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), 4,782% surge in interest
• Affimed (AFMD), 1,357% surge in interest
• Precigen (PGEN), 392% surge in interest
• Fibrogen (FGEN), 291% surge in interest
• CNS Pharmaceuticals (CNSP), 208% surge in interest
• Cyclacel Pharmaceuticals (CYCC), 92% surge in interest
• Intellia Therapeutics (NTLA), 71% surge in interest
• Macrogenics (MGNX), 61% 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.

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.

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.

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.

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.

Intellia Therapeutics, a clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. “Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine,” the company says.

MacroGenics is a biopharmaceutical company focused on developing, manufacturing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. “The combination of MacroGenics’ technology platforms and protein engineering expertise has allowed the company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies,” MacroGenics states.

Recent news on these stocks:

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.

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.

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.

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.

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.

Intellia Therapeutics announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema, a rare genetic condition that leads to potentially life-threatening swelling attacks. The data were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology Congress 2024, being held May 31 – June 3 in Valencia, Spain. Across all patients, a 98% mean reduction in monthly attack rate and a 99% mean reduction in moderate to severe attacks were observed after a single dose of NTLA-2002 through the latest follow-up. The median duration of follow-up was 20.1 months. At each dose level tested, a robust level of HAE attack rate reduction was achieved and long lasting. The longest attack-free interval for an individual patient post-infusion is over 26 months and ongoing. Additionally, the reduction in HAE attacks has been persistent in patients with the most severe HAE symptoms. The two patients with the highest historic monthly HAE attack rates at the start of the study were attack-free by the end of the 16-week primary observation period and have remained free of attacks through the latest follow-up. The longest attack-free duration amongst these two patients is 23.5 months and ongoing. Further, 100% of patients who discontinued prophylaxis treatment after NTLA-2002 remain free of chronic prophylaxis treatment. Eight of 10 patients had no attacks following the 16-week primary observation period. These patients have experienced ongoing attack-free durations of greater than 18 months. Of the two patients who had any attacks, one had a mild attack that did not require treatment and a single patient experienced a moderate attack. Amongst these two patients, their mean reduction in monthly HAE attack rate was 97% after a single dose of NTLA-2002 through the latest follow-up. As previously reported, administration of NTLA-2002 led to dose-dependent, robust and durable reductions in plasma kallikrein. Mean reduction in plasma kallikrein levels from baseline through latest assessment was 60%, 88%, and 95%. At all three dose levels, NTLA-2002 was well-tolerated, and the majority of adverse events were mild in severity. Consistent with previously reported results, the most frequent adverse events were infusion-related reactions and fatigue, which were mostly Grade 1 and resolved within two days. There have been no dose-limiting toxicities, no serious adverse events and no adverse events of Grade 3 or higher observed to date. No clinically significant laboratory abnormalities were observed in any patient.

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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.