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: 

• ProQR Therapeutics (PRQR), 8,833% surge in interest
• Salarius Pharmaceuticals (SLRX), 3,600% surge in interest
• Bright Minds Biosciences (DRUG), 3,289% surge in interest
• Lipella Pharmaceuticals (LIPO), 2,245% surge in interest
• Wave Life Sciences (WVE), 1,346% surge in interest
• MeiraGTx (MGTX), 1,106% surge in interest
• Hoth Therapeutics (HOTH), 1,012% surge in interest
• Aprea Therapeutics (APRE), 716% surge in interest
• Seelos Therapeutics (SEEL), 442% surge in interest
• Exicure (XCUR), 410% surge in interest

Pipeline and key clinical candidates for these companies:

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.

Salarius Pharmaceuticals is a clinical-stage biopharmaceutical company developing therapies for patients with cancer in need of new treatment options. Salarius’ product portfolio includes seclidemstat, its lead candidate, which is being studied as a potential treatment for pediatric cancers, sarcomas and other cancers with limited treatment options, and SP-3164, an oral small molecule protein degrader being developed for the treatment of non-Hodgkin’s lymphoma. Salarius has received financial support from the National Pediatric Cancer Foundation to advance the Ewing sarcoma program.

Bright Minds is focused on developing novel transformative treatments for neuropsychiatric disorders, epilepsy, and pain. “Bright Minds has a portfolio of next-generation serotonin agonists designed to target neurocircuit abnormalities that are responsible for difficult to treat disorders such as resistant epilepsy, treatment resistant depression, PTSD, and pain. Bright Minds’ drugs have been designed to potentially retain the powerful therapeutic aspects of psychedelic and other serotonergic compounds, while minimizing the side effects, thereby creating superior drugs to first-generation compounds, such as psilocybin,” the company has stated.

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.”

Wave Life Sciences is a biotechnology company “focused on unlocking the broad potential of RNA medicines to transform human health” whose RNA medicines platform, PRISM, combines multiple modalities, chemistry innovation and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders, the company says. Wave’s pipeline includes clinical programs in Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity.

MeiraGTx is a vertically integrated, clinical-stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx says its initial focus is on three distinct areas of unmet medical need: ocular diseases, including both inherited retinal diseases as well as large degenerative ocular diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system, and salivary gland, MeiraGTx plans to expand its focus to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

Hoth Therapeutics refers to itself as “a catalyst in early-stage pharmaceutical research and development, elevating drugs from the bench to pre-clinical and clinical testing.” Hoth “collaborates and partners with a team of scientists, clinicians, and key opinion leaders to seek out and investigate therapeutics that hold immense potential to create breakthroughs and diversify treatment options,” the company stated.

Aprea Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology through synthetic lethality. The company’s lead program is ATRN-119, a clinical-stage small molecule ATR inhibitor in development for solid tumor indications. Aprea has completed all IND enabling studies for its oral, small molecule WEE1 inhibitor, APR-1051, and recently received FDA clearance of its IND.

Seelos Therapeutics is focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system, or CNS, disorders and other rare diseases. The company’s portfolio includes several late-stage clinical assets targeting indications including Acute Suicidal Ideation and Behavior in Major Depressive Disorder, amyotrophic lateral sclerosis, or ALS, and spinocerebellar ataxia, or SCA, as well as early-stage programs in Huntington’s disease, Alzheimer’s disease, and Parkinson’s disease.

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.

Recent news on these stocks:

October 16

JonesResearch raised the firm’s price target on Wave Life Sciences to $20 from $16 and kept a Buy rating on the shares after the company reported initial proof-of-mechanism for its lead RNA editing candidate, WVE-006. “In an impressive win,” Wave saw proof of RNA editing in the first two patients to reach the 57-day mark, the analyst tells investors in a research note. Jones points out shares of Korro Bio (KRRO) and ProQR Therapeutics are up in sympathy with Wave’s readout. The firm sees this as a positive readout for Wave, demonstrating “definitive proof-of-mechanism after just a single dose.” Jones increased its probability of success for WVE-006 in alpha-1 antitrypsin deficiency to 35% from 15%, and increased penetration to 25% from 15%.

Firefly Neuroscience (AIFF) announced collaborating with Bright Minds Biosciences to analyze the data from its first-in-human Phase 1 study of its lead compound, BMB-101. The study demonstrated results of the qEEG data. During the EEG recording, subjects were seated with a FDA approved 19 electrode EEG headset provided by Firefly strategic partner, Zeto Inc. Channels were sampled at 250 or 500 Hz and referenced to A1/A2 channels during recording. The EEG recording time was 10 minutes. There were four EEG recording timepoints: day 1 pre-dose and post-dose, and day 7 pre-dose and post-dose. Data was analyzed using the FireFly Neuroscience advanced EEG analysis platform.

October 15

Lipella Pharmaceuticals announced the receipt of a notice of allowance from the U.S. Patent and Trademark Office, or USPTO, for its proprietary liposomal drug delivery platform. The patent application, U.S. Patent No. 17/829,960, titled “Delivery of Agents Using Metastable Liposomes,” covers key innovations in Lipella’s platform technology for delivering therapeutic agents via liposome-based vehicles. The patent allows claims that cover Lipella’s method for using metastable liposomes to deliver a variety of therapeutic agents, including the company’s lead assets, LP-10 and LP-310. This technology enables precise, targeted delivery, improving the safety and efficacy of treatments across multiple therapeutic areas, including oncology, cancer survivorship, and immunotherapy. This newly allowed patent provides broad intellectual property protection for Lipella’s drug delivery platform, which optimizes delivery to epithelial tissues such as those lining the mouth, bladder, colon, esophagus, vagina, and urethra. It extends market exclusivity and strengthens Lipella’s competitive position, particularly for its two lead clinical assets currently in Phase 2 trials. With additional patents covering the company’s formulations in the U.S., Australia, and Canada until 2035, Lipella is poised for further growth and development as it continues to advance its clinical pipeline.

MeiraGTx announced top-line data from its clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease, MGT-GAD-025. Top-line data summary: AAV-GAD was safe and well tolerated, with no serious adverse events related to AAV-GAD treatment. At Week 26, a statistically significant 18-point average improvement from baseline in UPDRS Part 3 “off” medication score was demonstrated in the high dose group, with no significant change in the sham or low dose groups. Significant improvements from baseline in the disease-specific, patient-reported quality of life PDQ-39 score were demonstrated in both the high and low dose groups with no significant change in the sham group at Week 26: In the high dose AAV-GAD group, the PDQ-39 score improved by 8 points from baseline, the low dose group improved by 6 points from baseline, while the 0.2 point worsening in the sham surgery group was not statistically significant. A dose response in PDQ-39 score was observed, with 100% of participants in the high dose group, 60% of participants in the low dose group, and 25% of participants in the sham surgery group reporting an improvement. For the PDQ-39 score, there was a trend to significance between the high dose and sham surgery groups at 6 months.

Hoth Therapeutics announced the granting of a U.S. patent for its pioneering Alzheimer’s treatment, HT-ALZ. This patent secures the company’s intellectual property rights to its novel therapeutic approach, as Hoth accelerates preparations for clinical trials.

Seelos Therapeutics received notice that the Nasdaq Hearings Panel had determined to delist the company’s common stock from Nasdaq due to the company’s failure to comply with the minimum stockholder’s equity requirement. Trading in the company’s common stock will be suspended on Nasdaq effective with the open of business on October 16. As a result, the company expects its common stock to begin trading on the OTCQB on that date.

October 9

Aprea Therapeutics announced that it has engaged Philippe Pultar, MD as its senior medical advisor to support Aprea with developing and advancing APR-1051, Aprea’s potential best in class WEE1 inhibitor. Dr. Pultar is a seasoned pharmaceutical executive with extensive experience in oncology, including the development of a WEE1 inhibitor from early to late-stage clinical development. Aprea intends to provide an update on the progress of this clinical study by year end 2024. The WEE1 program is part of Aprea’s portfolio of DDR-targeted therapeutics aimed to deliver precision medicine solutions that ensure the right patients receive the most effective Aprea treatment, with the goals of improving outcomes and reducing treatment resistance.

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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.