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: 

• Alterity Therapeutics (ATHE), 8,388% surge in interest
• EyePoint Pharmaceuticals (EYPT), 1,245% surge in interest
• Kala Pharmaceuticals (KALA), 1,186% surge in interest
• Hoth Therapeutics (HOTH), 1,000% surge in interest
• Viking Therapeutics (VKTX), 893% surge in interest
• Replimune (REPL), 823% surge in interest
• Phio Pharmaceuticals (PHIO), 581% surge in interest
• Ocular Therapeutix (OCUL), 464% surge in interest
• Akero Therapeutics (AKRO), 438% surge in interest
• Scholar Rock (SRRK), 394% surge in interest

Pipeline and key clinical candidates for these companies:

Alterity Therapeutics is a clinical stage biotechnology company that says it is “dedicated to creating an alternate future for people living with neurodegenerative diseases.” The company’s lead asset, ATH434, has the potential to treat various Parkinsonian disorders and is currently being evaluated in two Phase 2 clinical trials in Multiple System Atrophy. Alterity also has a “broad drug discovery platform generating patentable chemical compounds to treat the underlying pathology of neurological diseases,” the company states.

EyePoint Pharmaceuticals is committed to developing and commercializing therapeutics to help improve the lives of patients with serious eye disorders. The company’s pipeline leverages its proprietary Durasert technology for sustained intraocular drug delivery including EYP-1901, an investigational sustained delivery intravitreal treatment currently in Phase 2 clinical trials. The proven Durasert drug delivery platform has been “safely administered to thousands of patients’ eyes” across four U.S. FDA approved products, including Yutiq for the treatment of posterior segment uveitis, EyePoint said.

Kala is dedicated to the research, development and commercialization of therapies for rare diseases of the eye. Kala’s lead product candidate, KPI-012, is in clinical development for the treatment of persistent corneal epithelial defect, or PCED, a rare disease of impaired corneal healing, which has received orphan drug designation from the FDA. Kala is also targeting the potential development of KPI-012 for the treatment of Partial Limbal Stem Cell Deficiency and ocular manifestations of moderate-to-severe Sjogren’s and plans to initiate preclinical studies to evaluate the utility of its MSC-S platform for retinal degenerative diseases, such as Retinitis Pigmentosa and Stargardt Disease.

Hoth Therapeutics refers to itself as “a catalyst in early-stage pharmaceutical research and development, elevating drugs from the bench to pre-clinical and clinical testing.” Hoth “collaborates and partners with a team of scientists, clinicians, and key opinion leaders to seek out and investigate therapeutics that hold immense potential to create breakthroughs and diversify treatment options,” the company stated.

Viking Therapeutics is focused on the development of novel first-in-class or best-in-class therapies for the treatment of metabolic and endocrine disorders. The company’s clinical programs include VK2809, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist for the treatment of lipid and metabolic disorders, which is currently being evaluated in a Phase 2b study for the treatment of biopsy-confirmed non-alcoholic steatohepatitis, or NASH, and fibrosis.

Replimune Group is focused on the development of novel tumor-directed oncolytic immunotherapies. Replimune’s proprietary RPx platform is based on a HSV-1 backbone with payloads added to maximize immunogenic cell death and the induction of a systemic anti-tumor immune response. The RPx platform’s dual local and systemic mechanism of action is “expected to be synergistic with most established and experimental cancer treatment modalities, and, with an attractive safety profile the RPx platform has the versatility to be developed alone or combined with a variety of other treatment options,” the company stated.

Phio Pharmaceuticals is a clinical stage biotechnology company whose proprietary INTASYL RNAi technology is designed to make immune cells more effective in killing tumor cells. INTASYL is the only self-delivering RNAi technology focused on immuno-oncology therapeutics. INTASYL drugs are designed to precisely target specific proteins that reduce the body’s ability to fight cancer, without the need for specialized formulations or drug delivery systems.

Ocular Therapeutix is focused on the formulation, development, and commercialization of therapies for diseases and conditions of the eye using its proprietary bioresorbable hydrogel-based formulation technology. Ocular Therapeutix’s first commercial drug product, Dextenza, is an FDA-approved corticosteroid for the treatment of ocular inflammation and pain following ophthalmic surgery and ocular itching associated with allergic conjunctivitis. Ocular Therapeutix’s earlier stage development assets include: OTX-TKI, currently in Phase 1 clinical trials for the treatment of wet AMD and diabetic retinopathy; OTX-TIC, currently in a Phase 2 clinical trial for the treatment of primary open-angle glaucoma or ocular hypertension; and OTX-CSI for the chronic treatment of dry eye disease and OTX-DED for the short-term treatment of the signs and symptoms of dry eye disease, both of which have completed Phase 2 clinical trials.

Akero Therapeutics is developing “transformational treatments for patients with serious metabolic diseases marked by high unmet medical need,” including NASH, a disease without any approved therapies. Akero’s lead product candidate, EFX, is a differentiated Fc-FGF21 fusion protein that has been engineered to mimic the balanced biological activity profile of native FGF21, an endogenous hormone that alleviates cellular stress and regulates metabolism throughout the body. EFX is currently being evaluated in two Phase 2b clinical trials: the HARMONY study in patients with pre-cirrhotic NASH and the SYMMETRY study in patients with cirrhotic NASH. EFX is also being evaluated in an expansion cohort of the SYMMETRY study, comparing the safety and tolerability of EFX to placebo when added to an existing GLP-1 receptor agonist in patients with pre-cirrhotic NASH and Type 2 diabetes.

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.

Recent news on these stocks:

December 7

Phio Pharmaceuticals announced the agreement by several accredited investors to exercise certain outstanding warrants to purchase up to an aggregate of 2,130,252 shares of common stock of the Company originally issued in October 2018 through June 2023, having exercise prices between $5.40 and $4.03 per share, at a reduced exercise price of $1.33 per share. The shares of common stock issuable upon exercise of the warrants are registered pursuant to effective registration statements on Form S-1 and Form S-3. The gross proceeds to the Company from the exercise of the warrants are expected to be approximately $2.8 million, prior to deducting placement agent fees and estimated offering expenses. H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering. The offering is expected to close on or about December 8, 2023, subject to satisfaction of customary closing conditions. The Company intends to use the net proceeds from the offering for working capital and other general corporate purposes.

December 5

Hoth Therapeutics has completed the manufacturing of its drug substance HT-KIT using good laboratory practice. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting and already has Orphan Drug Designation from the FDA. Hoth also recently completed its Pre-IND meeting with FDA in November. Hoth has now successfully completed manufacturing of the HT-KIT drug substance in collaboration with WuXi STA Pharmaceutical. Hoth plans to use the GLP drug substance in its upcoming pre-clinical studies required for its investigational new drug – IND – submission.

Replimune Group announced results from the primary analysis of the CERPASS trial evaluating RP1 in combination with cemiplimab for the treatment of locally advanced or metastatic cutaneous squamous cell carcinoma and provided initial data for all patients in the anti-PD1 failed melanoma cohort of the IGNYTE clinical trial. Results from the CERPASS Trial in CSCC: The study did not meet either of the two primary endpoints of complete response rate or overall response rate as assessed by blinded independent central review. RP1 in combination with cemiplimab increased the CRR versus cemiplimab alone, which was just short of the required threshold for statistical significance in this study. Notably, among the 83 patients with locally advanced disease, the complete response rate in the RP1 plus cemiplimab group was 48.1% versus 22.6% in the cemiplimab only group. The ORR was comparable between the two study groups. Importantly, RP1 in combination with cemiplimab also increased duration of response as compared to cemiplimab alone, however, these data are immature and further follow up is required. Of note, RP1 plus cemiplimab provided particularly meaningful clinical activity for many patients with difficult to treat, disfiguring tumors that typically have the greatest impact on quality of life, given their size and location. There was also an imbalance in baseline tumor burden across the treatment groups which may have impacted the number of responses seen. A significantly greater number of patients with high baseline tumor burden were treated in the RP1 plus cemiplimab group as compared to the cemiplimab alone group. The trial will continue as planned to assess DOR, progression free survival and overall survival with greater maturity. Initial Data from All Patients in the IGNYTE Cohort of RP1 in Anti-PD1 Failed Melanoma: The registration directed anti-PD1 failed melanoma cohort from the IGNYTE clinical trial includes 140 patients and completed enrollment earlier this year. In the RP1 plus nivolumab group, the ORR was 31.4% with a CR rate of 12% showing activity consistent with the prior snapshot of 91 anti-PD1 failed melanoma patients. In the full population, almost half of patients failed combination therapy with ipilimumab plus nivolumab as compared to the earlier snapshot where approximately a third were ipilimumab and nivolumab failures. Approximately 50% of patients experienced clinical benefit, defined as CR, PR, or stable disease. Of responders, 100% are ongoing at more than six months with 78% of responses still ongoing as of November 6, 2023. Responses reported for this snapshot were investigator-assessed. RP1 combined with nivolumab continues to be well-tolerated, with mainly Grade 1-2 “on target” side effects, observed.

December 4

Alterity Therapeutics announced that new data on the effect of ATH434 in a Parkinson’s disease primate model was presented at the Future of Parkinson’s Disease Conference 2023 that took place November 30 – December 3, 2023 in Austin, TX, USA. The poster, entitled, “Effects of ATH434, a Clinical-Phase Small Molecule with Moderate Affinity for Iron, in Hemiparkinsonian Macaques”, was presented by Margaret Bradbury, PhD, Vice President of Research and Nonclinical Development at Alterity and collaborators from Vanderbilt University Medical Center and the Florey Institute of Neuroscience in Melbourne. The presentation demonstrated that ATH434 treatment improved motor performance and general function in monkeys with experimentally induced Parkinson’s disease. The favorable impact on Parkinson’s symptoms was associated with lower iron levels in the area of pathology. In addition, ATH434 treatment increased levels of synaptophysin, a protein marker that reflects functional connections between neurons. The study compared daily oral doses of ATH434 versus a vehicle for 12-14 weeks after parkinsonian symptoms were evident. Monkeys were assessed with the Parkinson Behavior Rating Scale before, during and after dosing. At Week 12, all evaluable ATH434-treated monkeys had stable or improving PBRS scores from Baseline to Week 12 whereas two of three vehicle-treated monkeys did not demonstrate improvement or worsened, as expected from the progressive nature of the Parkinson model. The components of the PBRS scale indicate that ATH434 reduced motor impairment and improved general functions such as posture, balance, activity, and gait. Favorable parkinsonian outcomes observed in each of the ATH434-treated monkeys were associated with lower iron in the right substantia nigra. In addition, monkeys with improved scores had higher right dorsal striatal synaptophysin, indicating functional recovery of nerve endings in this critical motor pathway.

EyePoint Pharmaceuticals announced positive topline results of its Phase 2 DAVIO 2 trial of EYP-1901, an investigational sustained delivery maintenance treatment for wet age-related macular degeneration combining vorolanib, a selective tyrosine kinase inhibitor with bioerodible Durasert E. The clinical trial met its primary endpoint with both EYP-1901 doses demonstrating statistical non-inferiority change in best corrected visual acuity compared to aflibercept control and a favorable safety profile with no EYP-1901-related ocular or systemic serious adverse events. The trial also achieved key secondary endpoints with both EYP-1901 doses, including an over 80% reduction in treatment burden, nearly two-thirds of eyes supplement-free up to six months and over 80% receiving only zero or one supplement up to six-months. Additionally, there was strong anatomical control with both EYP-1901 cohorts as measured by optical coherence tomography. DAVIO 2 topline interim results include: Both EYP-1901 doses achieved all primary and secondary endpoints. Statistical non-inferiority in change in BCVA compared to aflibercept control, at weeks 28 and weeks 32 combined. The 2mg and 3mg doses were only -0.3 and -0.4 letters different, respectively, versus on-label aflibercept. The lower limit of the non-inferiority margin is defined as a -4.5 letters by the FDA with 5 letters representing one line on the eye chart. Continued positive safety and tolerability profile with no EYP-1901-related ocular or systemic SAEs. 89% and 85% reduction in treatment burden, respectively, for the 2mg and 3mg EYP-1901 doses. 65% and 64% of eyes were supplement free up to six-months, respectively, for the 2mg and 3mg doses of EYP-1901. Both EYP-1901 doses demonstrated strong anatomic control with OCT difference below 10 microns at week 32 compared to the aflibercept control. Patient discontinuation up to week 32 was low at 4%.

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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.