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RNAi Therapeutics Developer Reports Positive Amyloidosis Data
Research Report

Source:

Michael King, an analyst with JMP Securities, reported the new results that this biotech presented in March.

DNA

A March 28, 2018, research note by JMP Securities analyst Michael King indicated that at the recent International Symposium on Amyloidosis in Japan, Alnylam Pharmaceuticals Inc. (ALNY:NASDAQ) reported multiple sets of results of clinical trials, separately evaluating its investigational RNA interference (RNAi) therapeutics patisiran and ALN-TTRsc02. Patisiran and the subcutaneously administered form ALN-TTRsc02 are being developed for the treatment of hereditary ATTR amyloidosis.

With respect to patisiran, the company presented data from its APOLLO Phase 3 on a cardiac subpopulation composed of 56% of the study participants, or 126 individuals. The results "demonstrated improvements in measures of cardiomyopathy," specifically cardiac structure, cardiac function, gait speed and NT-proBNP level, King noted. Higher levels of this stress biomarker, NT-proBNP, are "associated with increased mortality in cardiac amyloidosis."

Data also showed a safety profile of patisiran that King described as "favorable," in that patients in the therapeutic and placebo arms reported similar adverse cardiac and other events.

Alnylam also delivered preliminary data from the global OLE study of patisiran, evaluating its long-term efficacy and safety in qualifying patients from the Phase 2 OLE and Phase 3 APOLLO studies.

Of the patients from the Phase 2 OLE study, 25 who received patisiran for four years displayed "sustained mean improvements in neuropathy" at three years, King relayed. Of the individuals from the APOLLO study, 52 of them showed "sustained improvement in neurologic impairment" at one year. Further, patisiran's safety profile was consistent with that established in APOLLO, with reactions at the infusion site being the most common adverse effect.

Alnylam also shared results of an analysis of data from the Phase 2 OLE and Phase 3 APOLLO trials. It revealed a "strong correlation between the degree of transthyretin (TTR) knockdown and change in mNIS+7," King pointed out, in that a higher degree of the former led to greater improvement in the latter. Also, results indicated that more than 80% TTR knockdown was linked to mNIS+7 levels moving into the negative. Negative mNIS+7 values signify patients are improving, progression of their disease is being reversed.

As for ALN-TTRsc02, a Phase 1, "ascending fixed dose (5300 milligrams) study" involving 80 healthy volunteers demonstrated "compelling" efficacy and safety data, reported King. Alnylam plans to launch pivotal Phase 3 studies of ALN-TTRsc02 late this year.

Single doses of ALN-TTRsc02 resulted in a mean maximum TTR knockdown of up to 97%, and it was sustained over 320 days. King highlighted that this finding supports the "potential for ALN-TTRsc02 to achieve levels of TTR knockdown comparable to those demonstrated by patisiran in the Phase 3 APOLLO study, but with a low dose (25 milligram), once-quarterly subcutaneous dosing regimen."

ALN-TTRsc02 showed a positive safety profile. Participants did not report any serious adverse or other events or drop out of the study because of adverse events. The adverse events that 77% of patients reported were mild.

These and previously reported data and the demonstrated positive effects of patisiran and ALN-TTRsc02 bolster "our belief that the company will become a dominant player in amyloidosis treatment," King concluded.

JMP Securities has an Outperform rating and a $207 per share target price on Alnylam, whose stock is currently trading at around $98.50 per share. "Alnylam has clearly made the transition from a platform story to one increasingly dominated by clinical results and progress," wrote King, evidenced by the advancements to date of its clinical pipeline.

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JMP Securities, Alnylam Pharmaceuticals Inc., Company Update, Mar. 28, 2018

Analyst Certification: The research analyst(s) who prepared this report does/do hereby certify that the views presented in this report are in accordance with my/our personal views on the securities and issuers discussed in this report. As mandated by SEC Regulation AC no part of my/our compensation was, is or will be directly or indirectly related to the specific views or recommendations expressed herein. This certification is made under the obligations set forth in SEC Regulation AC. Any other person or entity may not use it for any other purpose. This certification is made based on my/our analysis on the date of this report's publication. I/We assume no obligation to update this certification to reflect any facts, circumstances or events that may subsequently come to my/our attention. Signed Michael G. King and Michael Englander.

JMP Securities Disclosures:
JMP Securities currently makes a market in the security of Alnylam Pharmaceuticals, Inc.

JMP Securities was manager or co-manager of a public offering of securities for Alnylam Pharmaceuticals, Inc. (ALNY) in the past 12 months, and received compensation for doing so.

JMP Securities expects to receive OR intends to seek compensation for investment banking services from Alnylam Pharmaceuticals, Inc. in the next 3 months.

JMP Disclaimer:
JMP Securities LLC (the "Firm") compensates research analysts, like other Firm employees, based on the Firm's profitability, which includes revenues from the Firm's institutional sales, trading, and investment banking departments as well as on the quality of the services and activities performed that are intended to benefit the Firm's institutional clients.

JMP Securities LLC, its affiliates, JMP Group LLC, Harvest Capital Strategies LLC, and their respective partners, directors, officers, and associates may have a long or short position in, may act as a market maker for, or may purchase or sell a position in the securities mentioned herein. JMP Securities LLC or its affiliates may be performing, have performed, or seek to perform investment banking, advisory, or other services and may have acted as manager or co-manager for a public offering of securities for any company mentioned herein. The reader should assume that JMP Securities LLC will solicit business from the company covered in this report.




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