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), 5,757% surge in interest
• Affimed (AFMD), 1,847% surge in interest
• Fibrogen (FGEN), 1,060% surge in interest
• Equillium (EQ), 923% surge in interest
• Precigen (PGEN), 665% surge in interest
• CNS Pharmaceuticals (CNSP), 418% surge in interest
• Galera Therapeutics (GRTX), 261% surge in interest
• Amarin (AMRN), 198% surge in interest
• Liquidia (LQDA), 191% surge in interest
• Cyclacel Pharmaceuticals (CYCC), 178% 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.

Affimed is a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer by actualizing the untapped potential of the innate immune system. The company’s innate cell engagers, ICE, enable a tumor-targeted approach to recognize and kill a range of hematologic and solid tumors. ICE are generated on the company’s proprietary ROCK platform which predictably generates customized molecules that leverage the power of innate immune cells to destroy tumor cells. A number of ICE molecules are in clinical development, being studied as mono- or combination therapy.

FibroGen is “committed to leveraging its expertise in connective tissue growth factor biology and hypoxia-inducible factor to discovering, developing, and commercializing a pipeline of first-in-class therapeutics for the treatment of unmet needs.” Pamrevlumab, an anti-CTGF human monoclonal antibody, is in clinical development for the treatment of idiopathic pulmonary fibrosis, or IPF, locally advanced unresectable pancreatic cancer, metastatic pancreatic cancer, and Duchenne muscular dystrophy, or DMD. Roxadustat is currently approved in China, Europe, Japan, and numerous other countries for the treatment of anemia in CKD patients on dialysis and not on dialysis. Roxadustat is in Phase 3 clinical development in the U.S. and Europe for anemia associated with myelodysplastic syndromes, or MDS, and in Phase 3 clinical development in China for treatment of chemotherapy-induced anemia, or CIA.

Equillium is a clinical-stage biotechnology company working to develop novel therapeutics to treat severe autoimmune and inflammatory disorders with high unmet medical need. The company’s pipeline consists of the following novel first-in-class immunomodulatory assets and product platform targeting immuno-inflammatory pathways. EQ101: a selective tri-specific cytokine inhibitor targeting IL-2, IL-9, and IL-15; currently under evaluation in a Phase 2 proof-of-concept clinical study of patients with alopecia areata being conducted in Australia and New Zealand by Equillium’s Australian subsidiary as the trial sponsor. EQ302: an orally delivered, selective bi-specific cytokine inhibitor targeting IL-15 and IL-21; currently in pre-clinical development.

Precigen’s AdenoVerse immunotherapy platform utilizes a library of proprietary adenovectors for the efficient gene delivery of therapeutic effectors, immunomodulators, and vaccine antigens designed to modulate the immune system. Precigen’s gorilla adenovectors, part of the AdenoVerse library, “have potentially superior performance characteristics as compared to current competition. AdenoVerse immunotherapies have been shown to generate high-level and durable antigen-specific T-cell immune responses as well as an ability to boost these responses via repeat administration,” the company says.

CNS Pharmaceuticals is a clinical-stage pharmaceutical company developing a pipeline of anti-cancer drug candidates for the treatment of primary and metastatic cancers of the brain and central nervous system. The company’s lead drug candidate, Berubicin, is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is currently in development for the treatment of a number of serious brain and CNS oncology indications including glioblastoma multiforme, or GBM, an aggressive and incurable form of brain cancer.

Galera Therapeutics is focused on developing and commercializing a pipeline of therapeutic candidates that have the potential to transform radiotherapy in cancer. Galera’s selective dismutase mimetic product candidate avasopasem manganese, avasopasem, or GC4419, is being evaluated for radiotherapy-induced toxicities. The company’s second product candidate, rucosopasem manganese, rucosopasem, or GC4711, is in clinical-stage development to augment the anti-cancer efficacy of stereotactic body radiation therapy in patients with non-small cell lung cancer and locally advanced pancreatic cancer.

Amarin is an “innovative pharmaceutical company leading a new paradigm in cardiovascular disease management” and is “committed to increasing the scientific understanding of the cardiovascular risk that persists beyond traditional therapies and advancing the treatment of that risk for patients worldwide.”

Liquidia Corporation is a biopharmaceutical company focused on the development and commercialization of products in pulmonary hypertension and other applications of its PRINT Technology. Liquidia Technologies has developed Yutrepia inhalation powder for the treatment of pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease. Liquidia Technologies is also developing L606, an investigational liposomal formulation of treprostinil administered twice-daily with a short-duration next-generation nebulizer, for use in North America.

Cyclacel is a clinical-stage, biopharmaceutical company developing innovative cancer medicines based on cell cycle, transcriptional regulation, epigenetics and mitosis biology. The transcriptional regulation program is evaluating fadraciclib, a CDK2/9 inhibitor, and the epigenetic/anti-mitotic program plogosertib, a PLK1 inhibitor, in patients with both solid tumors and hematological malignancies. Cyclacel’s strategy is to build a diversified biopharmaceutical business based on a pipeline of novel drug candidates addressing oncology and hematology indications.

Recent news on these stocks:

June 4

Equillium announced topline data from its Phase 2, single dose, proof-of-concept, or PoC, study of EQ101 in adult patients with moderate, severe or very-severe alopecia areata, or AA, an autoimmune disease driven by an immune cell attack of the hair follicles that causes hair loss. The primary objectives of the study were to evaluate the safety and tolerability profile of EQ101, as well as signs of efficacy using Severity of Alopecia Tool, or SALT, scores, where a score of 100 represents total scalp hair loss and a score of 0 represents no scalp hair loss. Results from the study demonstrated a favorable safety and tolerability profile with no serious adverse events, or SAEs, and improvements in SALT scores above the published historically low placebo response rates. Of all subjects that completed 24 weeks of treatment, 20% achieved a SALT score of less than or equal to 20 by week 24. Of those subjects with moderate to severe AA at baseline that completed 24 weeks of treatment, 29% achieved a SALT score of less than or equal to 20 by week 24, and a mean SALT improvement from baseline of 18%. Throughout 24 weeks of treatment and 4 weeks of follow up, EQ101 was well tolerated with no SAEs and no notable changes in safety laboratory, electrocardiogram, vital signs, or physical exam findings were reported. The majority of adverse events were Grade 1 or 2, with the most common being upper respiratory tract infection, headache and fatigue. The two Grade 3 events in two subjects considered related to study treatment were a case of transient lymphocytopenia and fatigue.

Amarin Corporation announced that the company’s board appointed Aaron Berg, currently Amarin’s executive VP and president of the U.S. Business, as president and CEO. The appointment of Berg follows the resignation of Patrick Holt as president & CEO of the company. Berg joined Amarin in November 2012 and has more than 30 years of biopharmaceutical industry experience.

In a regulatory filing, Amarin disclosed that the company has been informed by “a large national pharmacy benefit manager,” or “PBM,” that, effective July 1, the PBM intends to no longer cover Vascepa as the exclusive icosapent ethyl product for its Commercial national formularies and will be transitioning Vascepa to not covered as of July 1, 2024. “Currently, Vascepa volume through these formularies represents approximately 25% of aggregate U.S. Vascepa prescription volume. This decision does not impact Vascepa coverage within Medicare Part D formularies of the PBM,” the company stated.

June 3

Bio-Path Holdings announced it presented interim results from the company’s Phase 2 study of prexigebersen in combination with decitabine and venetoclax for the treatment of acute myeloid leukemia in an oral presentation at the American Society of Clinical Oncology Annual Meeting, on June 1, 2024 in Chicago, IL. Maro Ohanian, D.O., Department of Leukemia, University of Texas MD Anderson Cancer Center, presented data showing prexigebersen continues to be well-tolerated and has demonstrated compelling efficacy results in two reporting cohorts including newly diagnosed AML patients and refractory/relapsed AML patients, both of which exceeded outcomes with frontline therapy. In Cohort 1, 31 newly diagnosed patients were enrolled; 20 evaluable patients with a median age of 75 years, treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk or secondary AML evolved from myelodysplastic syndromes, chronic myelomonocytic leukemia or treatment-related AML. Fifteen patients achieved complete remission, CRh, or CRi; two patients achieved partial remission and two patients achieved stable disease. In Cohort 2, 38 relapsed/refractory patients were enrolled; 23 evaluable patients with a median age of 63 years, treated with at least one cycle of prexigebersen, decitabine and venetoclax, had adverse-risk or sAM. Twelve patients achieved CR/CRi/CRh; one patient achieved PR, eight patients achieved SD and one patient had treatment failure. Among the evaluable patients of both cohorts, adverse events were consistent with those expected with decitabine and venetoclax and/or AML, including fatigue, anemia and neutropenia, while the most frequent severe adverse events were febrile neutropenia and sepsis. Given these promising interim results, Bio-Path expects to continue enrollment of up to 98 and 54 evaluable patients for Cohorts 1 and 2, respectively.

FibroGen announced that the FDA has cleared the company’s Investigational New Drug of FG-3165, a galectin-9 targeted monoclonal antibody under development for treatment of solid tumors characterized by high Gal9 levels of expression. “The FDA’s IND clearance is an important achievement for us, and we are pleased to advance another product from our promising oncology pipeline into the clinic,” said Thane Wettig, CEO of FibroGen. “To date, FG-3165 has demonstrated anti-tumor activity with improved survival in combination with other immune modulatory therapies in mouse cancer models and has shown excellent tolerability in nonclinical safety studies. We are excited to begin enrollment in a Phase 1 trial in the second half of this year and explore the potential of FG-3165 in enhancing anti-tumor immune responses in the tumor microenvironment.” The FDA IND clearance enables FibroGen to initiate a Phase 1 clinical trial evaluating the safety and efficacy of FG-3165 in patients with select solid tumors. The trial is anticipated to begin enrollment in the second half of 2024

FibroGen announced a clinical trial supply agreement with Regeneron Pharmaceuticals (REGN) to evaluate FibroGen’s immuno-oncology assets, FG-3165 and FG-3175, in combination with Regeneron’s anti-PD-1 therapy, LIBTAYO or cemiplimab, in patients with solid tumors. “We are very excited to collaborate with Regeneron Pharmaceuticals to evaluate two separate novel combination approaches to treat patients with select solid tumors,” said Deyaa Adib, M.D., Chief Medical Officer of FibroGen. “We believe that the mechanisms of action for both FG-3165 and FG-3175 have the potential to be synergistic with cemiplimab, providing the possibility for an improvement in clinical outcomes for patients. We look forward to building a collaborative relationship with Regeneron, who is a leader in oncology therapeutic products development and commercialization.” FG-3165 targets Gal9, which binds multiple immune checkpoints on lymphocytes that suppress T and natural killer NK cell activation. FG-3175 targets CCR8, a receptor frequently over-expressed on T regulatory cells in solid tumors. Both FG-3165 and FG-3175 have demonstrated complementary mechanisms of action with PD-1 inhibitors preclinically, and the Company believes that combining LIBTAYO with either FG-3165 or FG-3175 may result in added clinical benefit for patients.

Precigen released Phase 1/2 pivotal study results for the investigational PRGN-2012 off-the-shelf AdenoVerse gene therapy in patients with recurrent respiratory papillomatosis. Results were presented in a late-breaking oral presentation at the 2024 American Society of Clinical Oncology Annual Meeting by Scott M. Norberg, DO, Associate Research Physician, Center for Immuno-Oncology, Center for Cancer Research, National Cancer Institute and a lead investigator for the PRGN-2012 clinical study. The Phase 1/2 clinical study evaluated safety and efficacy of PRGN-2012. The study design included an initial 3+3 dose escalation cohort to identify the recommended Phase 2 dose. Adult RRP patients who had three or more surgeries in the prior 12 months were eligible for the study. The Phase 1/2 study enrolled a total of 38 patients. Of these, 3 patients received four administrations of PRGN-2012 at 1x 1011 particle units/dose and 35 patients received four administrations of PRGN-2012 at RP2D over a 12 week treatment period via subcutaneous injection. Primary endpoints included safety and Complete Response rate defined as the percentage of patients who require no RRP surgeries in the 12-month period after PRGN-2012 treatment completion. Key secondary endpoints included HPV-specific immune responses, extent of papilloma growth as measured by Derkay scoring, and quality of life measurement as measured by Vocal Handicap Index-10. Baseline patient characteristics of the 35 adult patients included a median age of 49 years; 20 of the patients were male and 15 were female. Patients had a median of 4 surgeries in the 12 months before PRGN-2012 treatment initiation. Average years since RRP diagnosis was 20 with 12 and 23 patients with juvenile and adult onset RRP, respectively. Primary efficacy endpoint analysis demonstrated that 51% patients achieved Complete Response, defined as no need for RRP surgeries in the 12-month period following completion of PRGN-2012 treatment. PRGN-2012 treatment showed significant improvement in anatomical Derkay scores, a tool used for research purposes to quantify RRP severity based on involvement of laryngeal structures, with mean Derkay scores reducing from 9 at baseline to 1 at 24 weeks post-treatment in patients with Complete Response.

CNS Pharmaceuticals announced a 1-for-50 reverse split of its common stock. Beginning on June 5, the company’s common stock will continue to trade on The Nasdaq Capital Market on a split adjusted basis under the trading symbol “CNSP”, but will trade under the following new CUSIP number: 18978H300. The reverse stock split is primarily intended to increase the company’s per share trading price and bring the company into compliance with the Nasdaq’s listing requirement regarding minimum share price.

Liquidia announced that on May 31, Judge Andrews of the U.S. District Court for the District of Delaware denied the motion for preliminary injunction filed by United Therapeutics (UTHR) that sought to block the launch of Liquidia’s Yutrepia inhalation powder to treat pulmonary hypertension associated with interstitial lung disease. The ruling reinforces the clear path for the FDA to issue a final decision on the amended New Drug Application for Yutrepia. The motion for preliminary injunction was filed in the lawsuit filed by UTHR in September 2023 in which it has alleged YUTREPIA would infringe U.S. Patent No. 11,826,327. While this ruling maintains the status quo in which there is no legal impediment to the FDA granting final approval to Yutrepia, this lawsuit will continue forward to trial, which is currently scheduled for June 2025. Friday’s ruling follows earlier legal rulings from multiple bodies finding that Yutrepia does not infringe any valid claim of the patents previously asserted by UTHR, including the decision by the Patent Trial and Appeal Board, affirmed by the U.S. Court of Appeals for the Federal Circuit, that all claims of U.S. Patent No. 10,716,793 to be unpatentable. UTHR has stated that it plans to appeal the Federal Circuit’s affirmation of the invalidity of the ‘793 Patent to the United States Supreme Court. Separately, UTHR has also appealed Judge Andrews’ ruling to set aside an injunction that he had previously issued blocking the launch of Yutrepia based solely on the ‘793 Patent.

Cyclacel Pharmaceuticals announced that new clinical, pharmacokinetic and pharmacodynamic data from the CYC065-101 study of fadraciclib as oral monotherapy was presented at a poster at the American Society of Clinical Oncology Annual Meeting from May 31-June 4, 2024 in Chicago, IL. “We are excited to report data with fadraciclib monotherapy from the entire Phase 1 population at ASCO. Clinical benefit was observed in heavily pretreated patients with several tumor types, including endometrial, lung, ovarian, pancreatic cancer, and T-cell lymphoma,” said Spiro Rombotis, President and CEO. “Retrospective analysis suggests that this activity may be associated in part with alterations in certain tumor suppressor genes forming a hypothesis which we are testing in the ongoing Phase 2 part of the study. We look forward to reporting initial proof of concept data in the second half of 2024.” New clinical, PK and PD data were presented at ASCO from the fully enrolled, Phase 1, dose escalation part of the CYC065-101 study of fadraciclib as monotherapy. The patients were heavily pretreated, having received a median of four prior lines of therapy. Fadraciclib was generally well tolerated with good compliance between dose levels 1 and 5. The most common treatment related adverse events reported were nausea, vomiting, diarrhea, fatigue, and hyperglycemia. A total of 25 drug-related SAEs were reported in 8 patients, with most common being hyperglycemia, platelet count decrease, and accidental overdose. There were no drug-related SAEs at dose level 5 which was selected for the Phase 2 proof of concept part of the 065-101 study. PKs were dose-proportional and exceeded the preclinical efficacy targets for both CDK2 and CDK9. PDs evaluated in peripheral blood showed suppression of CDKN2A/B by four hours post treatment in most patients who received 100 mg bid or higher. A total of 34 patients had measurable target lesions at baseline. Two partial responses were reported in patients with T-cell lymphoma, one of whom had CDKN2A loss. A squamous non-small cell lung cancer patient with CDKN2A and CDKN2B loss achieved 22% reduction in tumor burden at 4 weeks per RECIST 1.1 criteria. In addition, clinical benefit was reported in two patients with endometrial cancer, and one each with ovarian and pancreatic cancers. The proof of concept part of the study is now enrolling patients with CDKN2A/B loss or T-cell lymphoma.

June 2

Affimed announced longer follow-up data from the EGFRwt cohort and initial clinical efficacy data from the EGFRmut cohort from the on-going AFM24-102 study in NSCLC. As of the updated data cutoff on May 13, 2024 for the 17 EGFRwt patients previously reported on, 15 patients were response-evaluable. Four confirmed objective responses were seen: 1 complete response and 3 partial responses. In addition, 8 patients achieved stable disease, resulting in a disease control rate of 71%. Median progression-free survival was 5.9 months with median follow-up of 7.4 months. Importantly 3 of 4 responses were ongoing for more than 7 months. All responders were resistant to checkpoint inhibitor treatment prior to the study, which supports the hypothesis that combining AFM24 with atezolizumab may provide an alternative strategy to overcome resistance to existing therapies. As of May 21, 2024, 21 heavily pretreated EGFRmut patients had received the combination therapy of which 13 were response-evaluable. The combination of AFM24 with atezolizumab showed encouraging signals of clinical activity including 1 CR, 3 PRs and 6 patients with SD. As of the data cut-off, all responses were on-going. EGFRmut NSCLC is considered an immunogenically weak subtype where single-agent therapy with immune checkpoint inhibitors have exhibited poor response rates. The data suggests that the combination of AFM24 and atezolizumab could be acting synergistically to improve efficacy outcomes. AFM24 and atezolizumab combination therapy demonstrated a manageable safety profile. Side effects were consistent with the known safety profiles of these agents. The most frequent side effects observed were mild to moderate infusion related reactions and transient mild to moderate increase in liver enzymes. The EGFRwt NSCLC cohort of the study will enroll up to 40 patients and the EGFRmut NSCLC cohort will enroll up to 25 patients. Recruitment in both cohorts is ongoing, and further updates are expected in H2 2024.

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.