Neon Therapeutics announced that updated data from NT-001, its ongoing Phase 1b clinical trial evaluating NEO-PV-01 in the metastatic setting, will be presented in an upcoming oral presentation at the European Society for Medical Oncology, or ESMO.
Wednesday, October 3, 2018
Oasmia reports Phase 2 dog study meets primary endpoint
Oasmia reported its analysis of the data from the Phase II study and it’s the follow-up has been finalized and summarized in a report. Overall, 17 dogs participated and were treated with Doxophos Vet in the main study and eight of these continuing during the follow-up part. The objective was obtained, as 75% of the dogs with B-cell lymphoma showed a response after at least two treatment cycles. Progression free survival was defined as the time from screening to progression, or death of any cause. The study results confirmed that the dose of Doxophos Vet defined in a prior dose-finding study is appropriate with regards to both efficacy and safety in dogs with lymphoma. The pharmacokinetic profile analyzed shows there is a rapid distribution of doxorubicin into the tissues, and that the Doxophos Vet formulation does not alter the release properties of doxorubicin when infused into the blood. The most frequently reported adverse reaction after Doxophos Vet treatment was myelosuppression. This is the most expected adverse reaction with chemotherapy. Of the majority of the adverse events, 88% in the main study and 69% in the follow-up were mild to moderate.
PTC Therapeutics reports results of risdiplam in spinal muscular atrophy
PTC Therapeutics announced interim clinical data from the Part 1, open-label studies of FIREFISH and SUNFISH demonstrating the ‘benefit’ of risdiplam for the treatment of Type 1, 2 and 3 spinal muscular atrophy. The results showed that patients across all SMA types benefited from an oral systemic therapy indicated by increases in developmental motor milestones. Risdiplam was well tolerated at all doses across studies to date. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche. In Part 1 of the FIREFISH study, at Day 245 of treatment, 43% or 6/14, of infants were able to sit with or without support, including three who achieved unassisted sitting. Natural history indicates that Type 1 SMA babies never achieve this milestone. In Part 1 of the SUNFISH study in Type 2 and 3 SMA patients, 63% or 19/30, of patients treated with risdiplam for at least one year achieved a median increase in motor function of 3.13 points versus baseline. Typically patients with Type 2 or 3 SMA decline by 0.85 to 0.67 point per year. The pivotal portion of the SUNFISH clinical study has completed enrollment. Patients enrolled in the SUNFISH trial have a broad age range (2-24 years; median age 8 years) and with broad functional characteristics.
Genentech presents new data for risdiplam in Spinal Muscular Atrophy
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.
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.
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