Monday November 12, 2018 0 comments
ENGLEWOOD -- Ampio Pharmaceuticals, Inc. (NYSE MKT: AMPE) is providing an update of the regulatory and clinical path activities required to file a Biologics License Application (BLA) for Ampion for the treatment of severe osteoarthritis-of-the-knee (OAK).
According to Mike Macaluso, Ampio CEO, Ampio designed what it thought would be a final pivotal clinical trial of Ampion (AP-003-C), predominately under guidance from FDA CBER's Office of Blood Research and Review (OBRR).
However, a reorganization of CBER occurred, and the regulatory guidance for AP-003-C was transferred to the Office of Tissue and Advanced Therapies (OTAT). As previously disclosed in August 2018, OTAT expressed concerns over the trial design and advised the Company to execute an additional clinical trial designed under a Special Protocol Assessment (SPA).
Macaluso said Ampio is moving ahead with a new clinical trial design under a SPA but disagreed with the OTAT's decision regarding the insufficiency of AP-003-C and formally requested FDA to reconsider.
The FDA has now accepted a Type C meeting proposal from Ampio that could lead to the resolution of the company’s dispute regarding the acceptability of AP-003-C as the 2nd of the two required Pivotal trials for the filing of a BLA and scheduled the meeting before the end of November 2018. Ampio said it will promptly report any further news after it is received.
Regarding a clinical update, Ampio said the primary objective of the Open-Label Extension (OLE) study, which followed the completion of the AP-003-C study, was to investigate the safety and tolerability of patients with severe osteoarthritis of the knee (KL4) receiving up to 5 injections of Ampion administered every 12 weeks, but efficacy was tracked and reported.
- 94 subjects entered the OLE study; 8 (9%) subjects discontinued.
- Subjects received an average of 4 injections with a total exposure of 48 weeks. The longest exposure was 59 weeks.
- No serious adverse events were reported.
- Three of the 41 AEs were possibly related to the procedure (not Ampion), and all the remaining 38 AEs were unrelated. Two patients experienced post-injection knee pain.
Assessments of WOMAC A (pain) and C (function) as well as OMERACT-OARSI responder status were reported as the average percent improvement from baseline (%) following repeat injections of Ampion (spaced 12 weeks apart):
- Improvements in pain: 39%, 34%, 38%, 42%, and 47%, respectively for 1, 2, 3, 4, and up to 5 injections of Ampion.
- Functional improvements: 38%, 32%, 35%, 44%, and 47% respectively for 1, 2, 3, 4, and up to 5 injections of Ampion.
- Responder status: 70%, 68%, 71%, 75%, and 75% respectively for 1, 2, 3, 4, and up to 5 injections of Ampion.
"Severe OAK Patients (KL-4) whose therapeutic options are limited (strong opioids or total knee replacement) reported a consistent/persistent, beneficial effect from each repeat injection of Ampion,” said Macaluso.
The OLE study of the AP-003-C pivotal trial shows each subsequent injection of Ampion provided a sustained, significant reduction in pain, improvement in function and 70% responder rates from patients with severe OA disease.
Ampio said it believes Ampion, a low molecular weight fraction of human serum albumin with anti-inflammatory properties, will be identified as a "reference product" upon FDA approval of their BLA.
Reference products are granted 12 years of exclusivity under the PHS Act, 42 U.S.C. § 262(k)(7). Specifically, FDA is not permitted to approve an application for a biosimilar or interchangeable product until 12 years after the date of the first licensure of the reference product.
The existing Ampion portfolio has patent coverage in all major jurisdictions throughout the world (U.S., Europe, Australia, Brazil, Canada, China, Eurasia, Hong Kong, India, Indonesia, Israel, Japan, Korea, Mexico, Malaysia, New Zealand, Philippines, Singapore, South Africa) for pharmaceutical compositions and methods of treating a range of conditions.
The portfolio includes 125 issued patents and 85 pending applications throughout seven primary patent families having expiration dates that extend to 2035.