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), 1,012% surge in interest
• Bicycle Therapeutics (BCYC), 544% surge in interest
• Merrimack Pharmaceuticals (MACK), 355% surge in interest
• Acasti Pharma (ACST), 328% surge in interest
• Adial Pharmaceuticals (ADIL), 323% surge in interest
• G1 Therapeutics (GTHX), 243% surge in interest
• Chimerix (CMRX), 197% surge in interest
• Oramed Pharma (ORMP), 192% surge in interest
• Tonix Pharmaceuticals (TNXP), 191% surge in interest
• Sangamo Therapeutics (SGMO), 168% 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 through a pipeline of innovative therapies. 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.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company developing a novel class of medicines, referred to as Bicycle molecules, for diseases that are underserved by existing therapeutics. Bicycle molecules are fully synthetic short peptides constrained with small molecule scaffolds to form two loops that stabilize their structural geometry. The company is evaluating BT8009, a Bicycle Toxin Conjugate targeting Nectin-4, a well-validated tumor antigen; BT5528, a BTC targeting EphA2; and BT7480, a Bicycle TICA® targeting Nectin-4 and agonizing CD137, in company-sponsored Phase 1/2 trials.

Acasti is a late-stage specialty pharma company with drug delivery technologies and drug candidates addressing rare and orphan diseases. Acasti’s three lead clinical assets have each been granted Orphan Drug Designation by the FDA, which provide the assets with seven years of marketing exclusivity post-launch in the United States, and additional intellectual property protection with over 40 granted and pending patents.

Adial Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development of treatments for addictions. The company’s lead investigational new drug product, AD04, is a genetically targeted, serotonin-3 receptor antagonist, therapeutic agent for the treatment of Alcohol Use Disorder, or AUD, in heavy drinking patients and was recently investigated in the company’s ONWARD pivotal Phase 3 clinical trial for the potential treatment of AUD in subjects with certain target genotypes identified using the company’s proprietary companion diagnostic genetic test. The company is also developing adenosine analogs for the treatment of pain and other disorders.

G1 Therapeutics is a commercial-stage biopharmaceutical company whose first commercial product is Cosela, or trilaciclib. G1 is executing a tumor-agnostic development plan evaluating trilaciclib in a variety of solid tumors, including colorectal, breast, lung, and bladder cancers.

Chimerix is a biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The company’s most advanced clinical-stage development program, ONC201, is in development for H3 K27M-mutant glioma.

Oramed Pharmaceuticals calls itself “a platform technology pioneer in the field of oral delivery solutions for drugs currently delivered via injection.” The company’s novel Protein Oral Delivery technology is designed to protect drug integrity and increase absorption. Oramed has offices in the United States and Israel.

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.

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 13

Merrimack reported that Ipsen (IPSEY) issued a press release announcing the FDA has approved the supplemental new drug application for Onivyde plus 5 fluorouracil/leucovorin and oxaliplatin as a first-line treatment for people living with metastatic pancreatic ductal adenocarcinoma. Under the terms of the 2017 Asset Purchase Agreement between Ipsen and Merrimack, previously approved by Merrimack’s stockholders, this FDA approval triggers a $225M milestone payment from Ipsen to Merrimack which is due on or before March 29, 2024. “Today’s announcement is the culmination of nearly seven years of clinical trials and regulatory approval efforts by Ipsen following its 2017 purchase of the Onivyde technology from Merrimack. The company has received several payments from the Ipsen Agreement including: a $575M payment in 2017, a $5.7M working capital adjustment payment later that year, and a $5M milestone payment in 2019” said Gary Crocker, chairman of Merrimack’s board. “The Board of Merrimack plans to hold a Special Meeting of Stockholders to approve a plan for a corporate dissolution and followed by a distribution of the proceeds to our stockholders from this new $225M milestone payment, plus residual cash, net of corporate taxes and interest charges accruing under IRS installment sale rules, and other expenses.”

Adial Pharmaceuticals announced it has been awarded an important patent which expands the estate covering the combination of the company’s proprietary genetic diagnostic to identify patients with specific genotypes for genetically targeted treatment of alcohol use disorder and drug dependencies, such as opioid use disorder, with the company’s lead investigational new drug product AD04.

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.

Acasti Pharma reported Q3 EPS of (21c) vs. (52c) last year. The company’s cash, cash equivalents and short-term investments as of December 31, 2023, were $25.M. The company believes its cash, cash equivalents and short-term investments are sufficient to fund its operations into the second calendar quarter of 2026. “During the third quarter we continued to execute our focused strategy around our biggest value driver program GTX-104 and its pivotal Phase 3 STRIVE-ON safety trial. Having dosed the first patient in October, we’ve continued to enroll more patients and sites since that time,” said Prashant Kohli, CEO of Acasti. “With our balance sheet enhanced by the $7.5 million private placement secured last quarter and prudent use of resources announced in our strategic realignment plan in May 2023, our cash runway is now expected to extend into the second calendar quarter of 2026, well beyond our potential submission of GTX-104 NDA in the first half of 2025.”

G1 Therapeutics announced that the independent Data Monitoring Committee, or DMC, recommended continuation of the pivotal Phase 3 PRESERVE 2 trial evaluating trilaciclib in combination with gemcitabine and carboplatin for the first line treatment of metastatic triple negative breast cancer, or mTNBC, to the final analysis. This final analysis evaluating Overall Survival, or OS, is estimated to occur in the third quarter of 2024 and will be conducted on the intent-to-treat, or ITT, population. “The DMC did not express any concerns regarding safety or recommend any other changes to the study. G1 remains blinded to all data as the early stopping criteria were not met during the interim analysis,” the company stated. “We remain confident in the ability of trilaciclib to ultimately achieve the OS primary endpoint based on the robust survival benefit demonstrated in the prior randomized Phase 2 study, which continued to meaningfully increase over time as patients received subsequent therapies, as well as the increased statistical power for the final analysis of this pivotal study. While a positive interim analysis would have enabled us to bring this therapy to patients in need sooner, we look forward to completing the study and potentially making this meaningful new treatment option available to patients with this highly aggressive form of breast cancer as early as next year,” added Jack Bailey, CEO at G1 Therapeutics.

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.

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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.