The Fort Worth Press - Adolore BioTherapeutics Announces FDA Orphan Drug Designation for its Kv7-activating rdHSV-CA8* Therapy in the Treatment of Erythromelalgia

DELRAY BEACH, FL / ACCESS Newswire / January 14, 2026 / Adolore BioTherapeutics ("Adolore" or the "Company"), a biotechnology company focused on developing breakthrough treatments for neurological disorders, including chronic pain, with opioid-free locally-administered gene-therapies, today announced the FDA Office of Orphan Products Development has approved an Orphan Drug Designation, (ODD), for its innovative Kv7 activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia, (EM).

"Approval of this ODD underscores the importance of treating EM and recognizes the rationale for treatment with our novel approach. Upon FDA approval, Adolore may be eligible for up to seven years of U.S. market exclusivity for this indication, along with other development incentives available under the Orphan Drug Act.This designation, together with emerging clinical data, may support consideration of expedited development and review pathways intended to enable faster patient access," commented Roelof Rongen, CEO of Adolore. "This ODD approval has the potential for adding significant value and further validates Adolore's transformational gene-therapy approach as we progress toward commercialization."

In Primary EM, gain-of-function mutations in the SCN9A gene encoding for the Nav1.7 sodium channel, primarily found in sensory and some autonomic nerves, cause these sodium channels to become more easily activated, leading to hyperexcitability in pain sensing neurons. Activation of Kv7 channels by Adolore's CA8* gene therapy can reverse this hyperexcitability, thus targeting the underlying root cause of EM. Further validation of our approach comes from genetic studies of EM families showing that concurrent activating mutations of Kv7-potassium channels can overcome the severe chronic pain caused by neuronal Nav1.7 sodium channel mutations.

"Adolore's gene therapy could address EM patients' significant and unmet medical need because there is no FDA approved therapy. Existing therapies target EM symptoms but are largely ineffective, leaving EM patients with debilitating pain" said Roy Clifford Levitt, MD, pain physician, inventor and founder/chairman of Adolore. "Adolore has generated compelling safety, biodistribution, shedding, histology, and clinical safety data; as well as substantial preclinical efficacy data for its localized rdHSV-CA8* gene therapy. These largely published data demonstrate Kv7 voltage-gated potassium channel activation by a single dose of rdHSV-CA8* gene therapy, reversal of neuronal hyperexcitability, and the production of profound long-lived analgesia, equipotent to opioid treatment, but without any of the opioid-related side effects. These preclinical data strongly support progression of the EM program toward the clinic."

There are currently very few safe, sufficiently potent efficacious non-opioid analgesic treatments for chronic pain on the market, leaving a large and very urgent unmet medical need given the opioid crisis. Leveraging its innovative rdHSV-CA8* gene therapy, Adolore is currently advancing two preclinical development programs:

1. The Company's lead development program for the treatment of chronic pain due to knee osteoarthritis is funded by a UG3/UH3 grant from the NIH/NINDS/HEAL Program for all formal GLP/GMP/GCP development work through a first-in-human study in patients, expected to commence in 2027.

2. This ODD approval provides for further rationale and momentum for Adolore's rdHSV-CA8* gene therapy in this second application - the treatment of the relatively rare genetically-determined life-long recurrent severe neuropathic pain due to EM.

Development of our rdHSV-CA8* gene therapy in EM is of strategic importance to the Company, as it creates an additional path to treating patients with high-unmet-need in clinical studies and may support future consideration of expedited development and review pathways, potentially enabling faster patient access and product commercialization..

About Erythromelalgia

Approximately 50,000 patients in the U.S. (~200,000 in economically developed countries across the world) are suffering predominantly from primary EM. Primary EM is a rare autosomal dominant heritable pain disorder that is most often caused by gain-of-function mutations in the SCN9A gene which codes for Nav1.7 voltage-gated sodium channels, primarily found in sensory and some autonomic nerves. These mutations cause these sodium channels to become more easily activated, leading to hyperexcitability in pain sensing neurons. EM patients typically experience severe burning pain, redness, heat and sometimes swelling. These symptoms often arise as debilitating attacks, which can last minutes to hours and are triggered by minimal stressors including heat (warm rooms, hot weather, hot showers), exercise (walking), wearing warm socks or tight shoes, and alcohol consumption. Treatment regimens typically involve minimizing these stressors and symptomatic treatment with ice-baths, topical local anesthetics, and sometimes opioids.

About Carbonic Anhydrase-8 (CA8*) Gene Therapy

CA8* (*Carbonic anhydrase-8 like analgesic peptides, CA8 variants) gene therapies are a novel class of neuronal intracellular calcium release channel inhibitors that indirectly activate Kv7 voltage-gated potassium channels. These therapies are locally administered and are long-acting. Precision targeting of Kv7 potassium channels directly as a drug target has been proven very challenging due to off-target effects associated with small-molecule activators. Oral therapeutics that activate Kv7 voltage-gated potassium channels demonstrated significant analgesic and anti-epilepsy efficacy before they were removed from the market due to severe adverse events related to systemic small-molecule exposure. Locally administered rdHSV-CA8* gene therapy provides a highly targeted efficient intracellular delivery approach and versatile dosing regimens, including intra-articular, intra-neuronal (nerve block) and intradermal injection. The non-opioid CA8* mechanism-of-action addresses neuropathic, inflammatory, and nociceptive pain, which apply to a broad range of neuronal conditions including chronic pain indications, such as osteoarthritis pain, diabetic- and other forms of peripheral-neuropathy, post-herpetic neuralgia, lower back pain, ocular and cancer pain, as well as rare pain conditions such as erythromelalgia, an orphan drug disease. Additional potential indications include epilepsy and hearing loss. Using a replication-defective HSV vector enables disease-free localized delivery to inside the peripheral somatosensory nervous system with an excellent safety profile. Preclinical studies show Kv7-activating rdHSV-CA8* gene-therapy is equipotent to opioid-induced-analgesia.

About Adolore BioTherapeutics, Inc.

Adolore BioTherapeutics, Inc., is a biotechnology company focused on developing novel therapies for the treatment of chronic pain and other nervous system conditions or disorders. Our best-in-class programs are long-acting, locally acting gene-therapies that are opioid-free Disease Modifying Anti-Pain therapies (DMAPs) for the treatment of chronic pain. The Company's two current CA8* gene therapy programs are in preclinical development for treatment of patients suffering from erythromelalgia, a life-long heritable chronic pain condition representing an orphan drug disease with no approved therapy, and chronic osteoarthritis knee pain, affecting a large number of patients that is often treated with opioids due to the lack of good alternatives, thus contributing to the ongoing opioid crisis.

For more information, visit adolore.com.

Forward Looking Statements

To the extent this announcement contains information and statements that are not historical, they are considered forward-looking statements within the meaning of the federal securities laws. You can identify forward-looking statements by the use of the words "believe," "expect," "anticipate," "intend," "estimate," "project," "will," "should," "may," "plan," "intend," "assume" and other expressions which predict or indicate future events and trends and which do not relate to historical matters. You should not rely on forward-looking statements, because they involve known and unknown risks, uncertainties and other factors, some of which are beyond the control of the Company. These risks and uncertainties include, but are not limited to, those associated with drug development. These risks, uncertainties and other factors may cause the actual results, performance or achievements of the Company to be materially different from the anticipated future results, performance or achievements expressed or implied by the forward-looking statements.

Investor Relations Contact

Paul Barone
(215)622-4542
[email protected]

SOURCE: Adolore Biotherapeutics, Inc.

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S.Jordan--TFWP