Alnylam Pharmaceuticals, Inc.NASDAQ
More Data From Alnylam Pharmaceuticals, But Not Much More Clarity
Stephen Simpson, CFA • 12 Comments
Stephen Simpson, CFA • 12 Comments
After Multiple Updates, Alnylam Hasn't Cleared Up Much
Stephen Simpson, CFA
Stephen Simpson, CFA
Mon, Oct. 10, 8:09 AM
- Alnylam Pharmaceuticals (NASDAQ:ALNY) announces that the Data Monitoring Committee (DMC) for its Phase 3 APOLLO study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN) met on October 7 and recommended continuation of the trial without modification. The APOLLO DMC met at the request of the Company following the decision announced on October 5 to discontinue development of revusiran for the treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM). The DMC will continue to meet periodically per their remit to monitor the overall safety of patisiran in APOLLO through its completion. Top-line data are expected in mid-2017.
- ATTR amyloidosis is a progressively debilitating and often fatal disease caused by deposition of transthyretin (TTR) in peripheral tissues. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. In hereditary ATTR amyloidosis (hATTR), mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.
- Shares are up 2% premarket, but only on turnover of 457.
Fri, Oct. 7, 11:57 AM
- Ionis Pharmaceuticals (NASDAQ:IONS) upgraded to Outperform from Market Perform by BMO Capital. Price target raised to $48 (42% upside) from $42.
- Catalyst Pharmaceuticals (NASDAQ:CPRX) upgraded to Overweight from Neutral by Piper Jaffray. Price target raised to $4 (199% upside) from $1.
- AmerisourceBergen (NYSE:ABC) upgraded to Market Outperform from Market Perform by Avondale Partners.
- Alnylam Pharmaceuticals (NASDAQ:ALNY) downgraded to Market Perform from Outperform by Leerink Swann. Price target lowered to $40 (14% upside) from $107. Downgraded to Equal Weight from Overweight by Barclays. Price target lowered to $50 (42% upside) from $85. Downgraded to Neutral from Overweight by JPMorgan. Downgraded to Equal Weight from Overweight by Morgan Stanley. Price target lowered to $36 (3% upside) from $38.
- Merrimack Pharmaceuticals (NASDAQ:MACK) downgraded to Neutral from Overweight with a $5.75 (2% upside) price target by JPMorgan.
- Iradimed (NASDAQ:IRMD) downgraded to Neutral from Buy by Roth Capital. Price target lowered to $11.50 (11% upside) from $28.
- Trinity Biotech (NASDAQ:TRIB) downgraded to Equal Weight from Overweight by Stephens & Co.
- Depomed (NASDAQ:DEPO) downgraded to Neutral from Buy by Mizuho Securities.
- Align Technology (NASDAQ:ALGN) downgraded to Neutral from Overweight with a $96 (8% upside) price target by Baird.
Thu, Oct. 6, 3:32 PM
- Biotech investors are sitting on the sell button today. The iShares Nasdaq Biotechnology ETF (IBB -2.4%) is down on increased volume. Shares have retraced over 5% since the recent close of 300 on September 22.
- Alnylam (ALNY -48%) is leading the rout after it announced that it was dropping development of RNAi candidate revusiran.
- Representative tickers: (AMGN -0.3%)(BIIB -1.3%)(GILD -1.9%)(CELG -1.4%)(BMY)(MRK -0.7%)(PFE -1.2%)(VRTX -3%)(ALXN -2.2%)
Thu, Oct. 6, 12:48 PM
Thu, Oct. 6, 11:00 AM
Thu, Oct. 6, 9:14 AM
Thu, Oct. 6, 6:46 AM
- BMO Capital upgrades Ionis Pharma (NASDAQ:IONS) to Outperform from Market Perform following yesterday's news that Alnylam (NASDAQ:ALNY) had discontinued its Revusiran program after observing an imbalance of deaths in treated patients.
- "We are increasing our TTR-Rx revenues in familial amyloidotic cardiomyopathy (FAC) and wild-type (wt) to reflect no competition, which is partly offset by a lower probability of success (45% from 50%), given similar drug mechanisms. Consequently, our price target increases to $48 from $42. We believe IONS shares could be pressured as investors perceive added risk to TTR-Rx. We recommend buying on weakness."
- In pre-market trading, IONS is -1% and ALNY is -43%.
Wed, Oct. 5, 6:39 PM
- Alnylam (NASDAQ:ALNY) craters 43% after hours on robust volume in response to its announcement that it has decided to terminate development of revusiran, an RNAi therapeutic for the potential treatment of hereditary ATTR amyloidosis (hATTR) with cardiomyopathy, an Orphan Drug designation. The company made its decision yesterday and communicated it to investigators, study sites and regulatory authorities.
- The problem appeared to be an unacceptable safety profile in the Phase 3 ENDEAVOR study following reports of new onset or worsening peripheral neuropathy (nerve damage) in revusiran-treated patients in the Phase 2 OLE trial. At the company's request, the Data Monitoring Committee met to review the reports and unblinded data from ENDEAVOR. Although, it did not find conclusive evidence of revusiran-related neuropathy it concluded that continued dosing was inappropriate due to an unfavorable benefit-risk profile, specifically an "imbalance" in mortality in the revusiran arm compared to placebo.
- The company says its decision does not affect Phase 3-stage patisiran, a candidate for the treatment of hATTR with polyneuropathy, or any other RNAi therapeutic program. It adds that an assessment of safety data across its other programs, including the ALN-PCSsc program with The Medicines Company (NASDAQ:MDCO), revealed no evidence of a drug-related neuropathy signal in over 800 patients with exposures up to 34 months. Investors appear unconvinced. MDCO is down 12% after hours on robust volume.
Wed, Oct. 5, 5:35 PM
Thu, Aug. 4, 4:02 PM
Wed, Aug. 3, 5:35 PM
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Thu, Jul. 28, 11:32 AM
- Long-term follow-up data from a 16-subject Phase 1/2 clinical trial assessing Roche's (OTCQX:RHHBY) investigational biologic emicizumab in patients with severe hemophilia A show encouraging safety and prophylactic efficacy regardless of the presence of factor VIII inhibitors. The results were presented at the World Federation of Hemophilia 2016 World Congress in Orlando, FL.
- The data show patients who received once-weekly subcutaneous injections of either 0.3,1.0 or 3.0 mg/kg/week of emicizumab with median follow-up periods of 32.6 months, 27.0 months and 21.4 months, respectively, experienced a sustained decrease of 95% in annualized bleeding rate (ABR), irrespective of their inhibitor status and prior treatment regimen. The ABRs for the three cohorts were 1.4, 0.2 and 0.0, respectively.
- The product candidate's safety profile was consistent with what was previously reported in the Phase 1 study. No thromboembolic (blood clot that breaks loose and clogs another vessel) adverse events or clinically significant laboratory abnormalities were observed.
- Emicizumab is an bispecific antibody engineered to bind to both factors IXa and X, replacing the function of the missing factor VIII, thereby improving clotting function and preventing spontaneous bleeding. It was created by Chugai Pharmaceutical Co. and is being co-developed by Roche. Future clinical trials will explore less frequent dosing schedules.
- Related tickers: (OTC:BIOVF)(NASDAQ:BIIB)(BXLT)(OTCPK:BAYRY)(NASDAQ:OPK)(NYSE:SNY)(NASDAQ:ALNY)(NASDAQ:SGMO)(NASDAQ:CBIO)(NASDAQ:BMRN)
Mon, Jul. 11, 5:37 PM
Thu, Jun. 2, 7:54 AM
- The Medicines Company (NASDAQ:MDCO) completes the enrollment of 501 subjects in its Phase 2 study, called ORION-1, assessing its RNA interference therapeutic targeting PCSK9 (PCSK9si) in patients with high cardiovascular risk and elevated LDL-C ("bad cholesterol") despite receiving the maximum tolerated doses of LDL-C-lowering therapies. The study will compare the effect of different doses of PCSK9si and will evaluate the potential for quarterly or biannual dosing. The primary endpoint is the change in LDL-C from baseline to day 180.
- Interim three-month and six-month data should be available by year end. If all goes well, Phase 3 studies will commence in early 2017.
- PCSK9si is an investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9, a protein regulator of LDL receptor metabolism. In contrast to PCSK9 inhibitors, PCSK9si works by turning off PCSK9 synthesis in the liver. The company is collaborating with Alnylam (NASDAQ:ALNY) on its development.
- Related tickers: (NASDAQ:AMGN)(NASDAQ:REGN)(NYSE:SNY)
Thu, May 26, 5:09 PM
- The FDA approves CSL Behring's AFSTYLA [Antihemophilic Factor (Recombinant) Single Chain], its long-acting recombinant factor VIII single-chain therapy for the treatment of adults and children with hemophilia A.
- AFSTYLA is indicated for routine prophylaxis to reduce the frequency of bleeding episodes with dosing of two-to-three times per week, on-demand treatment and control of bleeding episodes and the perioperative management of bleeding.
- Market launch will commence this summer.
- Hemophilia A-related tickers: (NYSE:BXLT)(OTCPK:BAYRY)(NASDAQ:BMRN)(NYSE:OPK)(NYSE:SNY)(NASDAQ:ALNY)(NASDAQ:BIIB)(NASDAQ:SGMO)(OTCQX:RHHBY)(NASDAQ:CBIO)
Thu, May 26, 12:29 PM
- Ionis Pharmaceuticals' (IONS -38%) hiccup related to its antisense drug candidate for familial amyloidotic polyneuropathy (FAP), IONIS-TTRrx, stokes buying in RNA interference (RNAi) therapeutics developer Alnylam Pharmaceuticals (ALNY +13%) who is developing its own FAP candidate called patisiran which recently showed positive results in a Phase 2 study.
- Previously: Alnylam's patisiran shows treatment benefit in mid-stage study in rare inherited disorder (April 20)
- Previously: Glaxo declines to proceed with late-stage study of Ionis Pharma's IONIS-TTRrx; Ionis down 7% premarket (May 26)