Monday, December 10, 2018
Vanda Pharmaceuticals announces ‘positive’ study results for HETLIOZ
Vanda Pharmaceuticals announced that HETLIOZ improved sleep quality and increased sleep duration in patients with Smith-Magenis Syndrome in a pivotal placebo controlled clinical study. “We are extremely pleased with the results of this study of tasimelteon in patients with Smith-Magenis Syndrome. Tasimelteon was shown to meaningfully improve sleep in SMS patients, addressing an unmet medical need for the most severe symptom constellation of this rare disorder,” said Mihael H. Polymeropoulos MD, Vanda’s President and Chief Executive Officer. VEC-162-2401 was a double masked 4 week cross-over pivotal clinical trial that studied the effects of tasimelteon versus placebo in 25 patients with SMS. Patients were evaluated for daily diary sleep quality and for daily diary total nighttime sleep duration via a parental post sleep questionnaire. Total nighttime sleep duration was also measured via daily actigraphy. The study had two predefined primary endpoints: DDSQ and DDTST. Tasimelteon met the primary endpoint of improvement in the 50% worst sleep quality and also showed improvement on the primary endpoint of 50% worst total nighttime sleep duration. Tasimelteon demonstrated significant improvement in overall sleep quality and overall total nighttime sleep duration. Tasimelteon improved the overall total nighttime sleep duration by an average of approximately 41 minutes per night, a highly clinically meaningful effect. Given that most of the baseline nights were of shortened sleep duration, tasimelteon also improved sleep duration for the best half of the baseline nights versus placebo. Tasimelteon also showed significant improvement in subjective measures of total nighttime sleep duration via actigraphy, for 50% worst TST and overall TST. In this study, aberrant behaviors improved from baseline on both tasimelteon and placebo but likely due to the relative short duration of the study the differences were not significant between the two groups. In a longer open label study of tasimelteon in SMS, patients were treated for a period of approximately 27 weeks following a 6 week baseline evaluation. In that study, significant improvements from baseline were observed in sleep quality as well as in aberrant behaviors. The improvements in sleep quality and sleep duration demonstrated in the 2401 study were consistent across patients with chromosomal deletions of various lengths as well as a single patient with a point mutation in the RAI1 gene on chromosome 17p. The detailed results are expected to be presented in upcoming meetings and peer reviewed publications.
https://thefly.com/landingPageNews.php?id=2834329
Menlo Therapeutics says serlopitant met primary endpoint in trial
Menlo Therapeutics announced top-line results from MTI-109, a 204-patient Phase 2 clinical trial of serlopitant for the treatment of pruritus associated with psoriasis. The trial successfully met its primary endpoint, showing a statistically significant reduction in pruritus based upon a 4-point improvement responder analysis. In the trial, 33% of patients treated with serlopitant 5 mg daily achieved a 4-point or greater improvement on the worst-itch numeric rating scale, or WI-NRS, at week 8 compared to baseline vs. 21% of patients treated with placebo. The trial also prospectively defined three key secondary endpoints for sequential step-down analyses at week 4 and days 7 and 3. The trial successfully met the secondary endpoint of WI-NRS responder rate at week 4. At week 4, 21% of patients treated with serlopitant achieved a 4-point or greater improvement on the WI-NRS vs. 11% of patients treated with placebo. Assessment of the secondary endpoints of the absolute change in WI-NRS from baseline to day 7 and day 3 for serlopitant compared to placebo showed a greater numerical, but not statistically significant, improvement for the serlopitant group. At every assessed time point in the trial, the serlopitant treated group demonstrated greater numerical improvement than the placebo group in both the WI-NRS 4-point responder analysis and in the mean change in WI-NRS from baseline. Serlopitant was well-tolerated in this clinical trial. No serious adverse events were reported for serlopitant treated patients. Treatment-emergent adverse events assessed as likely related to treatment were observed with similar frequency in both groups. The consolidated safety summary for serlopitant now includes more than 1,600 evaluable patients, including patients who have received treatment for up to one year.
https://thefly.com/landingPageNews.php?id=2834331
RA Pharmaceuticals says Phase 2 trial of zilucoplan meets primary endpoint
Ra Pharmaceuticals announced positive top-line results from the Company’s Phase 2 clinical trial evaluating zilucoplan for the treatment of generalized myasthenia gravis, achieving clinically meaningful and statistically significant reductions in both the primary and key secondary endpoints for both zilucoplan dose groups tested versus placebo at 12 weeks. Zilucoplan dosed at 0.3 mg/kg subcutaneously daily achieved a mean reduction from baseline of 6.0 points in the Quantitative Myasthenia Gravis score and a mean reduction from baseline of 3.4 points in the MG Activities of Daily Living score, with no patients treated with the 0.3 mg/kg dose of zilucoplan requiring rescue therapy. The Phase 2, multi-center, randomized, double-blind, placebo-controlled trial was designed to evaluate the safety, tolerability, and preliminary efficacy of zilucoplan in patients with gMG, regardless of prior therapies, who had a MGFA Disease Class of II-IVa at screening and a QMG score, a physician-administered assessment of MG-related muscle weakness, of greater than or equal to 12 at screening and randomization. The trial enrolled 44 patients in the U.S. and Canada. At the outset of the 12-week treatment period, patients were randomized in a 1:1:1 ratio to receive daily, SC doses of 0.1 mg/kg of zilucoplan, 0.3 mg/kg of zilucoplan, or matching placebo. The pre-specified primary efficacy endpoint was the change in QMG score from baseline to week 12. The key secondary efficacy endpoint was the change in MG-ADL score, a patient-reported outcome measure, from baseline to week 12. Significance testing was pre-specified at a 1-sided alpha of 0.1. All 44 patients completed the 12-week study and, of these, 43 elected to enter a long-term extension to receive active study drug. The pre-specified primary efficacy endpoint of change from baseline to Week 12 in QMG score was met with zilucoplan dosed at 0.3 mg/kg SC daily, resulting in a clinically meaningful and statistically significant improvement over placebo.The key secondary efficacy endpoint of change from baseline to Week 12 in the MG-ADL score was met with zilucoplan dosed at 0.3 mg/kg SC daily, resulting in a clinically meaningful and statistically significant improvement over placebo. QMG and MG-ADL outcomes for the 0.1 mg/kg SC daily dose were similar to but less pronounced than the 0.3 mg/kg SC daily dose, also achieving pre-specified statistical significance on both endpoints. The threshold for statistical significance used in pivotal Phase 3 studies was achieved in a pre-specified analysis of the pooled active arms versus placebo, which showed a placebo-corrected change in MG-ADL at Week 12 = -2.2. Rescue therapy with intravenous immunoglobulin or plasma exchange was required by 3/15 patients in the placebo arm, 1/15 patients in the 0.1 mg/kg zilucoplan arm, and in zero patients in the 0.3 mg/kg zilucoplan arm. Treatment with zilucoplan had a favorable safety and tolerability profile in the study, consistent with previously-completed Phase 1 and Phase 2 studies. The majority of adverse events reported were mild and were not considered by the investigators to be related to study drug. There were no serious AEs observed related to treatment with zilucoplan. Based on these data, Ra Pharma plans to engage with regulatory agencies, including the U.S. Food and Drug Administration, in the first half of 2019 regarding the design of a Phase 3 clinical trial evaluating the 0.3 mg/kg dose of zilucoplan versus placebo in patients with gMG.
https://thefly.com/landingPageNews.php?id=2834335
Ironwood price target lowered to $11.50 from $12.50 at H.C. Wainwright
H.C. Wainwright analyst Raghuram Selvaraju lowered his price target for Ironwood Pharmaceuticals to $11.50 and reiterates a Sell rating on the shares. The company’s “poor” recent financial performance is unlikely to improve in the near-term, Selvaraju tells investors in a research note. He expects Linzess pricing decreases and challenges to uptake will continue.
https://thefly.com/landingPageNews.php?id=2834345
MacroGenics cut to Underperform from Outperform at Raymond James
https://thefly.com/landingPageNews.php?id=2834355
Lonza Sets China Biomanufacturing Base Using GE Healthcare Solution
- New biologics facility, scheduled to be operational in 2020, will provide development and manufacturing facilities from early- to late-clinical and early-commercial stages
- GE Healthcare’s off-the-shelf biologics factory, KUBio, will provide the base for GMP manufacturing
- Memorandum of understanding to be signed with Guangzhou Development District for leasing of the 17,000m2 site
Lonza today announced an agreement with GE Healthcare under which GE Healthcare will deliver a biologics facility to Lonza in Guangzhou (CN). As the facility is part of a larger biomanufacturing initiative between GE Healthcare and Guangzhou Development District (GDD), the agreement will be finalized when contracts have been signed with the GDD. Later today, Lonza will sign a memorandum of understanding with the GDD to take the project forward. The new facility will give Lonza Pharma & Biotech a strategic base in China to respond to growing demand for high-quality CDMO services in the country.
By working with GE Healthcare to develop the new facility, based on the KUBio platform, an off-the-shelf biologics factory, Lonza intends to offer its suite of antibody development services and manufacture GMP-certified batches by 2020. The two companies will work closely on the design of the 17,000m2 site, which will include 6,500m2 of lab space and one KUBio facility.
The labs will house Lonza’s proprietary platforms for cell-line construction, including the GS Gene Expression System®, as well as process development, cell banking and pilot labs. The KUBio unit will enable small-scale GMP manufacturing equipped with GE Healthcare’s single-use biomanufacturing technologies, including 1,000 and 2,000L bioreactors, combined with Lonza’s automation platforms for clinical and early-commercial supply.
“This partnership is the ideal way for Lonza to bring our expertise and technology to China,” said Marc Funk, COO Lonza Pharma & Biotech. “The combination of a strong and experienced technology provider with a long history in China, together with a dynamic regional authority, means that we can rapidly offer services to customers and ultimately get innovative therapies to patients more quickly.”
GE Healthcare is working with the GDD to support large-scale manufacturing of biopharmaceuticals in China.
“With Lonza coming on site, a hub of biotech is truly taking shape,” said Emmanuel Ligner, President and CEO, GE Healthcare Life Sciences. “This agreement is yet another step in realizing our vision of enabling and supporting manufacturers of all sizes to quickly deploy capacity and meet increasing customer demand for biologics.”
The facility in China will extend Lonza Pharma & Biotech’s global biologics network that provides development and manufacturing services from gene through IND and BLA to mature commercial supply. In addition to the new Chinese site, the network comprises small-, mid- and large-scale assets in Switzerland, the United States, the UK, Spain and Singapore.
Lonza plans to hire and train more than 160 staff, and the new site will benefit from the company’s global standards of technological and operational excellence. With more than 20 successful FDA approvals for biologics, combined with international regulatory expertise, Lonza will now be able to offer the same high standards in China as it does elsewhere.
China has been historically underserved by biologics, but demand for antibody therapies is expected to grow significantly over the coming years. A strong scientific base and commercial instruments put into place by the Chinese authorities are encouraging their domestic companies developing innovative therapies for the global market, as well as multinationals with manufacturing requirements in the country. In particular, national rollout of the Marketing Authorization Holder scheme by 2019 paves the way for dedicated CDMO services in China.
Gilead Sciences Names Daniel O’Day Chairman and Chief Executive Officer
– Mr. O’Day Brings Global Pharmaceutical Leadership Experience Across Geographies and Therapeutic Areas —
— Appointment Effective March 1, 2019 —
Gilead Sciences, Inc. (Nasdaq: GILD) announced todaythat its Board of Directors has named Daniel O’Day Chairman of the Board and Chief Executive Officer, effective March 1, 2019. Mr. O’Day is currently the CEO of Roche Pharmaceuticals. He has held the position since 2012, and prior to that led Roche Diagnostics. His career spans three decades of diverse leadership roles across North America, Asia Pacific and Europe.
The Board has also appointed Gregg Alton as interim Chief Executive Officer for the period of January 1, 2019 until Mr. O’Day’s start date of March 1, 2019. Mr. Alton has held a number of executive positions at Gilead over the past 20 years, with experience in legal, medical affairs, policy and commercial. He previously served as general counsel and in August of this year, was appointed Chief Patient Officer.
“Following a comprehensive search, the Board became convinced that Dan is the right leader to bring Gilead into the future,” said John C. Martin, PhD, Chairman, Gilead Sciences Board of Directors. “He is uniquely qualified to take on this role given his track record of success in highly scientific and competitive therapeutic areas, deep understanding of the evolving healthcare environment around the world, and unwavering commitment to driving innovation across all aspects of a business, which will serve Gilead and our stakeholders well. Additionally, Dan brings expertise and values that are aligned with our organization, and I, along with Gilead’s entire Board, am confident in his ability to work alongside our talented leadership team and deliver on our ambitious goals.”
After joining Roche Pharmaceuticals in 1987, Mr. O’Day held various positions in the United States before moving to Roche headquarters in Switzerland in 1998. During his time in Switzerland, he held leadership roles in Global Marketing and Lifecycle Management. In 2001, he moved to Tokyo to become Head of Corporate Planning for Roche Pharmaceuticals in Japan and later moved to Denmark to serve as General Manager. He became President of Roche Molecular Diagnostics in California in 2006 and subsequently returned to Roche headquarters to lead the Diagnostics Division before assuming his current position. He is a member of the corporate executive committee of F. Hoffmann La Roche AG and a member of the boards of Shanghai Roche Pharmaceuticals Ltd., Roche (China) Holding, Roche Pharma Schweiz AG, Genentech, Inc., Chugai Pharmaceuticals Co., Ltd., Flatiron Health, Inc., and Foundation Medicine, Inc. Additionally, he has served as a member of the board of the European Federation of Pharmaceutical Industries and Associations. Mr. O’Day holds a Bachelor of Science in Biology from Georgetown University in Washington, D.C. and an MBA from the Columbia Business School at Columbia University in New York.
“I have long admired Gilead for its work to develop medicines that have fundamentally changed the way HIV and viral hepatitis are treated. The company has successfully grown into a global organization, providing access to people around the world, while maintaining its focus on innovative science,” said Mr. O’Day. “Together with the Board, leadership team and Gilead’s 11,000 employees, I look forward to building on this in ways that I believe will – in keeping with Gilead’s mission – transform the lives of millions of individuals.”
As previously announced, Dr. Martin will step down from the company’s Board of Directors, effective March 1, 2019, Mr. O’Day’s first day of employment. Also as previously announced, John F. Milligan, PhD, will step down from his role as President and Chief Executive Officer and as a member of the Board at the end of 2018.
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