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: 

• Phio Pharmaceuticals (PHIO), 5,353% surge in interest
• Kamada (KMDA), 2,492% surge in interest
• Kala Pharmaceuticals (KALA), 1,734% surge in interest
• Syros Pharmaceuticals (SYRS), 945% surge in interest
• Hoth Therapeutics (HOTH), 820% surge in interest
• Protagonist Therapeutics (PTGX), 808% surge in interest
• Replimune (REPL), 669% surge in interest
• Akero Therapeutics (AKRO), 514% surge in interest
• Scholar Rock (SRRK), 452% surge in interest

Pipeline and key clinical candidates for these companies:

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,” the company states.

Kamada is a commercial stage global biopharmaceutical company with a portfolio of marketed products indicated for rare and serious conditions and “a leader in the specialty plasma-derived field, focused on diseases of limited treatment alternatives,” the company states. Kamada notes that it is also advancing “an innovative development pipeline targeting areas of significant unmet medical need.”

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.

Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARα agonist in frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression.

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.

Protagonist Therapeutics is a biopharmaceutical company with peptide-based new chemical entities rusfertide and JNJ-2113 in advanced stages of clinical development, both derived from the company’s proprietary technology platform. Rusfertide, a mimetic of the natural hormone hepcidin, is the company’s lead drug candidate currently in a global Phase 3 stage of development. The REVIVE study is now complete, with an open-label extension underway. The global Phase 3 VERIFY study of rusfertide in polycythemia vera is ongoing. Protagonist retains all worldwide development and commercialization rights to rusfertide.

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.

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. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path, it has noted.

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 $2.8M, 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.

 Kedrion Biopharm announces the execution of a binding memorandum of understanding with Kamada for the amendment and the 8-year extension of the KEDRAB U.S. distribution agreement between the two companies. KEDRAB is a Human Rabies Immune Globulin developed in partnership with Kamada. Kedrion started the distribution of this product in the U.S. in 2018. This new commercial agreement, which begins in January 2024, includes potential expansion of Kedrion’s distribution of KEDRAB in additional territories beyond the U.S. In addition, the two companies will collaborate to expand distribution of Kedrion products by Kamada in Israel.

December 6

Syros Pharmaceuticals announced strong and encouraging initial data from its ongoing SELECT-AML-1 Phase 2 trial evaluating tamibarotene, an oral, selective, retinoic acid receptor alpha RARalpha agonist, in combination with venetoclax and azacitidine in newly diagnosed, unfit patients with acute myeloid leukemia AML and RARA gene overexpression. “I am highly encouraged by the initial data from the randomized portion of SELECT-AML-1,” said Thomas Cluzeau, MD, PhD, Head of Hematology at Nice University Hospital, Cote d’Azur University in France. “Despite the recent advances in treatment for unfit AML patients, there remains a substantial need for options that offer higher response rates and improved overall survival, particularly for the one-third of patients who do not respond to existing standard-of-care. I believe tamibarotene may offer a significant therapeutic advance for the treatment of AML and I am eager to continue enrolling patients in the ongoing SELECT-AML-1 trial.” “These data highlight the potential of tamibarotene to be a cornerstone therapy for newly diagnosed, unfit AML patients with RARA overexpression, further demonstrating its differentiated product profile and validating our biologically targeted approach,” said David A. Roth, M.D., Chief Medical Officer of Syros.

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.

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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.