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: 

• Altimmune (ALT), 509% surge in interest
• BioCryst Pharma (BCRX), 192% surge in interest
• Appellis Pharmaceuticals (APLS), 68% surge in interest
• Aquestive Therapeutics (AQST), 18% surge in interest
• Arrowhead Pharma (ARWR), 15% surge in interest

Pipeline and key clinical candidates for these companies:

Altimmune is a clinical-stage biopharmaceutical company that says it is focused on developing “innovative next-generation therapeutics for the treatment of patients with liver diseases and obesity.” The company’s lead product candidate, pemvidutide, is a GLP-1/glucagon dual receptor agonist that is being developed for the treatment of obesity and NASH. In addition, Altimmune is developing HepTcell, an immunotherapeutic designed to achieve a functional cure for chronic hepatitis B.

BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily Orladeyo is approved in the United States and many global markets. BioCryst has active programs to develop oral medicines for multiple targets across the complement system, including BCX10013, an oral Factor D inhibitor in clinical development. Rapivab is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing.

Apellis Pharmaceuticals says the company “ushered in the first new class of complement medicine in 15 years” with the approval of the first and only targeted C3 therapy. Apellis is advancing this science to “continually develop transformative medicines for people living with rare, retinal, and neurological diseases,” it stated.

Aquestive Therapeutics is a pharmaceutical company advancing medicines to solve patients’ problems with current standards of care and provide transformative products to improve their lives. Aquestive has five commercialized products marketed by its licensees in the U.S. and around the world, and is the exclusive manufacturer of these licensed products. Aquestive is advancing a late-stage proprietary product pipeline focused on treating diseases of the central nervous system and an earlier stage pipeline for the treatment of severe allergic reactions, including anaphylaxis.

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.

Recent news on these stocks:

December 1

Wedbush kept a Neutral rating on Apellis shares after the FDA issued a revised label Syfovre in geographic atrophy following its analysis/review of recent retinal vasculitis events. The FDA’s move was largely anticipated given the focus on the retinal vasculitis events and the label incorporates new language in the warnings/precautions section, the analyst told investors in a research note. The key to unlocking additional Apellis value remains tied to re-accelerating Syfovre commercial uptake and securing ex-U.S. Syfovre approvals, Wedbush contended.

November 30

Altimmune announced topline results from its 48-week MOMENTUM Phase 2 obesity trial of pemvidutide. The trial enrolled 391 subjects with obesity or overweight with at least one co-morbidity and without diabetes. Subjects were randomized 1:1:1:1 to 1.2 mg, 1.8 mg, 2.4 mg pemvidutide or placebo administered weekly for 48 weeks in conjunction with diet and exercise. The 1.2 mg and 1.8 mg doses were administered without dose titration, while a short four-week titration period was employed for the 2.4 mg dose. At baseline, subjects had a mean age of approximately 50 years, mean body mass index, or BMI, of approximately 37 kg/m2 and mean body weight of approximately 104 kg. Approximately 75% of subjects were female. At Week 48, subjects receiving pemvidutide achieved mean weight losses of 10.3%, 11.2%, 15.6% and 2.2% at the 1.2 mg, 1.8 mg, and 2.4 mg doses and placebo, respectively, with a near-linear trajectory of continued weight loss observed on the 2.4 mg dose at the end of treatment. Over 50% of subjects achieved at least 15% weight loss and over 30% of subjects achieved at least 20% weight loss on the 2.4 mg dose. As in prior clinical trials, pemvidutide resulted in robust reductions in serum lipids and improvements in blood pressure without imbalances in cardiac events, arrhythmias or clinically meaningful increases in heart rate. Glucose homeostasis was maintained, with no significant changes in fasting glucose or HbA1c. More subjects receiving pemvidutide stayed on study compared to those receiving placebo, with 74.1% of pemvidutide subjects completing the trial compared to 61.9% of placebo subjects. Nausea and vomiting comprised the majority of adverse events, or AEs, and were predominantly mild to moderate in severity. Only one subject experienced a drug-related serious adverse event, or SAE, a case of vomiting at the 2.4 mg dose. Rates of AEs leading to treatment discontinuation were 6.2% in subjects receiving placebo and 5.1%, 19.2%, and 19.6% in subjects receiving 1.2 mg, 1.8 mg and 2.4 mg of pemvidutide, respectively. Study discontinuations related to study drug occurred in 2.1% of placebo subjects and 4.1%, 16.2% and 15.5% in subjects receiving 1.2 mg, 1.8 mg and 2.4 mg of pemvidutide, respectively, with most discontinuations due to AEs in the pemvidutide groups occurring in the first 16 weeks of treatment. No AEs of special interest or major adverse cardiac events were observed, and there were low rates of cardiac AEs, including arrhythmias, with no imbalance across pemvidutide or placebo groups.

November 29

BioCryst Pharmaceuticals announced that the National Administration of Drugs, Foods, and Medical Devices, ANMAT, in Argentina has granted approval for oral, once-daily ORLADEYO for the prophylaxis of hereditary angioedema, HAE, attacks in adults and pediatric patients 12 years of age or older. “We continue to make strides to bring ORLADEYO to patients living with HAE in Latin America in collaboration with our partner, Pint Pharma. We applaud ANMAT’s decision to grant approval to our oral, once-daily prophylactic therapy for HAE, which paves the way for patients living with HAE to receive ORLADEYO to help improve management of their HAE attacks,” said Charlie Gayer, chief commercial officer of BioCryst. BioCryst has an exclusive collaboration with Pint Pharma GmbH to register and promote ORLADEYO in the pan-Latin America region.

November 28

Arrowhead Pharmaceuticals announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-DM1, the company’s investigational RNA interference RNAi therapeutic being developed as a potential treatment for type 1 myotonic dystrophy DM1 the most common adult-onset muscular dystrophy. ARO-DM1 is designed to reduce expression of the dystrophia myotonica protein kinase DMPK gene. There is currently no approved disease-modifying therapy for DM1. Treatments have focused on symptomatic management, including physical therapy, exercise, ankle-foot orthoses, wheelchairs, and other assistive devices. James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “ARO-DM1 is Arrowhead’s second clinical candidate utilizing our TRiM platform to deliver RNAi therapeutics to skeletal muscle. Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities and may become physically disabled and have a shortened life expectancy. ARO-DM1 represents a novel approach to treat DM1 by silencing aberrantly transcribed DMPK mRNA, which could lead to improvements in multiple symptoms, including muscle strength and function.” An application for approval of the clinical trial was submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials. Pending clearance, Arrowhead intends to proceed with ARODM1-1001, a Phase 1/2a dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DM1 in up to 48 subjects with DM1.

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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.