Mon, Aug. 31, 9:16 AM
Mon, Aug. 31, 7:53 AM
- The Medicines Company (NASDAQ:MDCO) jumps 20% premarket on average volume in response to development partner Alnylam's (NASDAQ:ALNY) announcement that its investigational RNAi therapeutic, ALN-PCSsc, lowered LDL-C (bad cholesterol) up to 83% with a mean maximum reduction of up to 64% (+-5%) in an early stage study, results comparable to Amgen's (NASDAQ:AMGN) Repatha (evolocumab) and Sanofi (NYSE:SNY) and Regeneron's (NASDAQ:REGN) Praluent (alirocumab). The data were presented at the ESC Congress in London.
- What's notable in this case is the difference in dosing regimens. ALN-PCSsc was administered in one subcutaneous dose that was effective for over 140 days, giving it the potential for once per quarter or twice per year administration. Praluent is dosed once every two weeks and Repatha once every two weeks or once per month at a higher dose.
- ALN-PCSsc turns off PCSK9 synthesis in the liver. This is a different mechanism of action compared to Praluent and Repatha, both of which bind to PCSK9 in the blood.
- The Medicines Company will take the lead in developing ALN-PCSsc under the ORION Program. A Phase 2 study will commence by the end of the year and a Phase 3 trial is planned for 2017. The clinical development will include comparisons to the anti-PCSK9 monoclonal antibodies.
- The companies will host a conference call this morning at 9:30 am ET to discuss the results and their development plan.
Wed, Aug. 26, 6:57 PM
- Biotech analysts at Piper Jaffray say they are buyers on weakness of four biotech names - Vertex Pharma (NASDAQ:VRTX), Novavax (NASDAQ:NVAX), Alnylam Pharma (NASDAQ:ALNY) and Arrowhead Research (NASDAQ:ARWR) - that boast strong enough balance sheets to make it through upcoming value-driving events.
- The firm notes VRTX holds more than $1B in cash, and expects a dramatic increase in treatable cystic fibrosis patients with the newly approved Orkambi, which will help drive revenue growth and a return to near-term profitability.
- Jaffray says it has increased confidence in NVAX following positive Phase 2 data in its RSV-F in elderly subjects; the company ended the quarter with $315M in cash, which can fund "at least one" pivotal RSV trial.
- ALNY ended the recent quarter with $1.4B in cash, with key patisiran and revusiran Phase II in OLE updates expected in Q4.
- On ARWR, the firm looks to an expected September update of Phase IIa HEPARC 2001 data.
Thu, Aug. 6, 4:01 PM
Wed, Aug. 5, 5:35 PM
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Mon, Jul. 27, 11:05 AM
- Alnylam (ALNY -3.8%) commences a Phase 1/2 study assessing its RNAi therapeutic targeting alpha-1 antitrypsin, ALN-AAT, for the treatment of AAT deficiency-associated liver disease (alpha-1 liver disease). Initial data are expected in early 2016.
- The primary endpoint is safety as measured by the number of adverse events through days 70, 154 and 224. Secondary endpoints are the pharmacokinetic profile up to days 21, 105 and 161 and the effect of ALN-AAT on serum AAT protein levels through days 70, 154 and 224. The estimated study completion date is April 2017.
- ALN-AAT, administered via subcutaneous injection, utilizes the company's ESC-GalNAc-siRNA conjugate delivery technology.
- Alpha-1 antitrypsin (AAT) deficiency is an autosomal disorder that causes disease in the lungs and liver. Most (95%) people with AAT-deficiency carry two copies (homozygous) of an abnormal allele called PiZZ which expresses a protein called Z-AAT. The misfolding of mutant Z-AAT interferes with its normal release into the bloodstream causing it to accumulate in liver cells, leading to liver injury, fibrosis, cirrhosis and liver cancer. There are ~120K people in the U.S. and Europe with the PiZZ mutation. About 12K have the associated liver pathology. The only treatment options currently available for AAT-deficient patients is supportive care and, for those with advanced cirrhosis, liver transplantation.
Mon, Jul. 20, 10:07 AM
- Alnylam Pharmaceuticals (ALNY -0.9%) commences the Phase 3 open-label extension study of its RNAi therapeutic targeting transthyretin (TTR), patisiran, for the treatment of TTR-mediated amyloidosis in patients with Familial Amyloidotic Polyneuropathy (FAP). All patients who complete the randomized, double-blind Phase 3 study, APOLLO, will be eligible to enroll in the extension phase, called APOLLO-OLE.
- The primary endpoint of APOLLO is the change from baseline in modified Neuropathy Impairment Score (mNIS+7) over 18 months. The estimated study completion date is May 2017.
- FAP is an inherited disorder caused by mutations in the TTR gene. Sufferers produce variant TTR protein that results in the buildup of TTR amyloid deposits in the tissues, mainly in the nerves and heart. This eventually leads to organ damage and clinical symptoms similar to those of senile systemic amyloidosis.
Fri, Jun. 19, 5:36 PM
Fri, Jun. 12, 9:55 AM
- Initial results from an ongoing Phase 1/2 study evaluating Alnylam Pharmaceuticals' (NASDAQ:ALNY) ALN-CC5 for complement-mediated diseases showed that a single subcutaneous dose of ALN-CC5 in 12 healthy volunteers resulted in a knockdown of serum C5 of up to 96% and achieved inhibition of serum complement activity of up to 92%. The data were presented at the 20th Congress of the European Hematology Association in Vienna, Austria.
- ALN-CC5 is a first-in-class C5 synthesis inhibitor under development for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). Phase 2 enrollment is expected to commence by the end of the year.
- PNH is a rare, chronic debilitating disorder characterized by the breakdown of red blood cells by the complement system with releases of hemoglobin in the urine. Sufferers have dark-colored urine in the morning. It is caused by a defect in the formation of surface proteins on red blood cells. The only approved drug to treat the disorder is Alexion Pharmaceuticals' (ALXN +1.1%) blockbuster Soliris (eculizumab) which generated $2.2B in sales in 2014. It is the world's second most expensive drug (after uniQure's Glybera).
- C5 (fifth component of complement) plays a key role in inflammation and cell killing processes. The complement system is part of the innate immune system that helps (complements) it to clear pathogens.
- Under its 2014 global alliance with Sanofi's (SNY -2.6%) Genzyme, Alnylam retains the rights to ALN-CC5 in North America and Western Europe while Genzyme owns the rights in the rest of the world. Genzyme, under certain circumstances, can opt-in to co-develop and co-commercialize product candidates which could include global product rights.
Thu, Jun. 11, 8:10 AM
- Responding to Alnylam's (NASDAQ:ALNY) lawsuit accusing it of misappropriating trade secrets, Dicerna Pharmaceuticals (NASDAQ:DRNA) says the case is without merit and it will vigorously defend itself in the matter.
- The company says that earlier this year Alnylam was concerned about certain aspects of Dicerna's GalNAc delivery technologies. Dicerna conducted an internal investigation and confirmed that no Merck/Alnylam confidential information had been used, a finding that it communicated to Alnylam. It also says that many of the insinuations in Alnylam's complaint are either false or based on unfounded speculation.
- Previously: Alnylam sues Dicerna Pharma over trade secrets (June 10)
Wed, Jun. 10, 4:35 PM
- Alnylam Pharmaceuticals (NASDAQ:ALNY) files a lawsuit in Massachusetts against Dicerna Pharmaceuticals (NASDAQ:DRNA) accusing it of misappropriating trade secrets Alnylam acquired via its buyout of Sirna Therapeutics from Merck in January 2014.
- The suit accuses Dicerna of obtaining its trade secrets by hiring six scientists from Merck who were involved in RNAi work, despite each of them signing a confidentiality agreement. It alleges that Dicerna's subsequent product candidates and delivery technologies are "strikingly similar" to Sirna's/Merck's. It also alleges that certain scientists hired by Dicerna were seen taking material out of the Merck facility prior to their departure.
Mon, Jun. 8, 7:21 AM
Wed, May 20, 7:46 AM
- The FDA designates Alnylam's (NASDAQ:ALNY) revusiran an Orphan Drug for the treatment of transthyretin-mediated amyloidosis, a genetic disorder characterized by the buildup of proteins called amyloid in the body's organs and tissues. It is caused by mutations in the TTR gene which provides instructions for producing transthyretin, a protein that transports vitamin A and thyroxine throughout the body. The buildup of beta amyloid in the brain is associated with Alzheimer's disease, a well-known example of the condition.
- Revusiran, an RNAi therapeutic that employs small interfering RNA (siRNA) targeting wild-type and all mutant forms of TTR, utilizes the company's proprietary GalNAc-siRNA conjugate delivery platform which enables subcutaneous (injection under the surface of the skin) delivery of the therapeutic.
- Among the benefits of Orphan Drug status is a seven-year period of market exclusivity for the indication, if approved.
Fri, May 15, 5:38 PM
Fri, May 8, 10:18 AM
- Alnylam Pharmaceuticals (ALNY +2.6%) Q1 results: Revenues: $18.5M (+122.9%); R&D Expense: $58M (+32.4%); SG&A: $12.7M (+42.7%); Operating Loss: ($52.2M) (+80.6%); Net Loss: ($50.8M) (+79.8%); Loss Per Share: ($0.62) (+83.2%); Quick Assets: $1,450.8M (+64.5%).
- 2015 Guidance: Cash, Cash Equivalents and Total Marketable Securities: > $1.2B.
Thu, May 7, 4:01 PM
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