These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include:
- Lipella Pharmaceuticals (LIPO), 30,214% surge in interest
- Regenxbio (RGNX), 2,068% surge in interest
- CNS Pharmaceuticals (CNSP), 1,265% surge in interest
- vTv Therapeutics (VTVT), 507% surge in interest
- Vaccinex (VCNX), 483% surge in interest
- CorMedix (CRMD), 329% surge in interest
- Corvus Pharmaceuticals (CRVS), 278% surge in interest
- VBI Vaccines (VBIV), 230% surge in interest
- Repligen (RGEN), 224% surge in interest
- Corcept Therapeutics (CORT), 165% surge in interest
Pipeline and key clinical candidates for these companies:
Lipella is focused on developing new drugs by reformulating the active agents in existing generic drugs and optimizing these reformulations for new applications. The company says it maintains a therapeutic focus on diseases with significant, unaddressed morbidity and mortality where no approved drug therapy currently exists, “believing that this focus can potentially help reduce the cost, time and risk associated with obtaining marketing approval.”
Regenxbio is a clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a “5x’25” strategy to progress five AAV Therapeutics from its internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
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.
vTv Therapeutics is focused on developing oral, small molecule drug candidates. vTv has a pipeline of clinical drug candidates led by programs for the treatment of type 1 diabetes. vTv’s development partners are pursuing additional indications in type 2 diabetes, chronic obstructive pulmonary disease, renal disease, primary mitochondrial myopathy, and pancreatic cancer.
Vaccinex says it is “pioneering a differentiated approach to treating slowly progressive neurodegenerative diseases and cancer through the inhibition of semaphorin 4D.” The company’s lead drug candidate, pepinemab, is designed to block SEMA4D, a potent biological effector that is believed to trigger damaging inflammation in chronic diseases of the brain and inhibit immune infiltration and activation in tumors. In neurodegenerative diseases, pepinemab is being studied as a monotherapy in the Phase 1/2a SIGNAL-AD study in Alzheimer’s Disease, with ongoing exploration of potential Phase 3 development in Huntington’s disease. In oncology, pepinemab is being evaluated in combination with KEYTRUDA in the Phase 1b/2 KEYNOTE-B84 study in recurrent or metastatic head and neck cancer and in combination with BAVENCIO in a Phase 1b/2 study in patients with metastatic pancreatic adenocarcinoma.
CorMedix is a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening conditions and diseases. The company is focused on developing its lead product DefenCath, a novel, non-antibiotic antimicrobial solution designed to prevent costly and life-threatening bloodstream infections associated with the use of central venous catheters in patients undergoing chronic hemodialysis.
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.
VBI Vaccines is a biopharmaceutical company focused on virus-like particles, or VLPs, including a proprietary enveloped VLP, eVLP, platform technology. VBI develops vaccine candidates that mimic the natural presentation of viruses, designed to elicit the innate power of the human immune system. “VBI is committed to targeting and overcoming significant infectious diseases, including hepatitis B, coronaviruses, and cytomegalovirus, as well as aggressive cancers including glioblastoma,” the company says.
Repligen Corporation develops and commercializes bioprocessing technologies and systems that enable efficiencies in the process of manufacturing biological drugs. The customers served are primarily biopharmaceutical drug developers and contract development and manufacturing organizations, or CDMOs.
Corcept’s focus on cortisol modulation and its potential to treat patients across a wide variety of serious disorders has led to the discovery of more than 1,000 proprietary selective cortisol modulators. Corcept’s advanced clinical trials are being conducted in patients with hypercortisolism, solid tumors, amyotrophic lateral sclerosis and non-alcoholic steatohepatitis. In February 2012, the company introduced Korlym, the first medication approved by the FDA for the treatment of patients with Cushing’s syndrome.
Recent news on these stocks:
August 1
Lipella Pharmaceuticals announced that it has entered into a definitive agreement for the purchase and sale of an aggregate of 2.066M shares of common stock at a purchase price of 62c per share in a registered direct offering priced at-the-market under Nasdaq rules. H.C. Wainwright is acting as the exclusive placement agent for the offering. The gross proceeds to Lipella from the offering are expected to be approximately $1.28M, before deducting the placement agent’s fees and other offering expenses payable by the company. Lipella currently intends to use the net proceeds from the offering for working capital and general corporate purposes.
Corvus Pharmaceuticals announced that the FDA granted Fast Track Designation to soquelitinib for the treatment of adult patients with relapsed or refractory peripheral T cell lymphoma, PTCL, after at least two lines of systemic therapy. “The granting of Fast Track Designation by the FDA highlights the significant unmet need for patients with relapsed or refractory PTCL,” said Richard A. Miller, M.D., co-founder, president and CEO of Corvus. “The current treatment options for these patients provide limited efficacy and are associated with significant toxicity, and there are no FDA fully approved agents. There continues to be strong interest in soquelitinib from investigators at sites with deep experience treating T cell lymphomas and we are on track to initiate patient enrollment in our registrational Phase 3 trial in PTCL in the third quarter 2024.”
July 31
Vaccinex announced positive results of its early stage SIGNAL-AD clinical trial of pepinemab antibody in AD. Topline results were presented by Eric Siemers, MD, Principal Investigator of the SIGNAL-AD trial, at the Alzheimer’s Association International Conference in Philadelphia. The clinical trial met its designated primary endpoint of safety by indicating that pepinemab is well-tolerated by patients with AD. No Serious Treatment Emergent Adverse Events Related to Treatment were reported by investigators at any of the 16 clinical sites that participated in this study. The only TEAE leading to discontinuation in the entire trial was in the placebo group. Vaccinex had previously reported that pepinemab was well-tolerated by patients with Huntington’s disease and multiple sclerosis. An important secondary endpoint of the study was to determine whether pepinemab prevents decline in brain metabolic activity consistent with blocking astrocyte reactivity as evidenced by an increase in FDG-PET imaging signal in a major brain region known to be affected by disease progression. This was determined over the course of 12-months treatment with pepinemab relative to placebo. We report that pepinemab treatment resulted in a statistically significant increase in FDG-PET signal in the medial temporal cortex of patients with Mild Cognitive Impairment due to AD.
July 30
CNS Pharmaceuticals announced that it has entered into an exclusive license agreement with Cortice Biosciences. Additionally, CNS announced the launch of its new corporate branding and website, cnspharma.com. Under the terms of the Agreement, CNS Pharmaceuticals has obtained an exclusive license and the intellectual property rights to TPI 287, a potentially blood brain barrier permeable microtubule inhibitor, currently in development for the treatment of GBM, in exchange for an upfront payment of 616,698 shares of the Company’s common stock, as well as the possibility of future success-dependent milestone payments of cash or the Company’s common stock to Cortice. CNS Pharmaceuticals intends to advance the development of TPI 287 for an oncology indication in the United States, Canada, Mexico, and Japan, which is the territory covered by the Agreement. Such development efforts will include, but may not be limited to, the prosecution and maintenance of existing and new intellectual property; preclinical and clinical development of TPI 287 including research, manufacturing, laboratory and clinical testing, regulatory filing, and marketing of TPI 287 in the Territory. TPI 287 Key Highlights: TPI 287 is an abeotaxane and has the same mechanism of action as other taxanes, e.g. paclitaxel and docetaxel, in which it stabilizes microtubules and inhibits cell division, causing apoptosis and cell death. While most taxanes are substrates for multi-drug resistant transporters, which maintain the blood brain barrier, similarly to Berubicin, TPI 287 has shown the potential to cross the BBB and treat CNS tumors. TPI 287 has been well tolerated in over 350 patients to date, including in clinical trials as a monotherapy and in combination with bevacizumab for the treatment of recurrent neuroblastoma and medulloblastoma, as well as refractory prostate cancer and melanoma, and in tauopathy disease, which can result in dementia. In a multicenter Phase 1 study evaluating TPI 287 in combination with bevacizumab in patients with recurrent GBM, results demonstrated an objective response rate of 60% and disease control rate of 96% in 23 subjects. Progression-free survival of 5.5 months and overall survival of 13.4 months compare favorably to bevacizumab either as monotherapy or in combination with chemotherapy in similar patients yielding PFS of 2-4 months and OS of 6-9 months. The data from this study were recently published in a manuscript titled, ” Phase 1 trial of TPI 287, a microtubule stabilizing agent, in combination with bevacizumab in adults with recurrent glioblastoma,” in Neuro-Oncology Advances . CNS Pharmaceuticals plans to engage the FDA and obtain feedback on the design of a study focused on the registration of TPI 287 in recurrent GBM, with the goal of initiating the study in 2025.
VBI Vaccines announced that the Ontario Superior Court of Justice has issued an initial order granting the company protection under the Companies’ Creditors Arrangement Act, R.S.C. 1985, c. C-36, as amended. The Initial Order provides for, among other things: a stay of proceeding in favour of VBI, approval of the DIP Loan, and the appointment of Ernst & Young to serve as monitor in the Court during the restructuring. The decision to seek creditor protection was made in the best interest of its stakeholders after careful evaluation of VBI’s financial situation and all available alternatives following consultation with its legal and financial advisors. The board of directors of VBI will remain in place and VBI will remain responsible for the sale process under the supervision of the CCAA Court and the general oversight of the Monitor. VBI intends to fund the CCAA process from cash on hand as well as through the authorized interim debtor-in-possession financing entered into with K2 HealthVentures, as the secured creditor and DIP lender. VBI intends to seek approval of a sale and investment solicitation process, which, if approved, would allow interested parties to participate in the process in accordance with the SISP procedure. VBI intends to use this process to build on the work it undertook prior to the filing to identify one or multiple purchasers of its assets on an efficient basis. The SISP, if approved by the CCAA Court, will be administered by VBI, with the assistance of its financial advisor and the Monitor, EY. Additional detail relating to the SISP will be disclosed in due course. VBI intends to commence a case under Chapter 15 of the United States Bankruptcy Code to seek recognition and enforcement in the United States of the CCAA Court’s orders, and to commence a case under the relevant provisions of the Israeli Insolvency and Economic Rehabilitation Law, 2018, to protect VBI’s subsidiaries and assets located in the United States and Israel, respectively. The company has notified Nasdaq of the foregoing and expects its common shares will cease trading on the Nasdaq Capital Market upon such date that Nasdaq determines. The company expects to cease reporting as a public reporting company.
Repligen reported Q2 adjusted EPS 33c against a consensus of 33c, and reported Q2 revenue of $154M against a consensus of $154.12M. Tony Hunt, CEO of Repligen said, “We delivered solid second quarter revenue of $154M and first half revenue of $30M, achieving our year-to-date sales target. We are very encouraged by orders in the quarter, with continued strength in Pharma demand and a pickup in CDMO activity. New modalities continued to show strong year-over-year momentum in both revenue and orders. Strategically, we strengthened our Proteins business with new product launches and our pending acquisition of Tantti Laboratory. We continue to expect the second half of 2024 to outpace the first half on both revenue and orders. While we are narrowing our revenue guidance to reflect incremental China weakness, we are confident in Repligen’s ability to resume above-industry growth in the second half of 2024 and into 2025.” Additionally, Repligen said it sees 2024 adjusted EPS $1.42-$1.49 against a consensus of $1.45, and FY24 revenue of $620M-$635M against a consensus of $636.56M.
July 29
Lipella Pharmaceuticals announced the enrollment of the first patients in its multi-center Phase 2a clinical trial evaluating LP-310 for the treatment of Oral Lichen Planus, or OLP. Lipella’s Phase 2a trial is a multi-center, dose-ranging study of LP-310, a proprietary liposomal-tacrolimus oral rinse formulation of the company’s lead candidate LP-10 for hemorrhagic cystitis. The trial has enrolled its first patients across multiple research sites nationwide, spanning from Philadelphia to San Francisco, and is actively screening additional subjects with symptomatic Oral Lichen Planus.
Regenxbio reported new, positive interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy ages 1 to 11 years old. RGX-202 is an investigational one-time AAV Therapeutic. In patients aged 5.8 and 8.5 who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be 77.2%, and 46.5%, respectively, compared to control at three months. As of July 8, 2024, RGX-202 has been well tolerated with no serious adverse events. All seven patients who completed three-month trial assessments indicate meaningful increases in expression of RGX-202 microdystrophin and reduction from baseline in serum creatinine kinase levels, supporting evidence of clinical improvement. Regenxbio expects to complete enrollment in the dose level 2 expansion cohort early in Q3 2024 and has initiated enrollment in the cohort for boys aged 1-3. Initiation of the pivotal trial is expected in Q4 2024. Regenxbio expects to share initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024.
July 26
vTv Therapeutics announced that the FDA has placed a clinical hold on the cadisegliatin clinical program which includes the ongoing CATT1 Phase 3 trial in type 1 diabetes. Cadisegliatin is an oral, liver selective, glucokinase activator that has been well-tolerated in over 500 subjects to date with up to six months of treatment. The clinical hold was based on the discovery of a chromatographic signal in a recent human absorption, distribution, metabolism, and excretion, or ADME, study of cadisegliatin that could not be resolved by standard mass spectroscopy. The Agency requires a single in vitro study to characterize this signal before the cadisegliatin program can resume. No patient had been dosed in CATT1 at the time of the clinical hold, and past clinical studies did not reveal any clinically concerning safety issues.
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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.