The Medicines Company and Alnylam Rise on Positive Phase 1 Results

For some background, ALN-PCSsc is an investigational RNAi therapeutic targeting PCSK9 being developed for the treatment of hypercholesterolemia. In contrast to anti-PCSK9 monoclonal antibodies (MAbs) that bind to PCSK9 in blood, ALN-PCSsc is a first-in-class investigational medicine that acts by turning off PCSK9 synthesis in the liver.

Alnylam and The Medicines Company are collaborating in the advancement of ALN-PCSsc per the companies’ agreement formed in early 2013. Under the terms of the agreement, Alnylam will complete certain preclinical studies and a Phase 1 clinical study of ALN-PCSsc and The Medicines Company is responsible for leading and funding development from Phase 2 forward as well as potential commercialization.

Akshay Vaishnaw, M.D., Ph.D., Executive Vice President of R&D and chief medical officer at Alnylam, commented on the release:

Our initial Phase 1 results with ALN-PCSsc, a first-in-class investigational PCSK9 synthesis inhibitor, demonstrate robust, dose-dependent, and durable reductions in LDL-C of up to 83%. Remarkably, significant and clamped lowering of LDL-C is achieved for over 140 days after a single dose. At the 300 mg dose – which we believe is optimal, with fully saturating effects on both LDL-C lowering and PCSK9 knockdown – an injection volume of 1.5 mL and possibly lower can be achieved. Accordingly, we believe that these results support a quarterly, and possibly bi-annual, low volume subcutaneous dose regimen for further development. Importantly, ALN-PCSsc was generally well tolerated with no clinically drug-related significant adverse events to date. Based on these positive results, we believe that ALN-PCSsc potentially represents an innovative, differentiated, and well validated approach for the treatment of hypercholesterolemia. We very much look forward to our continued partnership with The Medicines Company as they now take the lead in developing ALN-PCSsc in the ORION program.

Shares of The Medicines Company closed Friday up 6%, at $33.64 in its 52-week trading range of $19.92 to $34.70. In early trading indications on Monday, shares were up nearly 20% to $40.34. The stock has a consensus analyst price target of $38.00.

Shares of Alnylam closed Friday up 4% to $109.07, within a 52-week trading range of $63.48 to $140.00. In early trading indications, shares were up 6.4% to $116.00. The consensus analyst price target is $145.64.

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