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: 

• Bio-Path Holdings (BPTH), 3,348% surge in interest
• PTC Therapeutics (PTCT), 679% surge in interest
• Clearside Biomedical (CLSD), 536% surge in interest
• Kezar Life Sciences (KZR), , 333% surge in interest
• Oragenics (OGEN), 312% surge in interest
• Cassava Sciences (SAVA), , 180% surge in interest
• CorMedix (CRMD), , 143% surge in interest
• Catalyst Pharma (CPRX), , 140% surge in interest
• Trevena (TRVN), 103% surge in interest

Pipeline and key clinical candidates for these companies:

Bio-Path is developing DNAbilize, a technology that has yielded a pipeline of RNAi nanoparticle drugs that can be administered with an intravenous transfusion. Bio-Path’s lead product candidate, prexigebersen , or BP1001, is in a Phase 2 study for blood cancers and BP1001-A, a drug product modification of prexigebersen, has been cleared by the FDA and Phase 1 studies in solid tumors will commence in 2022. The company’s second product, BP1002, is being evaluated for the treatment of blood cancers and solid tumors, including lymphoma and acute myeloid leukemia. In addition, an IND is expected to be filed for BP1003, a novel liposome-incorporated STAT3 antisense oligodeoxynucleotide developed by Bio-Path as a specific inhibitor of STAT3, in 2022 or Q1 of 2023.

PTC Therapeutics is a biopharmaceutical company that says it is “focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders.” The company adds: “PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have little to no treatment options.”

Clearside Biomedical is a biopharmaceutical company that says it is “revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space to improve patient outcomes.” Clearside’s SCS injection platform, utilizing the company’s patented SCS Microinjector, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. 

Kezar Life Sciences is a clinical-stage biopharmaceutical company developing novel small molecule therapeutics to treat unmet needs in immune-mediated diseases. Zetomipzomib, a selective immunoproteasome inhibitor, is currently being evaluated in a Phase 2b clinical trial for lupus nephritis and a Phase 2a clinical trial for autoimmune hepatitis.

Oragenics is a development-stage company dedicated to fighting infectious diseases including coronaviruses and multidrug-resistant organisms. Its lead product is Terra CoV-2, an intranasal vaccine candidate to prevent COVID-19 and variants of the SARS-CoV-2 virus.

Cassava Sciences is a clinical-stage biotechnology company that says its mission is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease. “Its novel science is based on stabilizing-but not removing-a critical protein in the brain. The company’s product candidates have not been approved by any regulatory authority, and their safety, efficacy or other desirable attributes have not been established,” Cassava has stated.

CorMedix is a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening conditions and diseases. The company is focused on commercializing its lead product DefenCath, which was approved by the FDA on November 15, 2023. CorMedix commercially launched DefenCath in inpatient settings in April 2024 and in outpatient settings in July 2024. CorMedix also intends to develop DefenCath as a catheter lock solution for use in other patient populations.

Catalyst says it is “committed to developing and commercializing innovative first-in-class medicines that address rare neurological and epileptic diseases”. Catalyst’s flagship U.S. commercial product is Firdapse tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome for adults and for children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights to Fycompa CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older.

Trevena is a biopharmaceutical company focused on the development and commercialization of medicines for patients with CNS disorders. The company has one approved product in the United States, OLINVYK injection, indicated in adults for the management of acute pain severe enough to require an intravenous opioid analgesic and for whom alternative treatments are inadequate. The company’s pipeline includes three investigational drug candidates: TRV045 for diabetic neuropathic pain and epilepsy, TRV250 for the acute treatment of migraine and TRV734 for maintenance treatment of opioid use disorder.

Recent news on these stocks:

October 10

Kezar Life Sciences announced that it has received an unsolicited, non-binding proposal from Concentra Biosciences, a Delaware limited liability company, to acquire all of the outstanding shares of common stock of Kezar for cash consideration of $1.10 per share, plus a contingent value right that represents the right to receive 80% of the net proceeds from any out-license or disposition of Kezar’s development programs or intellectual property. According to a Schedule 13D filed on October 8, with the SEC disclosing Concentra’s proposal, Tang Capital Management is the controlling stockholder of Concentra, and Tang Capital beneficially owns approximately 9.9% of the company’s outstanding common stock. Consistent with its fiduciary duties, Kezar’s board and management team will evaluate the non-binding proposal to determine the course of action that it believes is in the best interest of the company and all Kezar stockholders and respond appropriately.

October 9

Clearside Biomedical announced that the ODYSSEY Phase 2b clinical trial of CLS-AX, or axitinib injectable suspension, for the treatment of neovascular age-related macular degeneration, or wet AMD, achieved both its primary and secondary outcomes. In participants who received CLS-AX delivered suprachoroidally, best corrected visual acuity, or BCVA, and ocular anatomy were stable up to 6 months compared to participants who received aflibercept. In addition, CLS-AX demonstrated a well-tolerated safety profile to Week 36 inclusive of mandatory re-dosing of CLS-AX at Week 24. “We are very excited to report positive topline data from our successful ODYSSEY Phase 2b trial,” said George Lasezkay, President and CEO. “These encouraging results strongly support advancing our CLS-AX wet AMD program into Phase 3 development and provide further evidence of the potential benefits of delivering medicines to the back of the eye using our proprietary SCS Microinjector. We achieved our primary outcome of maintaining stable BCVA throughout the trial as measured by the mean change in BCVA from baseline to Week 36. CLS-AX consistently reduced the frequency of injections after the initial dose of CLS-AX with approximately 90% of CLS-AX participants not requiring any additional treatment up to 4 months, 81% not requiring any additional treatment up to 5 months, and 67% not requiring any additional treatment up to 6 months before mandatory re-dosing at Week 24. We believe this data supports our goal to potentially provide a safe, convenient wet AMD treatment option with the advantage of a flexible maintenance dosing regimen between 3 to 6 months. We look forward to continuing to analyze the results and share additional data analysis with the retina community at upcoming medical meetings.” ODYSSEY was a randomized, double-masked, parallel-group, active-controlled, multicenter, 36-week, Phase 2b clinical trial in participants with wet AMD previously treated with intravitreal anti-vascular endothelial growth factor standard of care therapy. A total of 60 participants were treated for 36 weeks and randomized to either CLS-AX or aflibercept with a 2:1 randomization schedule. CLS-AX was administered by suprachoroidal injection via Clearside’s SCS Microinjector, and aflibercept was administered via intravitreal injection. Participants in the trial were determined to have active disease with a median duration of wet AMD diagnosis of 9.9 months.

Oragenics provided a corporate update reflecting on the company’s progress throughout 2024, including key milestones in the development of ONP-002, its lead candidate for the treatment of concussions. The company’s lead program, ONP-002, is a first-in-class neurosteroid being developed to treat moderate to severe concussions. Intranasal delivery provides numerous advantages over traditional systemic methods. Oragenics has made significant advancements in the development of ONP-002 during 2024, including: Strengthened Clinical Leadership; Phase II Clinical Trial Preparation; Successful Cardiotoxicity Test; FDA-Required Genotoxicity Studies; Partnership with Avance Clinical; Temperature Stability Achieved; Spray-Dry Manufacturing and Device Completion; Improved Drug Percentage in Final Formulationl Completion of FDA-Recognized Study for Concussion Drug, ONP-002: Intranasal casting studies are critical for FDA approval of pharmaceuticals delivered via the nasal passage. Looking Ahead: Key Milestones: Oragenics anticipates several key milestones in the coming months, including: Initiation of Phase II Clinical Trials: The company plans to begin Phase II trials later this year, initially in Australia, followed by U.S. trials. These trials will evaluate safety and efficacy – evaluating the effects of ONP-002 on concussion patient symptom reduction and functional recovery. In preparation for its upcoming Phase II clinical trials, Oragenics plans to submit the Australian Regulatory Submission Brochure during the fourth quarter of 2024. This submission is a critical component for the regulatory approval of the trial in Australia and outlines the clinical trial safety and efficacy protocols, informed consent protocols, and data collection methods.

Cassava Sciences published a letter from its CEO Rick Barry, stating: “A lot has happened in the few months since I last wrote to you. It’s time that I update you on what has taken place here at Cassava and the substantial progress that we have made. I am happy to inform you that our ReTHINK Phase 3 clinical trial recently completed its last patient, last visit. All patients have completed dosing, and all that remains is to collect and analyze the data. For a company our size, this is a remarkable achievement. This study enrolled 804 patients at 77 sites in the US, Puerto Rico, Canada, and Australia. Patients were randomized 1:1 between placebo and simufilam 100 mg, twice daily. Approximately 70% of the patients entered the study with mild Alzheimer’s disease, while 30% entered with moderate Alzheimer’s. We expect to announce top-line results of the ReTHINK trial before year end. We submitted our proposed Statistical Analysis Plan to FDA in July and are now responding to comments that we have received from the agency. We expect to finalize the Statistical Analysis Plan in the near term.”

CorMedix announced that it has entered into a new commercial supply contract with a top-five mid-sized dialysis operator for the supply of DefenCath to dialysis clinics in the US. This new agreement will provide access to DefenCath for adult patients with kidney failure receiving hemodialysis through a central venous catheter at more than 200 outpatient dialysis clinics located across the United States. “This new agreement, in combination with our existing customer agreements, gives CorMedix the opportunity to make DefenCath available to patients receiving hemodialysis though a CVC at roughly 60% of the outpatient dialysis centers in the US,” says CorMedix CEO, Joseph Todisco. “We are working closely with all of our new customer relationships on the operational steps needed to ensure DefenCath availability in their clinics in the coming months.”

October 8

Bio-Path Holdings announced that it has entered into definitive agreements for the issuance and sale of an aggregate of 4.6M shares of its common stock, series A warrants to purchase up to 6.41M shares of common stock and short-term series B warrants to purchase up to 6.41M shares of common stock at a purchase price of $0.87 per share of common stock and accompanying warrants in a private placement priced at-the-market under Nasdaq rules. The closing of the offering is expected to occur on or about October 10, 2024, subject to the satisfaction of customary closing conditions. H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

PTC Therapeutics provided today several positive updates on the vatiquinone Friedreich ataxia – FA – program. The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression. In addition, PTC recently aligned with FDA on key aspects of the planned NDA submission for vatiquinone. Analysis of the MOVE-FA long-term extension study demonstrated that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale relative to a matched natural history cohort. This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over 3 years. These results confirm that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial are maintained over 144 weeks of treatment. In addition, vatiquinone continued to be safe and well tolerated without any treatment-related serious adverse events reported. Following 24-months of treatment with vatiquinone, subjects had a 4.8-point benefit on the mFARS relative to a matched natural history population. PTC plans to submit the vatiquinone NDA in December.

October 4

Trevena announced that the company received notice that the Nasdaq Hearings Panel had determined to delist the company’s common stock from The Nasdaq Stock Market due to the company’s failure to comply with the minimum stockholder’s equity requirement under Nasdaq Listing Rule 5550(b)(1). As previously disclosed, the Panel had provided the company until October 2, to regain compliance with the Equity Standard Rule.

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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.