAH stabilizer as a potential treatment for phenylketonuria.

Anavex Life Sciences is a biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, and other central nervous system diseases, pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX2-73, has successfully completed a Phase 2a and a Phase 2b/3 clinical trial for Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia, and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX2-73 for the treatment of Parkinson’s disease.

Applied Therapeutics is “developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.” The company’s lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.

Enlivex is a clinical stage macrophage reprogramming immunotherapy company developing Allocetra, a universal, off-the-shelf cell therapy designed to reprogram macrophages into their homeostatic state. Resetting non-homeostatic macrophages into their homeostatic state is critical for immune system rebalancing and resolution of life-threatening conditions.

Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA, or mRNA, to “an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio and integrated manufacturing facilities that allow for rapid clinical and commercial production at scale,” the company states. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has “allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing.” Most recently, Moderna’s capabilities have come together to allow the authorized use and approval of vaccines against the COVID pandemic. The company is pursuing the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases.

Recent news on these stocks:

January 4

Soligenix announced preliminary top-line results of its ongoing Phase 2a trial of SGX302 – synthetic hypericin – for the treatment of mild-to-moderate psoriasis. In the expanded portion of the trial, an additional five patients were enrolled. The Cohort 2 patients were treated more aggressively than the patients enrolled in Cohort 1 during an 18-week treatment period. SGX302 therapy was well tolerated by all patients with no drug related adverse events identified. In the four evaluable patients from Cohort 2, two reached a disease status of “Almost Clear”. In addition, the Psoriasis Activity and Severity Index score for patients in Cohort 2 had a mean drop of approximately 50% over the 18-week treatment. The company anticipates continuing to pursue SGX302 in psoriasis as it completes discussions with the FDA and the European Medicines Agency regarding a feasible confirmatory trial design for HyBryte in the treatment of early-stage CTCL.

January 3

Agios Pharmaceuticals announced that the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia achieved its primary endpoint of hemoglobin response. Statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration. “The results of the Phase 3 ENERGIZE study underscore the potential of mitapivat to be a meaningful treatment option for adults with non-transfusion dependent alpha- or beta-thalassemia. All subgroup analyses favored the mitapivat treatment arm compared to placebo,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We are grateful to all of the patients who participated in this trial, our collaborators, study investigators and advisors in the patient and clinical communities for their partnership in achieving this milestone. These data bring us one step closer to a treatment for all thalassemia patients, and we look forward to the ENERGIZE-T readout mid-year.”

Applied Therapeutics announced it has submitted a New Drug Application, NDA, to the FDA for govorestat, AT-007 for the treatment of Classic Galactosemia. The NDA was submitted in December 2023. In addition, the Company submitted a Marketing Authorization Application, MAA, to the European Medicines Agency, EMA, in the fourth quarter of 2023, which was subsequently validated and accepted for review in December 2023. “The submissions of both the NDA and MAA for govorestat are supported by rapid and sustained reduction in galactitol, which resulted in a meaningful benefit on clinical outcomes across pediatric patients, alongside a favorable safety profile,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “We look forward to working closely with both regulatory agencies throughout the review process and hope to bring the first treatment to patients with Galactosemia soon.”

January 2

NGM Biopharmaceuticals confirmed that it received a non-binding expression of interest dated December 28, 2023 from The Column Group and certain affiliated investment funds and persons, outlining TCG’s intent to explore and evaluate a potential acquisition of all of the outstanding shares of common stock of the company not already owned by TCG in a going-private transaction. TCG currently beneficially owns approximately 26.7% of the outstanding shares of NGM Bio’s common stock. In response to the EOI, the company’s board formed a special committee consisting of disinterested and independent directors to consider the EOI and any other strategic alternatives that may be available to the company, including remaining a standalone publicly-traded company. The special committee has retained Hogan Lovells US as its independent legal advisor, and intends to retain an independent financial advisor, to assist it in considering the EOI. The special committee intends to carefully consider the EOI with the assistance of its independent financial and legal advisors. There can be no assurance that any definitive agreement will be executed, or that the proposed transaction with TCG or any other transaction will be approved or consummated. NGM Bio and the special committee do not intend to comment on or disclose further developments regarding the special committee’s consideration of the EOI unless and until it deems further disclosure is appropriate or required. NGM Bio’s stockholders are advised to take no action at this time.

Voyager Therapeutics announced a strategic collaboration and capsid license agreement with Novartis Pharma AG, a subsidiary of Novartis AG (NVS) to advance potential gene therapies for Huntington’s disease, HD, and spinal muscular atrophy, SMA. Voyager will provide Novartis a target-exclusive license to access Voyager’s TRACER capsids and other intellectual property for the respective diseases, and Voyager and Novartis will collaborate to advance a preclinical gene therapy candidate for HD. Under the terms of the agreement, Novartis has agreed to pay Voyager $100 million of consideration up front, including a $20 million purchase of newly issued equity in Voyager. Voyager is eligible to receive up to $1.2 billion in preclinical, development, regulatory and sales milestones, as well as tiered royalties on global net sales of products incorporating Voyager’s TRACER capsids. Novartis will obtain target-exclusive access to Voyager’s TRACER capsids related to SMA for the duration of the agreement and will be responsible for all development and commercialization. Novartis will also receive worldwide rights to Voyager’s AAV gene therapy for HD, leveraging Voyager’s TRACER capsids and proprietary payloads.

Corcept Therapeutics announced the Court for the District of New Jersey issued a decision that Teva Pharmaceuticals (TEVA) does not infringe any asserted claims of Corcept’s U.S. Patents 10,195,214 and 10,842,800, both of which concern methods of safely administering Korlym with drugs that are strong CYP3A4 inhibitors. “This disappointing decision is based on legal and factual errors we are confident will be reversed on appeal,” said Joseph K. Belanoff, MD, Corcept’s CEO. “Our patents describe medical discoveries that have greatly expanded the therapeutic options available to patients suffering from Cushing’s syndrome, which is why the FDA added them to Korlym’s prescribing instructions. Doctors have followed these instructions and will continue to do so to safely treat patients. Failure to recognize the novelty and import of discoveries like these would fundamentally change the law, to the detriment of the many patients whom innovative companies seek to serve. We will pursue our appeal vigorously,” added Dr. Belanoff, “and will continue to assert our intellectual property rights whenever they are infringed.”

Anavex Life Sciences reported topline results from the randomized, double-blind, placebo-controlled, Phase 2/3 EXCELLENCE clinical trial, which evaluated the clinical efficacy, safety, and tolerability of 30 mg ANAVEX2-73 in 92 pediatric patients with Rett syndrome between the ages of 5 through 17 years. This was the very first study of ANAVEX2-73 in pediatric patients with Rett syndrome. After 12 weeks, the study showed improvement on the key co-primary endpoint Rett Syndrome Behaviour Questionnaire, which is a detailed 45-item questionnaire for assessing multiple Rett syndrome characteristics by the patients’ caregivers. The other co-primary endpoint, the Clinical Global Impression – Improvement scale, which represents a less granular assessment by the site investigators using a seven-point scoring, was not met. In an ad-hoc analysis, using the predefined mixed-effect model for repeated measure method, after 12 weeks of treatment, ANAVEX2-73-treated patients improved LS Mean -12.93 points on their RSBQ total score compared to LS Mean -8.32 points in placebo-treated patients. The LS Mean difference of -4.65 points between treated and placebo groups was statistically significant. When looking at other placebo-controlled Rett syndrome trials, ANAVEX2-73 compares favorably in terms of absolute RSBQ improvements, with the caveat that cross trials comparisons have their limitations. The key secondary endpoint, the Anxiety, Depression, and Mood Scale, trended favorably. In the same analysis, scores for all RSBQ and ADAMS subscales improved over the course of the study. Collectively, the RSBQ and ADAMS demonstrated improvements in multiple areas, impacting positively in particular repetitive movements, nighttime disruptive behaviors and social avoidance. In the EXCELLENCE study, a large placebo effect was observed which may have masked the compound’s therapeutic effect. Anavex believes to have identified the probable causes. A preliminary review of the safety results indicates there were no new safety signals in the EXCELLENCE study, reinforcing the favorable and manageable safety profile observed with ANAVEX2-73 to date. The most common treatment-related adverse events in the drug-treated group were somnolence and lethargy and were predominantly mild to moderate in severity. There were no clinically meaningful changes observed in SAEs associated with known risks of ANAVEX2-73. Over 91% of patients completing the trial continued into a 48-week open-label extension study, which is ongoing.

Oppenheimer analyst Hartaj Singh upgraded Moderna to Outperform from Perform with a $142 price target. The firm sees “increasing visibility” on the company’s Covid-19 vaccine sales and says catalysts in 2024 and 2025 give it reason to believe Moderna could be a five-product commercial company by 2026. The company’s Covid-19 vaccine sales “could hit a low-point in 2024,” but its vaccine sales will increase in 2025 and beyond, the analyst tells investors in a research note. Oppenheimer says Moderna’s execution in recent years has addressed the majority of its concerns.

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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.