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Alnylam Provides Pipeline Update, Completes Phase I Trial of Liver Cancer Drug

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By Doug Macron

Alnylam Pharmaceuticals this week provided an update on its drug-development pipeline, noting that it remains on track to report phase I data from two programs and unveil two additional clinical candidates before year's end.

The company also reported its second-quarter financial results and disclosed that it has finished a phase I study of the systemically delivered liver cancer treatment ALN-VSP, formerly ALN-VSP02.

In January, Alnylam launched its so-called 5x15 strategy, which calls for the company to have five products in advanced clinical development by 2015 (GSN 1/13/2011).

Leading the pack is the phase I transthyretin-mediated amyloidosis therapy ALN-TTR01. The disease is caused by a mutation in the transthyretin gene, and the drug comprises siRNAs designed to inhibit both the wild-type and mutant forms of the gene.

The company continues to enroll patients in the phase I trial, and while it had previously expected to report data in the third quarter, it has pushed back that timing to the fourth quarter to “accommodate for patient enrollment at … European sites over the summer months,” Alnylam CMO Akshay Vaishnaw said during a conference call held to discuss the financial results.

Specifically, Alnylam will present the ALN-TTR01 data this November in Japan at the International Symposium on Familial Amyloidotic Polyneuropathy.

“We think this is the best venue for us to present what we expect to be important data and results,” Vaishnaw said.

Meantime, Alnylam continues to advance another version of the drug, called ALN-TTR02, which incorporates a second-generation lipid nanoparticle delivery technology the company developed in-house. It said it plans to file an investigational new drug application for the agent by the end of 2011.

Alnylam CEO John Maraganore noted during the call that the new nanoparticles offer “well over 10-fold improvements in potency” and, as a result, a “broader therapeutic index.” However, the company hasn't yet decided which of the TTR drugs will ultimately move into phase II testing.

“As we advance these two programs … we will be picking one as a go-forward program,” he said. “They’re really quite closer together in terms of their timeline development,” and the goal is to move one into the second stage of clinical evaluation in 2012.

Also on deck under the 5x15 initiative is the hypercholesterolemia treatment ALN-PCS, which Alnylam recently filed with UK regulators for phase I testing (GSN 7/14/2011), and the refractory anemia treatment ALN-HPN, for which the firm expects to file an IND in 2012.

According to Vaishnaw, Alnylam will unveil the two other remaining 5x15 programs in the third and fourth quarters of this year, respectively.

Outside of the 5x15 plan is ALN-VSP, which Alnylam confirmed this week it would only move into phase II testing once it finds a partner.

Maraganore said that “some discussions” regarding a potential partnership are “ongoing,” noting that before initiating negotiations, Alnylam was waiting until data was first presented earlier this summer at the American Society for Clinical Oncology's annual meeting in Chicago.

There, Alnylam reported that ALN-VSP is “generally” well tolerated, although there were a number of serious adverse events among trial participants that were “possibly related” to the drug (GSN 6/9/2011). Importantly, one patient with endometrial cancer and multiple liver lesions experienced a roughly 70-percent reduction in tumor burden.

Maraganore indicated that partnership talks were proceeding well, noting that the company was “swamped with interest [at ASCO] from both pharma as well as physicians.”

The Second Quarter

For the three months ended June 30, Alnylam net loss narrowed to $13.8 million, or $0.33 per share, from $14.6 million, or $0.35 per share, year over year.

Revenues in the quarter dropped to $20.6 million from $26.6 million in the same period last year, while research and development spending slipped to $25.3 million from $28.1 million year over year.

General and administrative costs fell to $8.4 million from $10.1 million, while the company reported a $1 million loss of equity related to expenses incurred by Regulus Therapeutics, its microRNA drugs joint venture with Isis Pharmaceuticals.

Alnylam lowered its 2011 cash guidance, saying it would have more than $250 million in the bank by Dec. 31, compared to its previous guidance of $275 million. The company attributed this to investments in its pipeline related to the 5x15 effort.


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