Aeterna Zentaris Inc. AEZS announced its plans to conduct two studies on its adult growth hormone deficiency (AGHD) candidate, Macrilen, following its meeting with the FDA regarding the new drug application (NDA) for the candidate.
The company intends to conduct a confirmatory phase III study to assess the efficacy of Macrilen for the treatment of AGHD and a dedicated thorough QT study to evaluate the effect of Macrilen on myocardial repolarization.
The confirmatory study will be conducted as a two-way crossover with the insulin tolerance test as the benchmark comparator on patients with a medical history of risks related to AGHD. Aeterna Zentaris will submit a proposed final protocol to the FDA before initiating the study.
We remind investors that in Nov 2014, Aeterna Zentaris received a complete response letter from the FDA for its NDA for Macrilen. The letter stated the FDA’s dissent to approve the candidate due to inability of the pivotal study on Macrilen to meet the primary efficacy objective as set down by the Special Protocol Assessment agreement between the company and the agency as well as lack of complete and verifiable data on the accurate diagnosis of the patients with AGHD.
Based on the recommendation by a panel of U.S. and EU endocrinology experts to continue seeking approval for Macrilen, the company has kept on evaluating the available data on Macrilen. Early last month, in an end-of-review meeting, the FDA agreed with the company on the general design and evaluation criteria for the confirmatory study on Macrilen.
Aeterna Zentaris believes that additional studies on Macrilen will confirm the efficacy and safety of the candidate. The confirmatory phase III study and the QT study will require a combined expenditure in the range of $5 million – $6 million and both are expected to be completed within 18 months from the date of commencement. The company is planning to design the confirmatory study in such a way that it may meet registration requirements of the FDA as well as the European Medicines Agency.
We note that Macrilen is one of the two principal candidates at Aeterna Zentaris. The other candidate, zoptarelin doxorubicin, is being evaluated in a phase III ZoptEC study for endometrial cancer. We expect investor focus to remain on pipeline updates from the company.
Aeterna Zentaris carries a Zacks Rank #1 (Strong Buy). Other well-ranked stocks in the health care sector are ANI Pharmaceuticals, Inc. ANIP, Horizon Pharma plc HZNP and Biogen Inc. BIIB. All these stocks carry the same Zacks Rank as Aeterna Zentaris.
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