Baxalta Incorporated BXLT reported positive results from an open-label, dose escalation phase I study for the safety and pharmacokinetic (PK) profile of BAX 930, an experimental recombinant ADAMTS13 to treat patients with severe hereditary thrombotic thrombocytopenic purpura (hTTP). Data were presented at the annual Scientific and Standardization Committee meeting of the International Society on Thrombosis and Haemostatis.
Data included results from fifteen patients with severe hTTP who completed the multi-center study. PK data showed a consistent half-life across the BAX 930 doses and a linear dose response. Moreover, single recombinant ADAMTS13 infusions demonstrated a favorable safety profile in adult and adolescent patients with severe hTTP. No serious adverse events were reported.
Based on the positive phase I study results, Baxalta intends to move the candidate into phase III development. Baxalta believes that as a recombinant ADAMTS13, BAX 930 may provide an alternative treatment option to patients with hTTP. The candidate is expected to offer faster reconstitution and shorter administration times and may be suitable for treatment at home in comparison to plasma infusions, upon approval.
We are encouraged by the company’s progress with the candidate. Per the company’s press release, hTTP is a rare blood clotting disease related to ADAMTS13 deficiency. If not treated immediately, mortality rates can approach 90%.
We note that BAX 930 enjoys orphan drug status both in the U.S. and EU.
Meanwhile, Baxalta is set to merge with Shire plc with shareholder votes scheduled for May 27 and transaction expected to close in early June.
Baxalta is a Zacks Rank #5 (Strong Sell) stock. A couple of better-ranked stocks in the health care sector are Retrophin, Inc. RTRX and ANI Pharmaceuticals, Inc. ANIP, each sporting a Zacks Rank #1 (Strong Buy).
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