Mylan Faces Further Scrutiny- Mylan (MYL) faces further scrutiny from lawmakers after the House Oversight and Government Reform Committee has launched an investigation into the cost of EpiPens. The committee noted that Mylan has a “virtual monopoly” over the auto-injector market. The committee also described the company’s pricing strategy as “unreasonable.”
Portola Announces Positive Interim Results from Phase 3b/4 Study of Andexanet Alfa- Portola Pharmaceuticals (PTLA) announced positive interim results from a Phase 3b/4 study, ANNEXA-4, evaluating its Factor Xa inhibitor antidote AndexXa (andexanet alfa) in reversing acute major bleeding associated with the anticoagulant. The data were presented today at the European Society of Cardiology Congress in Rome. Preliminary analysis on interim results from 67 patients (47 evaluated for efficacy) showed a 30-minute bolus of AndexXa rapidly and substantially reversed anti-Factor Xa activity and sustained the reversal when followed by a 120-minute infusion. The majority of patients (79%) achieved good or excellent hemostasis (bleeding stopped) over a 12-hour period after the infusion. The interim data showed no AndexXa-associated infusion reactions and no patients developed antibodies to Factor Xa or Factor X or neutralizing antibodies to AndexXa.
Rigel Pharmaceuticals’ Late-Stage Study Achieves Primary Endpoint- Rigel Pharmaceuticals (RIGL) shares surged almost 50% on Tuesday after the company announced results from a Phase 3 clinical trial, Study 047, assessing lead product candidate fostamatinib for the treatment of immune thrombocytopenia (ITP), a bleeding disorder resulting from a person's immune system destroying blood platelets that affects ~60K people. 18% (n=9/51) achieved the primary endpoint compared to 0% for placebo, a statistically valid result. The rates of treatment-related adverse events (TRAEs) were 77% (n=39/51) for fostamatinib and 28% (n=2/25) for placebo.
Kamada’s Mid-Stage Study of Inhaled AAT Therapy Succeeds- Kamada (KMDA) announced ositive results in a Phase 2 clinical trial assessing its proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the treatment of Alpha-1 Antitrypsin Deficiency (AATD), an orphan disease currently treated by intravenous AAT augmentation therapy. The primary efficacy endpoints included antigenic AAT levels and Anti-Neutrophil Elastase Inhibitory (ANEC) levels in the lung as well as additional anti-proteolitic and anti-inflammatory biomarkers. Patients in the inhaled AAT therapy cohort showed a statistically significant increase in endothelial lining fluid (ELF) AAT antigenic level compared to placebo (p<0.0005). The levels were more than twice the increase observed in an earlier study of intravenous (IV) AAT therapy.
Array BioPharma Announces Encouraging Results From Mid-Stage Study of ARRY-797- Array BioPharma (ARRY) announced positive results from a Phase 2 clinical trial evaluating ARRY-797 for the treatment of patients with lamin A/C-related dilated cardiomyopathy (LMNA-DCM), a rare degenerative cardiovascular disease caused by mutations in the LMNA gene that is associated with a poor prognosis. The data were presented at the European Society of Cardiology Congress in Rome. The mean change from baseline in the six-minute walk test (6MWT), the primary endpoint, was 69 meters. Sustained improvement in related biomarkers was also observed. A trend for greater improvement was seen with the 400 mg dose.
ARIAD Completes NDA Submission for Brigatinib- ARIAD Pharmaceuticals (ARIA) announced that it completed he rolling submission of its New Drug Application (NDA) to the FDA seeking approval of Breakthrough Therapy- and Orphan Drug-tagged brigatinib for the treatment of patients with ALK+ non-small cell lung cancer (NSCLC) who are resistant to Pfizer’s (PFE) XALKORI (crizotinib). The company has requested accelerated review of the filing.
OncoMed Announces Completion of Enrollment in Phase 2 Study of Tarextumab- OncoMed Pharmaceuticals (OMED) announced that it has completed enrollment in its Phase 2 clinical trial, PINNACLE, evaluating tarextumab (OMP-59R5) for the treatment of small cell lung cancer (SCLC). Top-line data should be available by the end of this year or early next year. The study is expected to be completed in April 2018.
Veltassa Succeeds in Late-Stage Study- Relypsa (RLYP) announced that a Phase 4 study evaluating its Veltassa (patiromer) for oral suspension, taken with or without food, for the treatment of hyperkalemia (excess blood potassium) met its primary endpoint. Results showed no statistically significant difference in the proportion of patients achieving serum potassium levels within the target range (3.8 - 5.0 mEq/L) by Week 3 or 4 between those taking Veltassa with food or without. Adverse events were consistent with earlier trials. Relypsa agreed to a $32 per share takeover by Vifor Pharma earlier in the year.
The Medicines Company’s Mid-Stage Study of MDCO-216 Fails to Achieve Efficacy Results- The Medicines Company (MDCO) announced that a Phase 2 proof-of-concept study evaluating its MDCO-216 for educing atherosclerotic plaque burden failed to achieve the efficacy results necessary for an early end to the trial. The Independent Data Monitoring Committee recommended the study continue citing the inconclusiveness of the interim data from 40 subjects. Results from the full group of 120 patients will be presented at the American Heart Association Scientific Sessions in mid-November.
Onconova Announces Workforce Reduction- Onconova Therapeutics (ONTX) announced that it is terminating 21% of its workforce. The workforce reduction will save around $1.4 million per year.