Biotech Values

[DewDiligence]

MDCO/ALNY—RNAi-based cholesterol drug shows durability:

http://www.reuters.com/article/2015/08/30/health-heart-alnylam-pharms-medicines-idUSL5N1133V420150830

A new drug in early development cuts cholesterol as much as two potent injections [SNY/REGN’s Praluent and AMGN’s Repatha] recently cleared for sale but lasts much longer, meaning it may need to be given only every three to six months, initial findings suggest.

Results with Alnylam Pharmaceuticals and Medicines Co's experimental product ALN-PCSsc highlight the competition for a market tipped to generate billions of dollars in sales. Unlike rival antibody-based PCSK9 medicines that work in the blood, the new under-the-skin injection is a first-in-class treatment that stops synthesis of the protein which is linked to "bad" LDL cholesterol in the liver.

Alnylam and Medicines Co's product will be relatively late to the party, as it is unlikely to go on sale before 2020 even if subsequent late-stage studies are successful, but its reduced injection frequency could make it an attractive option.

Results from 69 patients in a Phase I trial, reported at the European Society of Cardiology (ESC) congress on Sunday, found a single dose of 300 mg reduced LDL by just over 50 percent at 12 weeks, similar to the 50-60 percent seen with antibody drugs, and the reduction was still 44 percent after 140 days.

"We're not quite sure exactly how long this stuff lasts but we know it is more than 140 days for many patients," Medicines Co Chief Executive Clive Meanwell told Reuters. "The ability to control LDL cholesterol with an injection every three to six months will mean patients can get their cholesterol checked while doctors decide if they need another shot, which should improve adherence."

ALN-PCSsc was generally well tolerated with no significant adverse events, though bigger safety and efficacy studies are needed. Medicines Co plans to start a Phase II study by end-2015 and a final-stage Phase III trial by end-2017.

…Jefferies analysts currently model peak ALN-PCSsc sales of $980 million, after applying a 50 percent risk discount because the drug is still at an early stage.

Shares in Medicines Co and Alnylam rose 6 and 4 percent respectively on Friday ahead of the ESC meeting.

MDCO licensed the worldwide rights to ALN-PCSsc from ALNY in Feb 2013 for a paltry $25M up-front, $180M in milestone payments, and double-digit royalties on sales (#msg-84151150). At the time, I thought it was a decent deal for ALNY, but now (given the success of the PCSK9 drugs), it’s pretty clear that MDCO came out ahead.