Terns Pharmaceuticals Announces Orphan Drug Designation Granted to TERN-701 for the Treatment of Chronic Myeloid Leukemia

Rhea-AI Summary

Terns Pharmaceuticals, Inc. receives Orphan Drug Designation from the FDA for TERN-701, a potential treatment for chronic myeloid leukemia. The drug is an allosteric BCR-ABL tyrosine kinase inhibitor in Phase 1 clinical development, with promising interim data expected in the second half of 2024.

Insights

Medical Research Analyst

The Orphan Drug Designation (ODD) by the FDA for TERN-701 marks a significant milestone for Terns Pharmaceuticals in the development of treatments for chronic myeloid leukemia (CML). This designation is indicative of the therapy's potential to fill an unmet medical need. The implications of ODD are multifaceted, offering not just regulatory support and guidance but also financial incentives such as tax credits and fee waivers which can substantially reduce the cost burden of the drug development process. Furthermore, the seven-year marketing exclusivity post-approval is a critical competitive advantage, potentially increasing the commercial viability of TERN-701 should it reach market approval.

From a research perspective, the allosteric mechanism of TERN-701 is noteworthy. Allosteric TKIs target the BCR-ABL tyrosine kinase in a different manner compared to active-site TKIs, potentially offering improved efficacy and safety profiles. This could signal a shift in the standard of care for CML, pending the outcomes of clinical trials. As such, the industry is closely monitoring the progression of TERN-701 through its clinical phases, with interim data from Phase 1 trials being a key inflection point for the company's valuation and future investment considerations.

Financial Analyst

Receiving Orphan Drug Designation is a strong positive signal to investors, often leading to an uptick in a company's stock performance due to the perceived reduction of regulatory risk and the financial benefits associated with the designation. For Terns Pharmaceuticals, this could translate into increased investor confidence and liquidity, potentially improving stock valuation in the short term. The long-term financial impact hinges on successful clinical trial outcomes and eventual market approval. However, it is important to note that the biopharmaceutical sector is highly volatile, with significant investment risks, as clinical trial results can be unpredictable and regulatory hurdles can arise.

Investors will be particularly attentive to the interim data release from the Phase 1 trial of TERN-701. Positive results could lead to a further increase in stock value, while any setbacks may have a negative impact. It is also crucial to evaluate Terns' overall pipeline diversification and financial health, as reliance on a single investigational drug for future revenue growth can be risky. The ODD could also make Terns a more attractive partner or acquisition target, potentially leading to strategic corporate developments.

Oncology Doctor

Chronic myeloid leukemia (CML) is a type of cancer that affects the blood and bone marrow and the development of new treatments is crucial for improving patient outcomes. TERN-701's allosteric inhibition mechanism represents a novel approach in the treatment landscape of CML. Allosteric inhibitors, unlike active-site inhibitors, bind to a site on the enzyme other than the active site, which can result in fewer off-target effects and improved selectivity. This could potentially offer a better safety profile and reduce adverse effects for patients, which is a significant consideration in chronic conditions requiring long-term treatment.

As an oncologist, the FDA's recognition of TERN-701 through the Orphan Drug Designation is encouraging as it suggests a recognition of the therapeutic's potential and a commitment to expedite its development. The clinical community will be looking forward to the interim data from the Phase 1 trials to gauge the drug's efficacy and safety. Such data will be crucial for determining the future treatment protocols and the potential impact on patient care in CML.

FOSTER CITY, Calif., March 11, 2024 (GLOBE NEWSWIRE) -- Terns Pharmaceuticals, Inc. (“Terns” or the “Company”) (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for TERN-701 for the treatment of chronic myeloid leukemia (CML). TERN-701, the Company’s internally discovered allosteric BCR-ABL tyrosine kinase inhibitor (TKI), is in Phase 1 clinical development with interim data from initial dose escalation cohorts expected in the second half of 2024.

“CML is a serious leukemia that requires chronic, life-long treatment,” said Emil Kuriakose, MD, chief medical officer of Terns. “Allosteric TKIs are a novel class of inhibitors that are highly selective and have demonstrated significantly improved clinical efficacy, safety and tolerability compared to prior generation active-site TKIs. Orphan drug designation for TERN-701 underscores the FDA’s recognition of the unmet need for people living with CML and Terns’ commitment to developing new treatment options.”

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

About Terns Pharmaceuticals
Terns Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity. Terns’ pipeline includes three clinical-stage development programs including an allosteric BCR-ABL inhibitor, a small-molecule GLP-1 receptor agonist, a THR-β agonist and preclinical GIPR modulator programs. For more information, please visit: www.ternspharma.com.

Contacts for Terns

Investors
Justin Ng
investors@ternspharma.com

Media
Jenna Urban
Berry & Company Public Relations
media@ternspharma.com

FAQ

What is the significance of the FDA granting Orphan Drug Designation to TERN-701?

The FDA granting Orphan Drug Designation to TERN-701 signifies its potential as a treatment for chronic myeloid leukemia, a serious condition requiring long-term therapy.

What is TERN-701 and its current development stage?

TERN-701 is an allosteric BCR-ABL tyrosine kinase inhibitor being developed by Terns Pharmaceuticals. It is currently in Phase 1 clinical development, with interim data expected in the second half of 2024.

What are the benefits of Orphan Drug Designation for drug developers?

Orphan Drug Designation provides benefits such as assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees, and seven years of post-approval marketing exclusivity.