Genentech, a member of the Roche Group (RHHBY), announced interim clinical data from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam in spinal muscular atrophy. In the FIREFISH study in Type 1 SMA, six out of 14 infants were able to sit, including three who achieved unassisted stable sitting after eight months of treatment. In addition, four infants demonstrated rolling to the side; seven kicking and six achieved upright head control. These milestones were assessed according to the Hammersmith Infant Neurological Examination (HINE) Module 2 and are key secondary endpoints in the confirmatory part of FIREFISH. Roche and Genentech are leading the clinical development of risdiplam, an oral SMN2 splicing modifier, as part of a collaboration with the SMA Foundation and PTC Therapeutics (PTCT). Updated analyses of the Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders, a scale developed to assess motor function in infants with Type 1 SMA, demonstrated that eight out of 14 infants in FIREFISH achieved a score of 40 or above at their eight-month visit. Typically, an infant with Type 1 SMA does not demonstrate any motor improvement and can decline during this time period. The median CHOP-INTEND scores increased over time. The median age at first dose in FIREFISH was 6.7 months and median treatment duration was 9.5 months. Nineteen out of 21 infants enrolled remain alive with two having discontinued due to the fatal progression of their disease. Three patients are now over 24 months old. No infant has required a tracheostomy or permanent ventilation since study initiation, and no infant has lost the ability to swallow. The most common adverse events were fever, diarrhea, upper respiratory tract infections, ear infections, pneumonia, constipation, vomiting, cough and upper respiratory tract inflammation. In Part 1 of the SUNFISH study in Type 2 and 3 SMA, SMN protein median increases of greater than two-fold, as measured in blood, were seen after 12 months. A very broad patient population aged between two to 24 years was included, ranging in functional status from weak non-ambulant to strong ambulant, and with varying degrees of scoliosis from none to severe. Twenty-one patients initially received lower doses of risdiplam for at least 12 weeks. Of the patients treated with risdiplam for at least one year, the median change from baseline in Motor Function Measure, the primary endpoint in the confirmatory part of SUNFISH and a scale used to assess motor function in neuromuscular diseases, was a 3.1-point improvement. Sixty-three percent of patients experienced an improvement in MFM over baseline of three points or more after one year. Such improvements were seen both in patients under 12 years old and over 12 years old. When considering patients who experienced any amount of improvement over baseline, the percentages were 70 percent overall, 76 percent for the younger age group, and 62 percent for the older patients. Serious adverse events that occurred in two or more of the 51 patients exposed to risdiplam were nausea, upper respiratory tract infection and vomiting. To date there have been no drug-related safety findings leading to withdrawal from any study.
Wednesday, October 3, 2018
Express Scripts: Diabetes Care Value program delivers savings of $42.6M
Express Scripts released new data showing significantly improved clinical outcomes and reduced drug spending for diabetes. In its inaugural year, the Diabetes Care Value program, part of the technology-supported, value-based SafeGuardRx platform, reduced diabetes drug spending by 19% – $360 per member per year – for the more than 800 plan sponsors enrolled in the program, delivering a total savings of $42.6M in 2017. In addition, Express Scripts diabetes specialist pharmacists, using advanced clinical analytics, improved compliance with recommended treatment guidelines by starting an additional 15% of enrolled patients at risk for heart attack or stroke on statin therapy. If all Express Scripts plans were to similarly increase statin use among their members with diabetes for better cholesterol control, they could prevent an estimated 13,000 heart attacks over the next 10 years.
Trovagene signs license agreement with MIT for prostate cancer therapy
Trovagene has entered into an exclusive patent license agreement with the Massachusetts Institute of Technology, MIT, under which Trovagene has exclusive rights to develop combination therapies that include anti-androgen or androgen antagonist and a Polo-like Kinase inhibitor for the treatment of cancer. The exclusive license agreement is part of the company’s strategy to explore the efficacy of Onvansertib, its oral PLK1 inhibitor, in combination with anti-androgen drugs in cancers including prostate, breast, pancreatic, lung and gastrointestinal. In this Phase 2 trial, Onvansertib in combination with the standard dose of Zytiga and prednisone is being evaluated for safety and efficacy. The trial will enroll up to 45 patients with mCRPC showing early signs of disease progression. The primary efficacy endpoint is the proportion of patients achieving disease control after 12 weeks of study treatment.
JMP eyes continued dystrophin expression in Sarepta gene therapy news
JMP Securities analyst Liisa Bayko noted that Sarepta is scheduled to give an update on its Phase 1/2a study of a micro-dystrophin gene therapy at the World Muscle Society meeting and see hopes to see about 40% micro-dystrophin expression in the fourth patient and at least one vector copy per nucleus, which would be in line with the first three patients. She is also looking for details of the functional benefits that were previously acknowledged, said Bayko, who keeps an Outperform rating and $270 price target on Sarepta shares.
LabCorp price target lowered to $193 from $201 at Craig-Hallum
LabCorp price target lowered to $193 from $201 at Craig-Hallum. Craig-Hallum analyst Kevin lowered his price target for LabCorp to $193 from $201 after updating his estimates for the potential impact from Hurricane Florence and other items likely to affect the second half of 2018, such as the impact from the IT breach, PAMA, ASC 606 and the divestiture of the Covance Food Solutions business. While LabCorp has some cost levers it will likely pull and its free cash flow remains strong, he contends. The analyst reiterates a Buy rating on the shares.
VistaGen Therapeutics gets FDA fast track for non-opioid pain med
VistaGen Therapeutics announced that the FDA has granted fast track designation for development of AV-101 as a non-opioid, non-sedating treatment for neuropathic pain. This is the second fast track designation granted to VistaGen since December 2017. VistaGen’s receipt of the designation for the development of AV-101 for neuropathic pain comes on the heels of the recent official statement made by FDA Commissioner Scott Gottlieb, stating that the FDA plans to issue guidance documents to assist sponsors with the development of new non-opioid pain medications, such as AV-101, as therapeutic alternatives to the use of opioids. More specifically, over the next 6 to 12 months, the FDA has stated it anticipates issuing several documents intended to stimulate the development of medications for specific types of pain, resulting in smaller clinical trials, faster approvals and quicker launches of novel therapies.
Amneal keeps scoring approvals, but so do competitors, says Morgan Stanley
Morgan Stanley analyst David Risinger lowered his estimates for Amneal’s Q4, FY19 and FY20 as management comments at a conference on Monday drove him to be more conservative. While CEO Rob Stewart did not provide any new financial guidance, he is quoted as having said that in the company’s prior competition assumptions “specifically [for] yuvafem we didn’t expect competition and now that’s come a little bit earlier than expected. Same thing with dipyridamole/aspirin that we saw competition now on that that we didn’t expect and we’re working through what all that means.” In the note, entitled in part “FDA approvals a double-edged sword,” Risinger said that Amneal continues to score pipeline approvals, but so do competitors to existing Amneal products. He lowered his price target on the stock to $24 from $26 and keeps an Overweight rating on Amneal shares.
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