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:

• Zynerba Pharmaceuticals (ZYNE), 4,511% surge in interest
• BioXcel Therapeutics (BTAI), 410% surge in interest
• Eloxx Pharmaceuticals (ELOX), 195% surge in interest

Pipeline and key clinical candidates for these companies:

Zynerba Pharmaceuticals is a leader in innovative pharmaceutically produced, synthetic transdermal cannabidiol therapies for orphan neuropsychiatric disorders. The company is committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome and 22q11.2 deletion syndrome.

BioXcel Therapeutics is utilizing artificial intelligence approaches to develop medicines in neuroscience and immuno-oncology. The company’s drug re-innovation approach “leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indices,” BioXcel states. The company’s commercial product, IGALMI, developed as BXCL501, is a proprietary, sublingual film formulation of dexmedetomidine approved for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.

Eloxx Pharmaceuticals’ lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. It is in clinical development, focusing on cystic fibrosis. The FDA has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission.

Recent news on these stocks:

August 14

Harmony Biosciences (HRMY) announced a definitive agreement to acquire Zynerba Pharmaceuticals. Under the terms of the definitive agreement, Harmony will commence a tender offer to acquire all outstanding shares of Zynerba for a purchase price of $1.1059 per share in cash, or $60M in the aggregate, plus one non-tradeable contingent value right per share, representing the right to receive potential additional payments of up to $140M in the aggregate, subject to the achievement of certain clinical, regulatory and sales milestones. Zynerba’s lead asset, Zygel, is the first and only pharmaceutically manufactured, synthetic cannabidiol, a non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. Zygel is currently being evaluated in a pivotal Phase 3 clinical trial for patients living with FXS. Cannabidiol, the active ingredient in Zygel, has been granted orphan drug designation by the FDA and the EMA for the treatment of FXS and for the treatment of 22q. There are currently no FDA-approved therapies to treat 22q. Harmony will fund the transaction from its existing cash on hand. As of June 30, 2023, Harmony had cash, cash equivalents and investment securities of $429.6M. Zynerba’s existing cash and cash equivalent balance was approximately $36.0M as of June 30. The transaction is expected to close by Q4. Zynerba’s board of directors unanimously recommends that Zynerba’s stockholders tender their shares in the tender offer.

BioXcel Therapeutics reported Q2 EPS of ($1.83), consensus ($1.75), and Q2 revenue of $457,000, consensus $450,000. “We are shifting our primary focus to development in the at-home setting while maintaining our value-creating core capabilities ranging from AI innovation to commercialization,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “Our AI-driven drug re-innovation approach has led to the capital-efficient development of product candidates in underserved therapeutic areas. We intend to prioritize our resources to develop BXCL501 for use in assisted living facility and at-home settings and continue to advance our neuroscience pipeline. Unfortunately, this reprioritization requires us to reduce our workforce.” Cash and cash equivalents totaled $127.5M as of June 30. The company is undertaking a strategic reprioritization, which includes a reduction in force of more than 50%, that is expected to reduce expenses significantly. In the absence of additional capital becoming available to the company under the strategic financing agreements or otherwise, the company estimates that its current cash and cash equivalents will last through mid-2024.

Eloxx Pharmaceuticals reported Q2 EPS ($1.96) vs. ($4.90) last year. “This is a transformative time at Eloxx. With today’s confirmation of the disease modifying potential of ELX-02 in all three patient biopsies from the Alport syndrome trial, we look forward to advancing to a pivotal trial of ELX-02 for the treatment of Alport syndrome,” said Sumit Aggarwal, CEO of Eloxx. “We also plan to initiate a clinical study for our lead TURBO-ZM based molecule, ZKN-013, for the potential treatment of recessive dystrophic epidermolysis bullosa with first patient dosing expected by the end of 2023.” Eloxx Pharmaceuticals reported additional positive results from its proof-of-concept Phase 2 open-label clinical trial of ELX-02 for the treatment of Alport syndrome after eight weeks of treatment. Electron microscopy assessment of kidney biopsies demonstrated an improvement in foot process effacement in all three treated patients. These results are consistent with the disease modifying effect of restoration of COL4A4 protein with ELX-02 and with previously reported data from preclinical studies. “These are profound results. The improvement in podocyte foot process effacement in all three patients treated confirms the disease modifying potential of ELX-02. Combined with the reduction of proteinuria achieved in one patient, these data strengthen our confidence in ELX-02’s potential to treat Alport syndrome patients with nonsense mutations,” said Sumit Aggarwal, CEO of Eloxx. “We look forward to initiating a pivotal trial as we believe ELX-02 has the potential to be transformational in this underserved patient population.”

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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.