Intercept Pharmaceuticals announced two updates to its NASH development program. Intercept anticipates that it will read-out top-line data from the interim analysis of its Phase 3 REGENERATE trial evaluating obeticholic acid in non-cirrhotic NASH patients with advanced liver fibrosis in the first quarter of 2019. Intercept also expects to complete enrollment of its Phase 3 REVERSE trial evaluating OCA in NASH patients with compensated cirrhosis in 2019. “2019 is expected to be a transformative year for Intercept, as we look forward to announcing top-line results from the REGENERATE interim analysis this quarter and continuing to advance our leading Phase 3 development program for the treatment of NASH patients with advanced liver fibrosis and compensated cirrhosis where we believe there is the greatest unmet medical need,” said Mark Pruzanski, M.D., Intercept’s President and CEO.
Monday, January 7, 2019
Amag preliminary Q4 revenue $85M-$90M, consensus $101.9M
For Q4, AMAG expects an operating loss of between $17M-$27M. Adjusted EBITDA for Q4 is expected to be within the company’s forecasted range of between a loss of $5M and positive adjusted EBITDA of $5M.
Syndax provides 2019 clinical, corporate outlook
“2019 is slated to be a milestone-rich time for, with data expected from multiple trials within our ENCORE program of entinostat in combination with checkpoint therapy in platinum resistant ovarian cancer, triple negative breast cancer, and anti-PD-1-pretreated melanoma, all of which we believe represent underserved areas with significant market opportunity,” said Briggs W. Morrison, M.D., CEO of Syndax. “We also eagerly anticipate the next interim overall survival analysis in the second quarter from the Phase 3 E2112 trial of entinostat plus exemestane in HR+, HER2- breast cancer and remain highly encouraged by the potential to provide a survival benefit for HR+, HER2- breast cancer patients who have stopped responding to first line treatment with hormone therapy. Any positive overall survival assessment would enable the company to file for full regulatory approval.” Dr. Morrison added, “In addition, we remain on track for an IND filing for our Menin inhibitor, SNDX-5613, in the second quarter of 2019, followed by initiation of the clinical trial program. Acute leukemias characterized by MLL-rearrangements and nucleophosmin mutations represent areas of high unmet medical need, and the preclinical data we’ve generated thus far provide strong support that menin inhibition has the potential to serve as an effective therapy for patients lacking viable options. Finally, we continue to expect initial efficacy results for SNDX-6352 in chronic graft versus host disease in the second half of 2019.” Syndax ended 2018 with cash, cash equivalents and short-term investments of approximately $80M. For 2019, research and development expenses are expected to be $54M-$58M, and total operating expenses are expected to be $68M-$73M. Research and development expenses and total operating expenses for 2019 are expected to include approximately $2M-$6M, respectively, of non-cash stock compensation
Voyager says to increase size of RESTORE-1 Phase 2 trial following FDA feedback
Voyager Therapeutics announced an update to its VY-AADC clinical program for Parkinson’s disease. In December 2018, the company held a Type B meeting with the U.S. FDA to discuss the overall development program for VY-AADC. Based on the meeting discussion and subsequent written feedback from the FDA, Voyager plans to submit a revised trial protocol that will include an increase in the target number of patients in the RESTORE-1 Phase 2 trial, resulting in 75 to 100 total patients in the trial, and to conduct a staggered-parallel Phase 3 trial of similar size and design to RESTORE-1. These updates incorporate guidance from the FDA from the Type B meeting to conduct two adequate and well-controlled clinical trials for a large patient population such as Parkinson’s disease. “Our recent meeting with the FDA was informative and helps to clarify the expected regulatory pathway for VY-AADC,” said Andre Turenne, president and CEO of Voyager Therapeutics. “We look forward to continuing to engage with the FDA and other regulators as we advance our clinical development program and our work to bring VY-AADC to patients in need.” The RESTORE-1 Phase 2 trial is currently enrolling patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary. The primary efficacy endpoint of RESTORE-1 is ON time without troublesome dyskinesia, or good ON time, as measured by a validated self-reported patient diary at 12 months. Voyager expects RESTORE-1 will take approximately 15 to 21 months to enroll. Voyager plans to begin enrolling patients in RESTORE-2 in both active Phase 2 sites and additional sites globally in the first half of 2020. Voyager anticipates that, if positive, results from RESTORE-1 and RESTORE-2 could potentially form the basis for submission of a biologics license application to the FDA for VY-AADC for the treatment of Parkinson’s disease.
Regeneron sees 2019 Sanofi collaboration revenue $510M-$560M
Sees 2019: Effective tax rate 14%-16%; CapEx $410M-$490M.
ProQR Therapeutics announces design on ILLUMINATE trial
ProQR Therapeutics announced that it has reached agreement with the FDA on the design of a Phase 2/3 pivotal trial for QR-110, now renamed to sepofarsen, in patients with Leber’s congenital amaurosis 10, or LCA10 due to the p.Cys998X mutation in the CEP290 gene. LCA is the leading genetic cause of childhood blindness. The trial is expected to start in the first half of 2019. We anticipate top line results for base-case of 30 trial participants around year-end 2020. ILLUMINATE will be a randomized, double-masked, sham-controlled trial initially enrolling 30 adults and children assigned equally to three parallel arms with 10 participants in each arm. The trial incorporates adaptive sample size re-estimation and is designed with potential to serve as the sole registration trial for the program. Participants will receive a dose of sepofarsen or a sham-injection at the start of the trial, at three months and then every six months. The primary efficacy endpoint for the trial will be change in visual acuity from baseline in the treated arm compared to sham-treated control arm at the 12 month time point. Sham-treated participants may be offered cross-over to active treatment after 12 months, and all participants will continue to receive treatment for a total of 24 months after which they may be offered participation in an open label extension trial. Treatment of the second eye will be adaptively incorporated into the trial.
Cara Therapeutics completes enrollment of KALM-1 Phase 3 trial of Korsuva
Cara Therapeutics announced completion of enrollment in the KALM-1 Phase 3 trial of KORSUVA Injection in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus. More than 350 hemodialysis patients with CKD-aP have now been randomized across approximately 60 clinical sites in the United States. KALM-1 is a multicenter, randomized, double-blind, placebo-controlled 12-week treatment trial in the U.S. with a 52-week open label extension phase that is designed to evaluate the safety and efficacy of 0.5 mcg/kg KORSUVA CR845/difelikefalin injection in 350 hemodialysis patients with moderate-to-severe pruritus. The primary efficacy endpoint is the proportion of patients achieving at least a 3-point improvement from baseline in the weekly mean of the daily 24-hour Worst Itch Numeric Rating Scale score at week 12. In a completed Phase 2 trial, the proportion of patients with an improvement from baseline in the weekly mean Worst Itch NRS score of greater than or equal to3 points at week 8 was statistically significantly higher in the CR845/difelikefalin 0.5 mcg/kg group compared to the placebo group . Secondary endpoints include assessment of itch-related quality of life changes measured using the validated self-assessment 5-D itch and Skindex-10 scales, as well as the proportion of patients achieving greater than 4-point improvement from baseline in weekly mean of the daily 24-hour Worst Itch NRS score at week 12.
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