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: 

• Insmed (INSM), 2,821% surge in interest
• Agios Pharmaceuticals (AGIO), 819% surge in interest
• Exicure (XCUR), 755% surge in interest
• Arcturus Therapeutics (ARCT), 442% surge in interest
• Biohaven (BHVN), 428 surge in interest
• Corcept Therapeutics (CORT), 271% surge in interest
• Summit Therapeutics (SMMT), 188% surge in interest
• BioLine RX (BLRX), 303% surge in interest
• Sarepta Therapeutics (SRPT), 197% surge in interest

Pipeline and key clinical candidates for these companies:

Insmed Incorporated is a biopharmaceutical company that says its on “a mission to transform the lives of patients with serious and rare diseases.” Insmed’s first commercial product is a first-in-disease therapy approved in the United States, Europe, and Japan to treat a chronic, debilitating lung disease.

Agios says it is “the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases.” In the U.S., Agios markets a first-in-class pyruvate kinase, PK, activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera, and a preclinical PAH stabilizer as a potential treatment for phenylketonuria.

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.

Arcturus Therapeutics is a late-stage clinical mRNA medicines and vaccines company whose pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for COVID-19 and Influenza, and other programs to potentially treat ornithine transcarbamylase, or OTC, deficiency, and cystic fibrosis, or CF. The company’s partnered programs include glycogen storage disease type III and hepatitis B virus.

Biohaven is a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology. The company is advancing its portfolio of therapeutics, leveraging its proven drug development experience and multiple proprietary drug development platforms. Biohaven’s clinical and preclinical programs include Kv7 ion channel modulation for epilepsy and mood disorders; extracellular protein degradation for immunological diseases; TRPM3 antagonism for migraine and neuropathic pain; TYK2/JAK1 inhibition for neuroinflammatory disorders; glutamate modulation for OCD and SCA; myostatin inhibition for neuromuscular and metabolic diseases, including SMA and obesity; and antibody recruiting, bispecific molecules and antibody drug conjugates for cancer.

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.

Summit Therapeutics is initiating development activities for SMT112 and will do so first in NSCLC indications. Summit plans to start treating patients in clinical studies by the second quarter of 2023.

BioLine RX is a pre-commercial-stage biopharmaceutical company focused on oncology. The company’s lead development program, motixafortide, a novel selective inhibitor of the CXCR4 chemokine receptor, may support diverse therapeutic approaches in oncology and other diseases. APHEXDA was successfully evaluated in a Phase 3 study in stem cell mobilization for autologous transplantation for multiple myeloma patients, has reported positive results from a pre-planned pharmacoeconomic study in the U.S., and has had its NDA submission accepted by the FDA with an assigned PDUFA date of September 9, 2023.

Sarepta Therapeutics engineers precision genetic medicine for rare diseases. The company holds leadership positions in Duchenne muscular dystrophy, or DMD, and limb-girdle muscular dystrophies, or LGMDs, and currently has more than 40 programs in various stages of development. Sarepta’s pipeline is driven by its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

Recent news on these stocks:

May 30

BioLine RX announced a multi-center Phase 1 clinical trial sponsored by St. Jude Children’s Research Hospital, Inc. to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells used in the development of gene therapies for patients with sickle cell disease. Investigators in the trial from St. Jude Children’s Research Hospital, Inc. and two other clinical sites have extensive SCD gene therapy clinical development experience and are recognized leaders in the field.

May 29

Biohaven unveiled new positive data from its ongoing Phase 1 single ascending dose, or SAD, study with BHV-1300, a first-in-human IgG degrader that uses an ASGPR-bispecific from its MoDE platform. Emerging results in healthy subjects confirm that BHV-1300 rapidly and selectively lowers IgG in a dose-dependent manner in the first 4 cohorts completed to date. Preliminary IgG lowering data is consistent with modeling, with dose- and time-dependent IgG lowering observed even in initial low-dose cohorts. Some subjects experienced IgG reductions as low as 50 to 70% of baseline. BHV-1300 demonstrated reduction of IgG without significantly impacting LFTs, albumin, LDL cholesterol or other serum labs. BHV-1300 has been safe and well tolerated to date, with no serious or severe adverse events. Most AEs were mild, deemed unrelated to study drug and resolved spontaneously. As expected from the selectivity of the molecule for IgG, when compared to placebo, there were no meaningful reductions in average IgA, IgM or IgE levels during the week after dosing. No adverse trends have been observed in vital signs or ECGs. Given the levels of IgG lowering observed to date, the company plans to evaluate approximately 6 cohorts of BHV-1300. Modeling suggests additional cohorts in the Phase 1 study will achieve greater than 70% lowering of IgG utilizing doses compatible with subcutaneous administration. Given the promising results of the SAD study thus far, the MAD study will proceed in patients with rheumatoid arthritis.

Sarepta is replacing ShockWave Medical (SWAV) in the S&P 400 at the open on June 3, as Johnson & Johnson (JNJ) is acquiring Shockwave Medical in a deal expected to be completed soon pending final closing conditions.

May 28

Insmed announced topline results from the ASPEN study, a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety, and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis. The study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of pulmonary exacerbations, or PEs, versus placebo. The study also met several of its prespecified secondary endpoints with statistical significance. Based on these results, Insmed plans to file a New Drug Application, or NDA, with the FDA for brensocatib in patients with bronchiectasis in the fourth quarter of 2024. Pending regulatory approvals, Insmed anticipates a U.S. launch for brensocatib in mid-2025 followed by launches in Europe and Japan in the first half of 2026. If approved, brensocatib would be the first approved treatment for patients with bronchiectasis as well as the first approved dipeptidyl peptidase 1, or DPP1, inhibitor-a new mechanism of action with the potential to address a range of neutrophil-mediated diseases. As part of the ASPEN study’s conduct, more than 460 trial sites were engaged in nearly 40 countries. After excluding sites that did not enroll any patients and all sites in Ukraine, the total number of active sites in ASPEN was 391 sites in 35 countries. Adult patients were randomized 1:1:1 and adolescent patients were randomized 2:2:1 for treatment with brensocatib 10 mg, brensocatib 25 mg, or placebo once daily for 52 weeks, followed by 4 weeks off treatment. The primary efficacy analysis included data from 1,680 adult patients and 41 adolescent patients. Brensocatib was well-tolerated in the study. Treatment-emergent adverse events (TEAEs) occurring in at least 5.0% of patients treated with either dose of brensocatib and more frequently than in placebo were COVID-19, nasopharyngitis, cough, and headache for brensocatib 10 mg, brensocatib 25 mg, and placebo, respectively.

Agios Pharmaceuticals announced that the company has agreed to sell its rights to its 15% royalty on potential U.S. net sales of Servier’s vorasidenib to Royalty Pharma (RPRX). Under the terms of the agreement, Agios will receive an upfront payment of $905M upon approval of vorasidenib by the FDA and Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1B, and a 12% royalty on annual U.S. net sales greater than $1B. Agios will retain a 3% royalty on annual U.S. net sales greater than $1B. Vorasidenib is an oral, selective, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 enzymes for the treatment of IDH-mutant diffuse glioma. In 2021, Agios completed the sale of its oncology portfolio – including vorasidenib – to Servier. As part of that divestiture, Agios is owed a milestone payment of $200M upon vorasidenib’s approval by the FDA, as well as a 15% royalty on U.S. net sales of vorasidenib. Agios continues to retain the right to the approval milestone from Servier. Servier announced that the FDA has designated a Prescription Drug User Fee Act action date of August 20, 2024.

Arcturus Therapeutics announced that it will be presenting Phase 1 results in healthy volunteers and new Phase 1b interim data in CF patients for ARCT-032, an inhaled investigational mRNA therapeutic to treat CF, at the 47th European Cystic Fibrosis Conference on June 7, 2024. “We were pleased to observe ARCT-032 treatments for the first four patients were generally safe and well tolerated with no serious adverse events,” said Joseph Payne, President & CEO of Arcturus Therapeutics. “Furthermore, we observed an encouraging trend towards lung function improvements in the first four CF participants after only two inhaled administrations.” “The absolute change in percent predicted FEV1 (Forced Expiratory Volume in 1 second) in the first four CF participants was observed to be an average of 4.0% at day 8,” said Dr. Juergen Froehlich, Chief Medical Officer of Arcturus Therapeutics. “I am pleased to announce our team will present this new Phase 1b interim data at the upcoming European CF conference in Scotland, supplementing the favorable results from our Phase 1 single ascending dose study in healthy volunteers.”

Corcept Therapeutics announced that GRACE, the Phase 3 trial of its proprietary selective cortisol modulator relacorilant in patients with hypercortisolism, met its primary endpoint. The GRACE study has two parts. On April 22, Corcept announced that patients in GRACE’s initial, open-label phase exhibited clinically meaningful and statistically significant improvements in hypertension, hyperglycemia and other symptoms experienced by patients with Cushing’s syndrome. Patients who exhibited pre-specified improvements in hypertension, hyperglycemia or both were given the opportunity to enter the trial’s randomized, double-blind withdrawal phase, in which half of the patients continued to receive relacorilant and half received placebo, for 12 weeks. GRACE met its primary endpoint of loss of blood pressure control in the randomized withdrawal phase among patients receiving relacorilant as compared to placebo. Consistent with its known safety profile, relacorilant was well-tolerated in both phases of GRACE, with no differences in the randomized withdrawal phase between the relacorilant and placebo groups. Additional data will be presented at the Endocrine Society annual meeting in Boston and the Heart in Diabetes conference in Philadelphia. The company expects to submit its New Drug Application in the third quarter.

May 24

In a 8-K filing, Summit Therapeutics stated: “On May 24, 2024, the National Medical Products Administration, the regulatory authority in China responsible for providing marketing authorization for clinical drug candidates, has approved ivonescimab in combination with chemotherapy for use in patients with epidermal growth factor receptor mutated, locally advanced or metastatic non-squamous non-small cell lung cancer, or NSCLC, who have progressed after treatment with an EGFR tyrosine kinase inhibitor. This is the first approval for ivonescimab by any regulatory authority. The approval for ivonescimab was based on the trial called HARMONi-A (AK112-301), a single region Phase III clinical trial conducted in China comparing ivonescimab plus chemotherapy vs. placebo plus chemotherapy in the aforementioned setting. The trial was sponsored by our partner, Akeso, Inc. (Akeso). Data for this trial was generated and analyzed by Akeso. This data is planned to be released at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting during an oral presentation on Friday, May 31, 2024, scheduled for 4:57pm CT. On May 23, 2024, ASCO released abstracts for presentations to take place during the Annual Meeting. Included in these abstracts, was topline data associated with HARMONi-A. Notably, patients receiving ivonescimab plus chemotherapy experienced a median progression free survival (PFS) by independent radiology review committee of 7.06 months (95% CI: 5.85 – 8.74) as compared to 4.80 months (95% CI: 4.21 – 5.55) for those patients receiving chemotherapy alone (hazard ratio: 0.46; 95% CI: 0.34 – 0.62). Grade 3 or higher treatment emergent adverse events, or TEAEs, occurred in 61.5% patients receiving ivonescimab plus chemotherapy as compared to 49.1% patients receiving chemotherapy; the most common grade of 3 of higher in TEAEs were chemotherapy related adverse events. Additional data and context including, but not limited to, response rates, stable disease rates, progression free survival, overall survival, and safety is expected to be made available during ASCO’s Annual Meeting.”

May 23

Exicure announced it received notice of a delisting determination from the Listing Qualifications Department of The Nasdaq Stock Marke. The Staff Delisting Determination notified the company that since it has not yet filed its Form 10-K for the year ended December 31, 2023 per Nasdaq Listing Rule 5250(c)(1) by Nasdaq’s extended deadline of May 20, 2024 pursuant to the previously granted exception, trading of the company’s common stock would be suspended from The Nasdaq Capital Market at the opening of business on May 30, 2024, unless the company requests an appeal of the Staff Delisting Determination by May 28, 2024. In addition, unless an appeal is timely requested, a Form 25-NSE would be filed with the SEC, which would remove the company’s securities from listing and registration on Nasdaq. In addition to the delinquency related to the Form 10-K, the Staff Delisting Determination noted the company’s delinquency under the Rule because of its failure to file its Form 10-Q for the first quarter of 2024 and the company’s continued delinquency under Rule 5620 because it has not held its 2023 Annual Meeting of Stockholders, each as an additional and separate basis for delisting that should be addressed in any appeal. The company intends to file an appeal to Nasdaq’s Hearings Panel by the May 28, 2024 deadline and subsequently submit its plan to regain compliance with all applicable listing requirements. This request for an appeal, if timely submitted, would automatically stay the suspension of trading in the company’s securities for a period of 15 days from the date of the request. The company intends to seek an extended stay pending the hearing, although no assurance can be provided that such an extension would be granted.

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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.