Stay ahead of biotech stocks seeing a surge in interest from retail and financial professional investors with this exclusive recap from The Fly These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
Pipeline and key clinical candidates for these companies:
NuCana is a clinical-stage biopharmaceutical company that says it is "focused on significantly improving treatment outcomes for patients with cancer by applying its ProTide technology to transform some of the most widely prescribed chemotherapy agents, nucleoside analogs, into more effective and safer medicines."
BioMarin is a biotechnology company "dedicated to transforming lives through genetic discovery." The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders, the company says.
Exicure is an early-stage biotechnology company historically focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. The company says it continues to actively pursue out-licensing opportunities for its clinical asset, cavrotolimod, as well as for its preclinical candidates, including the SCN9A program for neuropathic pain, and to pursue all strategic alternatives with the goal of maximizing stockholder value.
Ascendis Pharma is applying its platform technology to "build a leading, fully integrated, global biopharmaceutical company focused on making a meaningful difference in patients' lives," the company states. The company uses its TransCon technologies to "create new and potentially best-in-class therapies," Ascendis says.
Aptevo Therapeutics is focused on developing novel immunotherapies for the treatment of cancer. APVO436 is a bispecific CD3xCD123 ADAPTIR currently in Phase 1b development in a multi-center, multi-cohort trial designed to evaluate safety, tolerability and efficacy in combination therapy and monotherapy for patients with acute myeloid leukemia, or AML. The company plans to initiate a Phase 2 trial, evaluating APVO436 in combination with venetoclax and azacitidine in patients with AML who are venetoclax treatment naive, in 2H23.
Coherus is a commercial-stage biopharmaceutical company focused on the research, development and commercialization of immunotherapies to treat cancer. Coherus' immuno-oncology pipeline includes multiple antibody immunotherapy candidates focused on enhancing the innate and adaptive immune responses to enable a robust immunologic response and enhance outcomes for patients with cancer.
Eton is a pharmaceutical company focused on developing and commercializing treatments for rare diseases. The company currently has five commercial rare disease products: ALKINDI SPRINKLE, PKU GOLIKE, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO hydrocortisone autoinjector.
Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company that says it is "pioneering the development of ITK inhibition as a new approach to immunotherapy for a broad range of cancer and immune diseases." The company's lead product candidate is CPI-818, an investigational, oral, small molecule drug that selectively inhibits ITK and is in a mid-stage clinical trial for patients with T cell lymphoma. Its other clinical-stage candidates are being developed for a variety of cancer indications.
Recent news on these stocks:
September 18
BioMarin Pharmaceutical announced that positive data from the CANOPY clinical program evaluating VOXZOGO, or vosoritide, in children with achondroplasia and other genetic skeletal conditions will be presented at the 16th International Skeletal Dysplasia Society meeting, or ISDS, in Madrid, Sept. 18-21. These results include data showing that children with achondroplasia treated with VOXZOGO experienced meaningful improvements beyond height, such as in health-related quality of life, or HRQoL, and increased bone length while maintaining bone strength. Researchers will also present encouraging data from ongoing investigator-led studies investigating treatment in children with other genetic skeletal conditions, including hypochondroplasia and Noonan syndrome, as well as those with genetic variants often associated with idiopathic short stature such as aggrecan (ACAN) deficiency and heterozygous NPR2 mutations. Qualitative insights elucidating caregiver perspectives of children with achondroplasia revealed that VOXZOGO had positive impacts on HRQoL across several physical, emotional and social functioning domains. Notable physical functioning improvements reported included improved motor skills and self-care, which are particularly meaningful outcomes for children and families impacted by the condition. Psychosocial functioning outcomes included improved confidence and social interactions. Additional results from an investigator-led analysis of BioMarin's Phase 2 111-205 study, previously shared at the 2024 International Conference on Children's Bone Health, demonstrated that children who received VOXZOGO had significant increases in bone length and metacarpal cortical area after approximately five years of therapy, suggesting that treatment allowed the bone to remain strong as it lengthened.
September 16
Ascendis Pharma announced topline data from the pivotal double-blind placebo-controlled ApproaCH Trial of TransCon CNP, which included 84 children with achondroplasia randomized 2:1. TransCon CNP is an investigational prodrug of C-type natriuretic peptide administered once weekly and designed to provide sustained release and continuous exposure of active CNP. In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity superior to placebo. TransCon CNP also demonstrated statistically significant improvements in other growth parameters, including height Z-score and change from baseline AGV. Primary Endpoint For the primary endpoint of AGV at Week 52, children treated with TransCon CNP demonstrated an LS mean AGV of 5.89 cm/year compared to 4.41 cm/year in the placebo arm, an LS mean difference of 1.49 cm/year. Children aged 2 to less than5 years, treated with TransCon CNP demonstrated a change from baseline AGV at Week 52 of 1.57 cm/year compared to 0.43 cm/year in the placebo arm. For the secondary endpoint of change in ACH Height Z-score, children treated with TransCon CNP demonstrated an LS mean change from baseline ACH Height Z-score of 0.30 compared to 0.01 in the placebo arm
Aptevo Therapeutics and Alligator Bioscience announced interim data from the dose escalation phase of their Phase 1 trial evaluating ALG.APV-527 for the treatment of solid tumors likely to express the tumor antigen 5T4. The results, which include clinical activity, safety, tolerability outcomes, pharmacokinetics and pharmacodynamics were presented in a poster session on Saturday, September 14, 2024, at the European Society for Medical Oncology Annual Congress in Barcelona, Spain. ALG.APV-527, is a first-in-class bispecific antibody that targets 4-1BB and the tumor antigen 5T4. The compound is being evaluated in a multi-center, dose escalation trial that has 18 patients included in the safety analysis. These patients received multiple, prior rounds of therapy for the treatment of solid tumor types. The trial is approaching full enrollment and interim results include: Clinical Activity/Efficacy: Nine of 15 efficacy evaluable patients have a best overall response to date of stable disease; The longest SD duration was in a breast cancer patient who entered the study with progressive disease, achieved stable disease and remained on study for greater than11 months. This patient successfully transitioned to a higher dose level twice; One colon cancer patient with sustained SD remains on study for more than four months. Safety and Tolerability; ALG.APV-527 demonstrated positive safety and tolerability across all cohorts; A maximum tolerated dose has not been identified; Evidence of favorable pharmacokinetics and biological activity of ALG.APV-527; ALG.APV-527 could be measured in all patients with serum concentration of ALG.APV-527 consistent with the administered dose and preclinical predictions.; Biomarker analyses confirm biological activity of ALG.APV-527 "4-1BB has been a target of interest - though with excess toxicity - for decades, and novel bispecific approaches like this will enable us to maximize anti-tumor immunity while limiting the systemic toxicity concerns that have plagued this key immune costimulatory receptor. With this backdrop, these Phase 1 interim results are very encouraging, with ALG.APV-527 showing a positive safety profile with 60% of evaluable patients achieving stable disease, meaning not progressing for variable time frames including one breast cancer patient being treated with monotherapy on study with SD for more than 11 months. We are excited to see signs of clinical activity, underscoring the potential of the drug to benefit patients with solid tumors in the clinical setting, supported by a positive safety and tolerability profile," stated Thomas Marron, MD, PhD, Professor in Immunology & Immunotherapy and in Medicine, Hematology and Medical Oncology at Icahn School of Medicine at Mount Sinai, participating Investigator of the trial.
September 15
NuCana presented final data from the Phase 2 NuTide:701 study at the ESMO Congress on NUC-7738 in combination with pembrolizumab for patients with metastatic melanoma who were refractory to or had relapsed on prior PD-1 inhibitor therapy. In this cohort of 12 patients, most of whom had received at least two prior lines of PD-1 inhibitor therapy, nine achieved disease control, including two patients who achieved Partial Responses. One of these patients, who had received two prior lines of PD-1 inhibitor-based therapy and had progressed on their latest treatment of ipilimumab plus nivolumab within two months, achieved a 55% reduction in tumor volume. Seven of the 12 patients had a progression free survival time of greater than five months, which is highly atypical in this patient population. In addition to achieving these encouraging efficacy signals, the combination of NUC-7738 and pembrolizumab had a favorable safety profile. NUC-7738's ability to sensitize PD-1 resistant tumors to rechallenge with PD-1 inhibitors is believed to be due to its ability to target multiple aspects of the tumor microenvironment via the disruption of RNA polyadenylation and subsequent changes to gene expression in cancer cells. In support of this hypothesis, data presented from tumor biopsies obtained before and after NUC-7738 based treatment demonstrated increases in genes related to antigen presentation and T-cell activation.
Ladenburg raised the firm's price target on Corvus Pharmaceuticals to $21 from $12 and kept a Buy rating on the shares. The firm expanded its model for soquelitinib to include atopic dermatitis, in addition to the current focus on peripheral T-cell lymphoma and solid tumors, as Corvus anticipates Phase 1 atopic dermatitis randomized data by year-end.
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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.
