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:
Morphic Holding is a biopharmaceutical company developing a portfolio of oral integrin therapies for the treatment of serious chronic diseases, including autoimmune, cardiovascular, and metabolic diseases, fibrosis, and cancer. Morphic is also advancing its pipeline and discovery activities in collaboration with Schrodinger using its proprietary MInT technology platform which leverages the company's unique understanding of integrin structure and biology.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.
Applied Therapeutics is developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The company's lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart.
Alkermes plc is a fully-integrated, global biopharmaceutical company developing innovative medicines in the fields of neuroscience and oncology. The company has a portfolio of proprietary commercial products focused on alcohol dependence, opioid dependence, schizophrenia and bipolar I disorder, and a pipeline of product candidates in development for neurological disorders and cancer.
Citius is a late-stage biopharmaceutical company focused on developing and commercializing critical care products, with a focus on oncology, anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapies. The company's pipeline includes two late-stage product candidates, Mino-Lok, an antibiotic lock solution for the treatment of patients with catheter-related bloodstream infections, which is currently enrolling patients in a Phase 3 Pivotal superiority trial, and I/ONTAK, a novel IL-2R immunotherapy for an initial indication in CTCL, for which a BLA is under review by the FDA. Mino-Lok was granted Fast Track designation by the FDA.
Recent news on these stocks:
Alkermes said its "continues to make meaningful progress on the previously announced planned separation of the company's oncology business." The separation "would allow Alkermes to maintain its focus on researching, developing and commercializing therapies for people living with complex neurological conditions and is expected to accelerate and enhance the profitability of the remaining neuroscience business," the company said in its earnings release. Alkermes continues to expect to complete the separation in the second half of 2023, subject to various customary conditions, including final approval from Alkermes' board of directors.
Morphic Therapeutic reported positive topline data from the main cohort of the open-label EMERALD-1 Phase 2a study of MORF-057, an oral small molecule inhibitor of the alpha4beta7 integrin, in adults with moderate to severe ulcerative colitis. "In EMERALD-1, patients with moderate-to-severe ulcerative colitis receiving MORF-057 experienced a statistically significant reduction in the RHI score and a 25.7% clinical remission rate as measured by the mMCS. In addition, MORF-057 was generally well tolerated in EMERALD-1 with no safety signal observed. We are emboldened by these positive results and excited to continue to enroll the ongoing EMERALD-2 Phase 2b study," commented Praveen Tipirneni, MD, Chief Executive Officer of Morphic Therapeutic. "I couldn't be more thankful to these patients and investigators, and proud of our team for advancing MORF-057 much closer to our goal of providing a safe and effective treatment option for IBD patients, in pill form." The main cohort of the EMERALD-1 open-label, single-arm Phase 2a trial of MORF-057 enrolled 35 patients. In the trial, MORF-057 achieved the primary endpoint, demonstrating a statistically significant reduction in the RHI score of 6.4 points from baseline to week 12. In the prespecified secondary endpoint, change in modified Mayo Composite Score, patients had a 2.3-point reduction from baseline. In the study, patients receiving MORF-057 experienced a 25.7% remission rate according to mMCS.
Arrowhead Pharmaceuticals announced interim results from an ongoing Phase 1/2 clinical study of ARO-RAGE, the company's investigational RNA interference therapeutic designed to reduce production of the receptor for advanced glycation end products RAGE as a potential treatment for inflammatory pulmonary diseases, such as asthma. These data represent the first clinical demonstration of the potential utility of Arrowhead's proprietary Targeted RNAi Molecule TRiMTM platform optimized for delivery to the lungs. Matthias Salathe, M.D., Professor, Pulmonary, Critical Care and Sleep Medicine, and Vice Chancellor for Research at the University of Kansas Medical Center, said: "These interim ARO-RAGE Phase 1/2 data are highly encouraging. Unmet need continues to exist for many patients with severe asthma who suffer from persistent symptoms and exacerbations, despite current therapies. Reducing expression of the RAGE protein in pulmonary epithelial cells to the degree that ARO-RAGE has demonstrated to date in this study has the potential to treat patients with asthma and other inflammatory lung diseases in a fundamentally new way. RAGE represents a promising target for intervention as its activation has been implicated as a proximal regulator of the inflammatory cascade in the asthmatic airway, and thus RAGE silencing may result in potent anti-inflammatory effects. I look forward to the availability of additional results from this important trial."
Applied Therapeutics announced results from the ACTION-Galactosemia Kids study of govorestat, AT-007, a novel, oral, small molecule, central nervous system penetrant Aldose Reductase inhibitor. "We believe Govorestat has demonstrated compelling evidence of clinical benefit alongside a favorable safety profile in patients with Galactosemia," said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. "We look forward to working collaboratively with regulators to ensure that this important treatment is available to patients with Galactosemia as quickly as possible. We plan to request a pre-NDA meeting with the FDA, and we have already received feedback from the EMA supportive of a submission in Europe." The ACTION-Galactosemia Kids Phase 3 study was designed to evaluate the impact of govorestat treatment vs. placebo on clinical outcomes over time in 47 children age 2-17 with Galactosemia. The primary endpoint, the Global Statistical Test, was a composite sum of change comprised of four endpoints: OWLS-2 Oral Expression, OWLS-2 Listening Comprehension, BASC-3 Behavior Symptoms Index and the BASC-3 Activities of Daily Living. An additional pre-specified sensitivity analysis included cognition in the primary endpoint. Additional clinical outcomes were assessed as secondary endpoints, including adaptive skills and tremor. Clinical outcomes were assessed every 6 months by a firewalled independent Data Monitoring Committee.
Citius Pharmaceuticals has advanced its clinical trial for Mino-Lok, an antibiotic lock solution to salvage catheters in patients with catheter-related bloodstream infections. Of the 92 events required to complete the trial, the company estimates that 85 events have been achieved to date. The trial has enrolled 190 patients, of which 16 patients are in active treatment or pending study completion data review, which may result in additional events. The multi-center Mino-Lok Phase 3 trial continues to recruit patients at 35 clinical trial sites in the U.S. and India. The primary endpoint for this study is the time to a catheter failure event between randomization and TOC in the Intent-to-Treat Population. Additional secondary outcome measures include overall success, microbiological eradication, and clinical cure, among others.
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.