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: 

• CymaBay Therapeutics (CBAY), 796% surge in interest
• Tonix Pharmaceuticals (TNXP), 73% surge in interest
• Sangamo Therapeutics (SGMO), 72% surge in interest
• Geron Corp (GERN), 40% surge in interest
• Lexicon Pharmaceuticals (LXRX), 25% surge in interest
• Ocugen (OCGN), 20% surge in interest

Pipeline and key clinical candidates for these companies:

CymaBay Therapeutics is a clinical-stage biopharmaceutical company focused on improving the lives of people with liver and other chronic diseases that have high unmet medical need. According to the company, its deep understanding of the underlying mechanisms of liver inflammation and fibrosis, and the unique targets that play a role in their progression, have helped it receive breakthrough therapy designation, Priority Medicines status and orphan drug status for seladelpar, a first-in-class investigational treatment for people with PBC.

Tonix is focused on central nervous system, rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the second quarter of 2023. TNX-1300 is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA.

Sangamo Therapeutics is a genomic medicine company that says it is “dedicated to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases who do not have adequate or any treatment options.” Sangamo’s zinc finger epigenetic regulators are being studied to potentially address neurological disorders and Sangamo’s capsid discovery platform is “making progress toward potentially expanding delivery beyond currently available intrathecal delivery capsids, including in the central nervous system,” Sangamo says.

Geron is a late-stage biopharmaceutical company pursuing therapies with the potential to extend and enrich the lives of patients living with hematologic malignancies. Its investigational first-in-class telomerase inhibitor, imetelstat, “harnesses Nobel Prize winning science in a treatment that may alter the underlying course of these diseases,” the company says.

Lexicon says it is “pioneering the discovery and development of innovative medicines to safely and effectively treat disease.” Lexicon has advanced one of these medicines to market and has a “pipeline of promising drug candidates in discovery and clinical and preclinical development in heart failure, neuropathic pain, diabetes and metabolism and other indications,” the company states.

Ocugen is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. The company’s “breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product,” says Ocugen, which adds that it is advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs.

Recent news on these stocks:

February 14

Tonix Pharmaceuticals Holding held a key opinion leader webinar to discuss the positive Phase 3 data of Tonmya for the management of fibromyalgia, and the path to file for FDA approval in the second half of 2024. The webinar was held on January 31, 2024 and hosted by Alliance Global Partners. The webinar featured two thought leaders in the field of fibromyalgia: Daniel Clauw, M.D., Professor of Anesthesiology, Medicine and Psychiatry, Director Chronic Pain & Fatigue Research Center, University of Michigan, and Lesley Arnold, M.D., Professor of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine. Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. As previously announced, Tonix’s second positive Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function. Tonix plans to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2024 for Tonmya for the management of fibromyalgia. “We believe that these positive results show that fibromyalgia can be successfully treated by Tonmya and may provide the opportunity for Tonix to have the first FDA-approved drug for fibromyalgia in more than a decade,” said Seth Lederman, M.D., President and CEO of Tonix Pharmaceuticals. “We are now an important step closer to bringing a new, first-line treatment to fibromyalgia patients that offers broad symptom relief and favorable tolerability for chronic use and adherence.”

February 12

Gilead (GILD) and CymaBay announced a definitive agreement under which Gilead will acquire CymaBay for $32.50 per share in cash or a total equity value of $4.3B. The company said the addition of CymaBay’s investigational lead product candidate, seladelpar for the treatment of primary biliary cholangitis including pruritus, complements Gilead’s existing liver portfolio and aligns with its long-standing commitment to bringing transformational medicines to patients. The transaction was approved by both the Gilead and CymaBay Boards of Directors and is anticipated to close during the first quarter of 2024, subject to regulatory approvals and other customary closing conditions. Under the terms of the merger agreement entered into in connection with the transaction, a wholly-owned subsidiary of Gilead will promptly commence a tender offer to acquire all of the outstanding shares of CymaBay’s common stock at a price of $32.50 per share in cash, which offer price represents a 27 percent premium to CymaBay’s closing share price on February 9, 2024. Following successful completion of the tender offer, Gilead will acquire all remaining shares not tendered in the offer through a second step merger at the same price as in the tender offer. Upon FDA approval of seladelpar, the proposed transaction is expected to enhance Gilead’s revenue growth, and it is also expected that the transaction will be approximately neutral to earnings per share in 2025 and significantly accretive thereafter. Consummation of the tender offer is subject to a minimum tender of at least a majority of then-outstanding CymaBay shares, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions.

CymaBay announced that the FDA accepted its New Drug Application for seladelpar, an investigational treatment for the management of primary biliary cholangitis – PBC – including pruritus in adults without cirrhosis or with compensated cirrhosis who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act target action date of August 14 and notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application. PBC is a rare, chronic condition that can lead to inflammation and eventually liver cirrhosis. Seladelpar is an investigational, potent, selective, orally active PPARdelta agonist, or delpar, in development for PBC treatment. It is the only investigational agent to have demonstrated a statistically significant improvement in biochemical markers of disease progression and pre-specified measures of PBC-related pruritus in a Phase 3 study.

Sangamo announced U.S. and European regulatory updates for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease. The FDA has agreed in a Type D meeting that data from a single, adequate, and well-controlled study may form the primary basis of approval of a BLA for isaralgagene civaparvovec. The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm. A head-to-head comparison with Enzyme Replacement Therapy, or ERT, is not part of the proposed study design deemed acceptable by the FDA. This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated. Additionally, the EMA has granted PRIME eligibility to isaralgagene civaparvovec. PRIME is a program designed to enhance support for the development of medicines that target an unmet medical need and is intended to optimize development plans and expedite review and approval processes so that these medicines may reach patients as early as possible. Isaralgagene civaparvovec has already received Orphan Medicinal Product designation from the EMA as well as Orphan Drug, Fast Track and RMAT designations from the FDA. Updated Phase 1/2 STAAR study data showing sustained clinical benefit and a differentiated safety profile across 24 patients were shared at the 20th Annual WORLDSymposium in San Diego, California on Wednesday, February 7. A total of 29 patients have been treated to date in the Phase 1/2 STAAR study. All 13 patients withdrawn from ERT remain off ERT as of February 12, 2024. Screening and enrollment are complete in the study and dosing of the remaining enrolled patients is expected in the first half of 2024. Sangamo is deferring additional investments in planning for a registrational trial until a collaboration partnership is secured.

Noble Capital analyst Robert LeBoyer raised the firm’s price target on Ocugen to $8 from $5 and kept an Outperform rating on the shares to reflect the company’s recent progress in its clinical programs. Since OCU410 has recently begun clinical trials in dry age-related macular degeneration, or dAMD, the firm has adjusted its view of the probability of market entry, the analyst told investors.

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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.