Scimitar Equity Blog

Previously reported interim results from the US Phase 2b RESTORE-CLI clinical trial investigating treatment with expanded autologous mixed-cell therapy in patients with critical limb ischemia (CLI) were also provided by Dr. Sharon Watling, Pharm.D, ASTM’s VP of clinical and regulatory at The Sixth International Conference on Cell Therapy for Cardiovascular Disease in NYC, NY; 

ASTM has received initial responses from the FDA regarding the special protocol assessments (SPA) for the 2 Phase 3 studies, which were submitted in 10/10.

• Tim Mayleben, President/CEO of ASTM states in the CLI and DCM release, “We are pleased with the initial responses from the FDA, especially for the no-option CLI study. We look forward to continuing to work with the FDA to finalize both special protocol assessments (SPAs) for the Phase 3 CLI program in the months ahead.”

However, quoting from Jason Kolbert, Managing Director, Healthcare Research, National Securities, 1/14/11 report:

• “The FDA answered ASTM’s 2 CLI SPA’s requests and is un-yielding on AFS as a primary endpoint and is emphatic that all analysis shall be based on intent to treat (ITT) which will begin when patients are enrolled in study (regardless of if they receive ASTM’s TRC product);
• The FDA says NO to MALEFS re ASTM’s request for a MALEFS endpoint and instead insists on AFS;
• Jason Kolbert views this as negative in HIS most recent analysis;
• ASTM’s TRC’s failed to show significance on an AFS endpoint in the last (PIIb) interim trial update;
• MALEFS was requested for the 2nd pivotal trial in which is designed to focus on slightly less severe patients (“poor options”) patients;
• ITT begins when ASTM start Enrolling Patients: it is disappointing to learn that the FDA will not accept a modified MITT score;
• All patients once enrolled in the study will be counted;
• In an autologous therapy there are several weeks of processing. This means that severely ill CLI patients may worsen sharply and never receive TRC cells, or by the time they receive them, they have deteriorated substantially and would not meet the original enrollment criteria;
• There are even a few instances of patients for whom for one reason or another, ASTM may not be able to produce a TRC treatment;
• These patients will all count as failures;
• As a negative, this skews going into an already complex trial;

Our (HWM and JK) question continues … Is there really an advantage to autologous therapy and the wait time for ASTM’s TRC autologous processing, manufacturing variability and cell source (age) issues.

These are muddy waters and skepticism abounds!

• So, can ASTM initiate the Phase 3 trial by mid 2011, maybe a no-option trial but …?
• ASTM is trading down $0.28 (9.62%) at $2.63 from 1/19/11 previous close of $2.91 which was down from $3.04 on 1/18/11.
• Disclosure: Scimitar Equity terminated coverage on ASTM on 12/29/10;
• National Securities had lowered its rating to a HOLD.