Rolling BLA for AMLN's Metreleptin - Analyst Blog

Amylin Pharmaceuticals, Inc. (AMLN) recently announced that it has initiated the filing of a rolling Biologics License Application (BLA) for metreleptin. The company is seeking US Food and Drug Administration (FDA) approval for the use of metreleptin in the treatment of diabetes and/or hypertriglyceridemia in patients with rare forms of lipodystrophy.

According to the company, a few thousand patients are afflicted by this ailment worldwide. Given the severity and rare nature of the disease, Amylin received both orphan drug status as well as fast track designation from the FDA for its candidate.

While the company submitted the clinical and non-clinical sections of the BLA, the company expects to finish submitting the chemistry, manufacturing and controls (CMC) section of the BLA by the end of 2011.

The approval of metreleptin would be a huge boost for investor confidence in the company. Amylin suffered a huge setback in October 2010 when the FDA issued a second CRL for its lead pipeline candidate, Bydureon. The latest CRL, which came a few days ahead of the FDA action date, was a complete surprise. At the time of issuing the first CRL, the FDA had not asked Amylin and its partners, Eli Lilly (LLY) and Alkermes (ALKS) to conduct additional studies. At that time, the FDA had only asked for additional information that was subsequently submitted by the companies.

However, in its latest CRL, the FDA asked the companies to conduct a thorough QT (tQT) study. We believe the FDA may have asked for this study based on recent concerns regarding the cardiovascular safety profile of diabetes drugs. The agency also asked Amylin and its partners to submit data from the DURATION-5 study, which was conducted to compare the safety and efficacy of Bydureon versus Byetta.

Amylin intends to meet with the FDA as early as possible so as to fix the design of the required tQT study. We note that at this point of time, we are looking at a delay of at least 1.5-2 years in the US launch of Bydureon, provided there are no additional stumbling blocks.

Neutral on Amylin

We currently have a Neutral recommendation on Amylin, which is supported by a Zacks #3 Rank (short-term “Hold” rating). Going forward, we expect investor focus to remain on Byetta's performance and updates on the approval process for Bydureon. Longer-term, we are optimistic about the mid-stage obesity pipeline. We view Amylin's deal with Takeda for the obesity pipeline as a major positive.

 
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