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: 

• Scholar Rock Holding (SRRK), 19,120% surge in interest
• Bio-Path Holdings (BPTH), 3,027% surge in interest
• Trevena (TRVN), 1,081% surge in interest
• Oragenics (OGEN), 728% surge in interest
• PTC Therapeutics (PTCT), 426% surge in interest
• OncoCyte (OCX), 400% surge in interest
• iBio (IBIO), 362% surge in interest
• X4 Pharmaceuticals (XFOR), 206% surge in interest
• Clearside Biomedical (CLSD), 163% surge in interest

Pipeline and key clinical candidates for these companies:

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with “the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, and fibrosis,” the company states. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path, it has noted.

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.

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.

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.

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.”

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.

iBio develops next-generation biopharmaceuticals using computational biology and 3D-modeling of subdominant and conformational epitopes, prospectively enabling the discovery of new antibody treatments for hard-to-target cancers and other diseases. iBio’s mission is to “decrease drug failures, shorten drug development timelines, and open up new frontiers against the most promising targets,” the company stated.

X4 Pharmaceuticals is focused on the discovery and development of novel therapies for people with diseases of the immune system. The company’s lead clinical candidate is mavorixafor, a small molecule antagonist of chemokine receptor CXCR4 that is being developed as an oral, once-daily therapy.

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. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The company’s lead program, CLS-AX, for the treatment of neovascular age-related macular degeneration, recently completed a Phase 2b clinical trial, and planning for a Phase 3 program is underway. Clearside developed and gained approval for its first product, XIPERE for suprachoroidal use, which is available in the U.S. through a commercial partner.

Recent news on these stocks:

October 10

iBio provided an update on the myostatin program for cardiometabolic disease and obesity in collaboration with AstralBio. iBio’s technology stack enabled the Company to rapidly advance the joint myostatin program from inception to in vitro proof-of-concept in human muscle cells. Following early discovery, the companies have identified a molecule with therapeutic potential for treating muscle wasting and obesity, which is designed for subcutaneous administration and has potential for an extended half-life. The companies are currently working on plans to advance this molecule into non-cGMP in vivo studies in rodents and non-human primates with potential early readouts of the NHP in early 2025.

October 9

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.

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.

October 8

A Scholar Rock 10.265M share Secondary priced at $28.25. The deal size was increased to $300M from $275M and the deal priced below the last closing price of $29.49. JPMorgan, Jefferies and Piper Sandler acted as joint book running managers for the offering.

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.

Oncocyte announced the peer-reviewed publication of positive data related to its proprietary gene expression test, DetermaIO. The NeoTRIP Phase 2 clinical trial randomized patients with triple-negative breast cancer, TNBC, to receive neoadjuvant carboplatin and nab-paclitaxel, with or without the immunotherapy, atezolizumab. As a secondary point of interest in the study, Oncocyte’s DetermaIO test was among several established biomarkers and gene signatures assessed for its ability to predict which patients with early stage TNBC are most likely to benefit from the immunotherapy. The study was performed in collaboration with the Michelangelo Foundation for Cancer Research, a well-regarded independent scientific organization based in Milan. Only DetermaIO was both statistically significant and predictive of a pathologic complete response among the various biomarkers assessed.

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.

Hear more from InvestingChannel by signing up for The Spill.

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.