Isis Pharmaceuticals, Inc. ISIS announced an encouraging update from an ongoing open-label extension (OLE) study in children suffering from type II or type III spinal muscular atrophy (SMA), who have completed an open-label, phase II multiple-dose study on ISIS-SMNRx. The company’s shares gained 3.3% following the announcement.
According to an analysis performed on May 15, 2015, ISIS-SMNRx led to continued and durable increase in measures of muscle function. Notably, 57% of the patients achieved an increase of at least three points in the Hammersmith Functional Motor Scale-Expanded (HFMSE) score.
Moreover, measures of muscle-function at 9-month evaluation in the OLE study demonstrated a mean increase in score in all the three scales (HFMSE, six-minute walk test (6MWT) for ambulatory patients and upper limb module (ULM) test for non-ambulatory patients) measuring the clinical endpoints of the study. At the time of issuing the press release, ISIS-SMNRx was well tolerated by patients.
According to the company’s press release, approximately 30,000–35,000 patients in the U.S., Europe and Japan suffer from SMA. However, no therapy has yet received regulatory approval for the treatment of this disease. This hints at the existence of unmet need for new therapies in this market.
We note that Isis Pharma has an agreement with Biogen BIIB for the development and commercialization of ISIS-SMNRx for the treatment of SMA. While Isis is responsible for the worldwide development of the candidate, Biogen has the option to license the compound. Currently, Isis Pharma is evaluating ISIS-SMNRx in two phase III studies – ENDEAR and CHERISH – for the treatment of infants and children suffering from SMA, respectively. Meanwhile, Biogen is operationalizing two phase II studies (NURTURE and EMBRACE) to augment the ongoing phase III program.
In the first quarter of 2015, Isis Pharma received $9 million and $7 million as milestone payments from Biogen for advancing the phase III study on ISIS-SMNRx in infants with SMA and the open-label extension study on ISIS-SMNRx in children with SMA, respectively. Additionally, the company expects to receive the next milestone payment of $8.5 million upon further advancement of the phase III study in infants with SMA.
Given the fast track status for ISIS-SMNRx for the treatment of SMA in the U.S., we expect the candidate’s development process to accelerate. It also enjoys orphan drug status in the U.S. Meanwhile, we expect investor focus to remain on further updates from the company.
Isis Pharma carries a Zacks Rank #3 (Hold). Better-ranked stocks in the health care sector include Gilead Sciences Inc. GILD and Acorda Therapeutics, Inc. ACOR. While Gilead carries a Zacks Rank #1 (Strong Buy), Acorda holds a Zacks Rank #2 (Buy).
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