Five Companies Earn Validation as FDA Recognizes New Breakthrough Therapies

FDA

The U.S. Food and Drug Administration (FDA) granted Novartisexperimental drug iptacopan Breakthrough Therapy designation in paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder.

Iptacopan (LNP023) is a potential first-in-class oral factor B inhibitor in development for several rare renal diseases and a hematological disorder. The FDA granted Breakthrough Therapy designation to iptacopan for the treatment of PNH based on positive interim results from two ongoing Phase II studies, where iptacopan showed substantial benefits both in patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment. Iptacopan also demonstrated positive interim results as a monotherapy treatment in anti-C5 naïve PNH patients.

Despite current standard of care, which is typically anti-C5 therapy eculizumab or treatment with ravulizumab, a large proportion of PNH patients remain anemic and dependent on transfusion, Novartis said. Breakthrough Therapy designation will expedite the development and review of iptacopan for this indication.

In addition to Breakthrough Therapy designation, the FDA also awarded Rare Pediatric Disease designation to iptacopan in C3 glomerulopathy.

Iptacopan isn’t the only drug to receive Breakthrough Therapy designation this week. Several other experimental treatments were also awarded the designation.

IO Biotech – Denmark-based IO Biotech said the FDA awarded Breakthrough Therapy designation for the potential combination therapy IO102 and IO103 with anti-PD-1 mAb for patients with unresectable/ metastatic melanoma.

IO102 and IO103 are IO Biotech's lead immuno-oncology candidates. Both compounds are based on IO Biotech's proprietary T-win technology platform which enables the identification of compounds with a dual mechanism of action targeting and directly killing immunosuppressive cells and tumor cells while indirectly activating other T-effectors, leading to strong anti-tumor responses without adding additional safety concern, the company said. Specifically, IO102 and IO103 are first-in-class, immune modulatory vaccines designed to engage and activate IDO and PD-L1 specific human T-cells, IO said in its announcement.

The designation was granted based on data from the MM1636 Phase I/II clinical trial that assessed 30 patients with metastatic melanoma who received IO102, IO103 and an anti-PD-1 therapy. Data showed the combination treatment demonstrated an overall response rate of 79%. Additionally, 45% of patients achieved a complete response (CR), or complete disappearance of their tumors, the company said.

Avelas BioSciences – On Monday, San Diego-based Avelas received Breakthrough Therapy designation for pegloprastide (AVB-620), which is intended to be used for the intraoperative detection and visualization of positive margins during breast cancer surgery. Pegloprastide is designed to deliver a fluorescent marker to cancer cells to aid surgeons in identifying positive margins in real time during surgery. Positive surgical margins occur when post-operative pathology indicates cancer cells are present at or very close to the edge of removed tissue, indicating that there may be residual cancer left behind after an operation, the company said.

Amgen – The FDA awarded Breakthrough Therapy designation to Amgen’s investigational KRAS G12C inhibitor, sotorasib. The drug is in development as a potential treatment for patients locally advanced or metastatic non-small cell lung cancer (NSCLC) with KRAS G12C mutation. KRAS G12C is the most common KRAS mutation in NSCLC, with an estimated 13% of NSCLC patients having this. The designation, along with Real Time Oncology Review, are supported by positive Phase I results in patients with advanced NSCLC from the CodeBreaK 100 clinical study. That study includes patients whose cancer had progressed despite prior treatment with chemotherapy and/or immunotherapy. Data from the study showed treatment with sotorasib provided durable anticancer activity with a positive benefit-risk profile. 

Fortress Biotech – Cyprium Therapeutics, a subsidiary of Fortress Biotech, received Breakthrough Therapy designation for its Menkes disease asset, CUTX-101. Cyprium’s CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate, which is designed to restore copper homeostasis and maintaining serum copper levels in patients with Menkes disease, an X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. The designation was awarded following Cyprium’s announcement of positive topline clinical efficacy data that demonstrated the drug’s safety and efficacy. Cyprium intends to begin a rolling NDA in the first quarter of 2021. The FDA previously granted Orphan Drug, Fast Track, and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease. Additionally, the European Medicines Agency previously granted Orphan Drug Designation to CUTX-101.

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