Biotech Alert: Searches spiking for these stocks today

Stay ahead of biotech stocks seeing a surge in interest from retail and financial professional investors with this exclusive recap from The Fly

These names in the biotech sector are seeing a substantial increase in search activity today, as determined by InvestingChannel. They include: 

• Hepion Pharmaceuticals (HEPA), 1,581% surge in interest
• Avrobio (AVRO), 913% surge in interest
• Akebia Therapeutics (AKBA), 551% surge in interest
• CNS Pharmaceuticals (CNSP), 414% surge in interest
• Krystal Biotech (KRYS), 254% surge in interest

Pipeline and key clinical candidates for these companies:

Hepion Pharmaceuticals’ lead drug candidate, rencofilstat, is a potent inhibitor of cyclophilins, which are involved in many disease processes. Rencofilstat has been shown to reduce liver fibrosis and hepatocellular carcinoma tumor burden in experimental disease models and is currently in Phase 2 clinical development for the treatment of NASH. In November 2021, the FDA granted Fast Track designation for rencofilstat for the treatment of NASH. That was followed in June 2022 by the FDA’s granting of Orphan Drug designation to rencofilstat for the treatment of HCC.

Avrobio targets the root cause of genetic disease by introducing a functional copy of the affected gene into the patient’s own hematopoietic stem cells, with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. The company’s pipeline includes clinical programs for Gaucher disease and cystinosis, as well as preclinical programs for Hunter syndrome and Pompe disease.

Akebia Therapeutics is a fully integrated biopharmaceutical company that says its purpose is "to better the lives of people impacted by kidney disease."

CNS Pharmaceuticals is 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.

Krystal Biotech is a biotechnology company focused on developing and commercializing genetic medicines for patients with rare diseases. The company’s wide-ranging pipeline is based on its proprietary redosable HSV vector. In May 2023, Vyjuvek was approved by the FDA for the treatment of dystrophic epidermolysis bullosa. Vyjuvek is a non-invasive, topical, redosable gene therapy designed to treat DEB at the molecular level.

Recent news on these stocks:

May 22

Hepion Pharmaceuticals announced positive topline results from its recently completed Phase 2 ALTITUDE-NASH clinical trial. ALTITUDE-NASH met its primary endpoint by demonstrating improved physiologic liver function and was well tolerated after four months of treatment in subjects with stage 3 or greater fibrosis based on the AGILE 3+ criteria. All additional secondary endpoints were also met, including reductions in the liver injury biomarkers, alanine and aspartate transaminases; and multiple fibrosis-associated biomarkers, including ProC3, PIIINP, TIMP1, hyaluronic acid, and enhanced liver fibrosis scores. These observations build on similar findings from a shorter Phase 2a trial and reinforce rencofilstat’s direct antifibrotic mode of action and increase the confidence for reductions in fibrosis in Hepion’s ongoing Phase 2b ASCEND-NASH paired biopsy trial. "We are thrilled that the ALTITUDE-NASH trial met both its primary efficacy and safety endpoints, in particular with the 225 mg rencofilstat dose showing the greatest benefit to liver function and multiple NASH-associated biomarkers," said Todd Hobbs, MD, Hepion’s Chief Medical Officer. "This trial was designed to inform us on how well rencofilstat improves hepatic function in those with significant impairment and risk for complications from their advanced NASH. Further, it provided an opportunity to evaluate data generated from different doses of rencofilstat. I am very pleased with the research team and the ALTITUDE-NASH sites that quickly recruited this study. Our improved understanding of which subjects best respond to rencofilstat can be immediately applied to increase the likelihood of success of our larger and longer ASCEND-NASH paired biopsy trial."

Avrobio announced an agreement to sell its investigational hematopoietic stem cell, or HSC, gene therapy program for the treatment of cystinosis to Novartis (NVS) for $87.5M in cash. Avrobio retains full rights to its portfolio of HSC gene therapies for Gaucher disease type 1 and type 3, Hunter syndrome and Pompe disease. Proceeds from this transaction are expected to extend the company’s cash runway into the fourth quarter of 2024. Pursuant to the terms of an asset purchase agreement, Novartis will pay Avrobio $87.5M in cash at closing, in consideration for the sale and transfer of certain assets related to the cystinosis program. In addition, Avrobio has exclusively licensed to Novartis certain other assets, know-how and other intellectual property related to Avrobio’s gene therapy platform for use in cystinosis. To support the transition of the program, Avrobio also has agreed to provide under a separate agreement certain transition, knowledge transfer and other related services.

Akebia announced that the UK Medicines and Healthcare products Regulatory Agency – MHRA – has granted marketing authorization for Vafseo, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor for the treatment of symptomatic anemia associated with chronic kidney disease in adults on chronic maintenance dialysis. Anemia due to CKD, common among patients on dialysis, is often associated with adverse clinical outcomes. In the study of adult patients on dialysis, vadadustat achieved the primary and key secondary efficacy endpoints, demonstrating non-inferiority to darbepoetin alfa as measured by a mean change in hemoglobin between baseline and the primary evaluation period and secondary evaluation period. Vadadustat also achieved the primary safety endpoint of the INNO2VATE program, defined as non-inferiority of vadadustat versus darbepoetin alfa in time to first occurrence of major adverse cardiovascular events, which is the composite of all-cause mortality, non-fatal myocardial infarction, or non-fatal stroke across both INNO2VATE studies.

Krystal Biotech said it sees cash runway through the end of 2026. "We are extremely pleased to have the support of this strong investor group," said Krish S Krishnan, CEO of Krystal Biotech. "These additional funds, together with our existing cash, cash equivalents and investments, should allow us to fund the VYJUVEK launch, future operations, and the advancement of our growing pipeline through the end of 2026. It also allows us to retain the optionality of monetizing the Priority Review Voucher we received in connection with the FDA approval of VYJUVEK at a future date with favorable terms and without any dilution."

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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.

Keywords: biotech, biotech sector, biotech alert, investingchannel, XBI, SPDR S&P Biotech ETF