On February 26, 2018 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that Reshma Kewalramani, M.D., has been appointed Chief Medical Officer and Executive Vice President, Global Medicines Development and Medical Affairs, effective April 1, 2018 (Press release, Vertex Pharmaceuticals, FEB 26, 2018, View Source [SID1234524171]). Dr. Kewalramani currently serves as Senior Vice President, Clinical Development and Medical Affairs at Vertex and will succeed Jeffrey A. Chodakewitz, M.D., who is retiring from his role as Chief Medical Officer and Executive Vice President, Global Medicines Development and Medical Affairs. Dr. Chodakewitz will remain with Vertex as a senior advisor through early 2019.

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Since joining Vertex in 2014, Dr. Chodakewitz has been an inspirational senior medical leader and has played an instrumental part in the company’s advances in cystic fibrosis (CF) and in expanding Vertex’s development pipeline. Prior to Vertex, Dr. Chodakewitz had an outstanding 20-year career at Merck, where he led the successful development of multiple medicines and vaccines across a number of therapeutic areas.

"I would like to personally thank Jeff for his leadership and dedication to improving the lives of people with cystic fibrosis and other serious and life-threatening diseases," said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. "He has played a critical role in leading multiple successful clinical development efforts at Vertex, and I’m pleased that we will continue to benefit from his guidance as a senior advisor over the next year as he transitions to retirement. I’m also delighted that Reshma has agreed to succeed Jeff as our Chief Medical Officer. Reshma’s depth of medical knowledge, paired with her experience and proven track record as a clinical leader at Vertex, makes her an ideal successor to Jeff. I look forward to working with Reshma to continue to advance our clinical development pipeline in cystic fibrosis and other serious diseases."

"It has been an honor to lead Vertex’s clinical development efforts and to be a part of the significant advancement in treatments for cystic fibrosis patients over the last four years," said Dr. Chodakewitz. "I have enjoyed working closely with Reshma since she joined the Company last year and am confident that Vertex will continue its success in pursuing innovative therapies under her leadership."

"It’s a privilege to work at a company with such a relentless focus on science and deep commitment to improving the lives of patients and their families," said Dr. Kewalramani. "I am excited to assume the role of CMO and continue our important work to help more people with cystic fibrosis and other serious diseases alongside the talented team at Vertex."

Dr. Kewalramani is an accomplished leader and physician who has a track record of building strong teams, putting patients first, leading change and delivering results. Prior to joining Vertex in February 2017, Dr. Kewalramani spent over 12 years at Amgen where she most recently served as Vice President and Head of the U.S. Medical Organization. During her tenure at Amgen, Dr. Kewalramani held roles with increasing responsibility across Clinical Development and Medical Affairs and served in leadership positions involving research and development and commercialization activities across a variety of therapeutic areas. Dr. Kewalramani is the industry representative to the Food and Drug Administration (FDA) Endocrine and Metabolic Drug Advisory Committee and was on the inaugural Board of Directors of the Kidney Health Initiative. She completed her internship and residency in Internal Medicine at the Massachusetts General Hospital and her fellowship in Nephrology at the Massachusetts General Hospital and Brigham and Women’s Hospital combined program. Dr. Kewalramani received her medical degree, with honors, from the Boston University School of Medicine and is also an alumnus of the Harvard Business School.

On February 26, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported that its 6-month study on storage of the frozen encapsulated cells necessary for the determination of an "initial shelf life" has been successfully completed by Austrianova (Press release, PharmaCyte Biotech, FEB 26, 2018, View Source [SID1234524167]). This work involved the removal of samples of encapsulated cells from -80o Celsius (C) storage at various intervals up to six months, thawing the encapsulated cells and testing them for various functional parameters as well as for sterility.

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Regulatory agencies, including the U.S. Food and Drug Administration (FDA), require that studies be conducted to determine an initial shelf life for a medicinal product before an advanced phase clinical trial can begin. This shelf life can then be extended with further testing as the product is being used in the clinical trial. Austrianova Singapore already has data demonstrating storage life of frozen encapsulated cells for over six years; however, these studies were done with a slightly different cell line than the cell line that will be used for PharmaCyte’s planned clinical trial. The current completed study uses the Research Bank of cells that will be used in the final product for PharmaCyte’s trial and from which Eurofins has already generated PharmaCyte’s Master Cell Bank.

The stability test results show that there are no significant changes in the viability or the enzymatic activity of the cells upon thawing after a 6-month storage period at -80oC. These results will allow an initial shelf life of six months to be set for the product; this will be extended as the product is tested further.

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, stated, "We are pleased with this significant result which will enable us to apply to the FDA for an initial shelf life of six months. This study, one of many being performed for us by Austrianova, is an essential part of the Investigational New Drug Application (IND) that we will be submitting to the FDA."

PharmaCyte is working on an IND to submit to the FDA so that it can begin a clinical trial involving locally advanced, inoperable, non-metastatic pancreatic cancer and expects to submit its IND later this year.

On February 26, 2018 Quentis Therapeutics Inc., a biotechnology company pursuing next-generation immuno-oncology research and drug development, reported that it debuted with the completion of a $48 million Series A financing co-led by founding investor Versant Ventures and by Polaris Partners and the affiliated LS Polaris Innovation Fund (Press release, Quentis Therapeutics, FEB 26, 2018, View Source [SID1234524356]). The syndicate also included AbbVie Ventures, Taiho Pharmaceutical Co., Ltd., Yonghua Capital, Alexandria Venture Investments and New York Ventures, the investment arm of Empire State Development.

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Quentis is pioneering first-in-class cancer immunotherapies that modulate endoplasmic reticulum (ER) stress response pathways in the tumor microenvironment. The Series A proceeds will support the advancement of Quentis’ lead program, a small molecule IRE1α inhibitor, into the clinic in 2019 and through clinical proof-of-concept. Additional uses of the capital include developing a pipeline of preclinical programs and building out Quentis’ team.

The company was enabled by Highline Therapeutics, a Versant Ventures Discovery Engine, under an agreement with Weill Cornell Medicine through its office of Biopharma Alliances and Research Collaborations to establish New York-based spinout companies to advance breakthroughs emerging from Weill Cornell Medicine research.

Quentis’ scientific foundation is based on landmark research conducted by Laurie Glimcher, M.D., previously Dean of Weill Cornell Medicine, now President and Chief Executive Officer of Dana-Farber Cancer Institute and Professor at Harvard Medical School, and Juan Cubillos-Ruiz, Ph.D., Assistant Professor of Microbiology and Immunology in Obstetrics and Gynecology at Weill Cornell Medicine. Drs. Glimcher and Cubillos-Ruiz’s research revealed critical roles that the ER stress response plays in compromising the immune system’s ability to detect and fight cancer.

Chronic activation of the ER stress response correlates with poor outcomes in multiple tumor types, including ovarian cancer, triple-negative breast cancer, pancreatic adenocarcinoma and glioblastoma. By modulating ER stress response pathways, Quentis aims to boost a patient’s own anti-cancer immunity and enable more patients to benefit from immunotherapy. The company is pursuing multiple ER stress pathway targets in the tumor microenvironment, as well as in other diseases where ER stress plays an important role.

"We’ve witnessed great progress in our ability to harness the immune system to fight cancer. However, despite these advances, the effectiveness of immunotherapy remains limited, and many patients and many types of cancer don’t respond to treatment," said Michael Aberman, M.D., president and CEO of Quentis Therapeutics. "The scientific community continues to learn about important mechanisms, like the ER stress response, that impact cancer immunity. At Quentis, we are excited to be part of the next generation of immuno-oncology companies that are pursuing new therapeutic approaches to hopefully enable more cancer patients to benefit from immunotherapy."

Dr. Aberman continued, "We are thrilled to introduce Quentis today, and we are grateful for the dedication and support of our investors and all those who have helped us reach this important milestone. We look forward to continuing to build out our team in the coming months as we advance toward entering the clinic in 2019."

"It is profoundly gratifying to see our discoveries on the key role played by the ER stress response in inhibiting effective anti-tumor immunity translate into the advancement of potentially meaningful medicines for patients," said Dr. Glimcher, a scientific co-founder of Quentis and chair of the company’s Scientific Advisory Board. "I look forward to continuing to work closely with the Quentis team as they advance development programs toward clinical study."

"Immunotherapy is changing the face of cancer treatment, but harsh conditions within tumors inhibit the protective activity of immune cells and present an impediment to broad efficacy with immunotherapies. We’ve made important strides in understanding how aberrant ER stress responses in cancer promote immune cell dysfunction, and we continue to expand our knowledge of this novel biology," said Dr. Cubillos-Ruiz, a scientific co-founder of Quentis and member of the company’s Scientific Advisory Board. "I look forward to seeing Quentis translate this maturing knowledge in the drug development setting."

"Quentis is assembling all the necessary elements to become a leading company in the ER stress response and immuno-oncology fields," said Carlo Rizzuto, Ph.D., a partner at Versant and a Quentis board member. "We have great confidence in Michael’s ability to build the company and advance its programs to develop new treatments for patients."

In connection with the financing, Amy Schulman, partner with Polaris Partners and LS Polaris Innovation Fund, will join Dr. Rizzuto, Michael A. Foley, Ph.D., Sanders Director, Tri-Institutional Therapeutics Discovery Institute, and Dr. Aberman on Quentis’ Board of Directors.

On February 26, 2018 Alder BioPharmaceuticals, Inc. (NASDAQ:ALDR), a biopharmaceutical company focused on developing novel therapeutic antibodies for the treatment of migraine, reported a corporate update and reported its financial results for the fourth quarter and full year ended December 31, 2017 (Press release, Alder Biopharmaceuticals, FEB 26, 2018, View Source [SID1234524175]).

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"The data from eptinezumab’s Phase 3 pivotal clinical trials in episodic and chronic migraine patients support Alder’s goal to advance the treatment paradigm for migraine prevention. In both trials, eptinezumab’s clinical profile included rapid Day 1 reduction in migraine risk, high levels of efficacy with patients achieving 75% and 100% responder rates and sustained migraine relief for 3 months following a single infusion administration," said Randall C. Schatzman, Ph.D., President and Chief Executive Officer of Alder. "If approved, eptinezumab has the potential to provide a meaningful treatment option for millions of migraine sufferers. Looking ahead to the remainder of 2018 and into 2019, we are focused on our BLA submission, gaining FDA approval and on our commercial readiness activities ahead of eptinezumab’s launch. Our 2017 year-end cash balances, together with the net proceeds of our two successful 2018 financings, total over $600 million, leaving us well-positioned to meet our projected operating requirements into 2020."

Recent 2018 Company Highlights

PROMISE 2 Phase 3 eptinezumab top-line data in chronic migraine patients:
Met the primary endpoint with very high statistical significance vs. placebo (p<0.0001) for both dose levels tested in the trial following the first quarterly infusion.
Met all key secondary endpoints with very high statistical significance vs. placebo including prevention beginning Day One (p<0.0001) and 50 percent (p<0.0001) and 75 percent (p<0.0001) responder rates month one through month three. Furthermore, an average of 15 percent of eptinezumab patients had no migraines (i.e., 100 percent response) for months one to three (p<0.0001 unadjusted).
Safety and tolerability were similar to previously reported eptinezumab studies.

European patent settlement and global license agreement with Teva Pharmaceuticals International GmbH clears Alder’s freedom to develop, manufacture and commercialize eptinezumab in the U.S. and globally.

Alder received approximately $97.7 million in net proceeds from the sale of shares of convertible preferred stock in a committed equity financing with certain institutional and other accredited investors affiliated with or managed by Redmile Group LLC.

Alder received approximately $277.7 million in net proceeds from an underwritten public offering of 2.5% convertible senior notes due 2025 (including approximately $36.3 million from the exercise of an over-allotment option granted to the underwriters in the offering).

Upcoming Corporate Milestones
Planned 2018 Corporate Milestones for eptinezumab Timing
PROMISE 1 12 month data (episodic migraine) 1H 2018
PROMISE 2 6 month data (chronic migraine) 1H 2018
12 month open label safety study 1H 2018
Pharmacokinetic comparability study 2H 2018
BLA submission 2H 2018

Key 2017 Company Highlights

PROMISE 1 Phase 3 eptinezumab data in episodic migraine patients:
Met the primary endpoint with highly statistically significant reductions in monthly migraine days in the trial following the first quarterly infusion.
Significant clinical benefit achieved on Day One post-infusion (p=0.0087 unadjusted) and significant 50 percent (p=0.0001) and 75 percent (p=0.0007) responder rates month one through month three.
Efficacy further improved following a second quarterly infusion; an average of 17% of patients had no migraines following the first administration and that rose to 26% of patients following the second administration.
The safety profile was similar to placebo and consistent with previously reported eptinezumab studies.

Alder completed a public offering of common stock on July 18, 2017 resulting in net proceeds to Alder of approximately $161.5 million, after underwriting discounts, commissions and offering expenses.

Eptinezumab data presentations at top tier medical conferences highlighted Phase 2b (chronic migraine) and PROMISE 1 Phase 3 (episodic migraine) clinical data and analyses:
Three scientific presentations at the 69th Annual American Academy of Neurology (AAN)April 22-28.
Four scientific presentations at the 59th Annual Scientific Meeting of the American Headache Society (AHS) June 8-11.
Seven scientific presentations at the 18th Congress of the International Headache Society (IHC) September 7-10.

Fourth Quarter and Year-End 2017 Financial Results

As of December 31, 2017, Alder had $286.2 million in cash, cash equivalents, short-term investments and restricted cash, compared to $340.9 million as of Sept. 30, 2017 and compared to $351.9 million as of December 31, 2016.

Research and development expenses for the fourth quarter ended December 31, 2017 totaled $44.7 million, compared to $41.8 million for the same period in 2016. For the full year 2017, research and development expenses totaled $252.9 million, compared to $132.8 million for the full year 2016. The increases in spending for both periods were primarily due to manufacturing and clinical trial costs for the company’s eptinezumab program and commercialization preparations.

General and administrative expenses for the fourth quarter ended December 31, 2017 totaled $10.3 million, compared to $7.4 million for the same period in 2016. For the full year 2017, general and administrative expenses totaled $38.1 million, compared to $26.1 million for the full year 2016. The increases in spending for both periods were primarily due to an increase in stock-based compensation expense and salaries due to headcount growth, and an increase in professional fees and other administrative costs, primarily to support commercial readiness activities.

Net loss for the fourth quarter ended December 31, 2017 totaled $54.4 million, or $0.80 per share, compared to net loss of $48.9 million, or $0.97 per share on a fully-diluted basis, for the same period in 2016. For the full year 2017, net loss totaled $288.9 million, or $4.95 per share on a fully-diluted basis, compared to net loss of $156.3 million, or $3.23 per share, for the full year 2016.

Financial Outlook

Alder estimates its available cash, cash equivalents, short-term investments and restricted cash totaling $286.2 million as of December 31, 2017, together with the net proceeds of approximately $97.7 million received in 2018 from the sale of shares of convertible preferred stock in its committed equity financing and approximately $277.7 million received in 2018 in its underwritten public offering of 2.5% convertible senior notes due 2025 will be sufficient to meet projected operating requirements into 2020. These projections assume the BLA filing and approval by the U.S. Food and Drug Administration and the commercial launch of the infusion formulation of eptinezumab in this time period.

Conference Call and Webcast
Alder will host a conference call today at 5:00 p.m. ET to discuss these financial results and recent corporate highlights. The live call may be accessed by dialing (877) 430-4657 for domestic callers or (484) 756-4339 for international callers, and providing conference ID number 6943609. The webcast will be broadcast live and be accessed from the Events & Presentations page in the investors section of Alder’s website at www.alderbio.com. The webcast will be available for replay following the call for at least 30 days.

Allergan plc (NYSE:AGN), a leading global biopharmaceutical company, reported that Chief Commercial Officer William Meury will present at the Cowen and Company 38th Annual Health Care Conference in Boston, Massachusetts (Press release, Allergan, FEB 26, 2018, View Source [SID1234524176]). The presentation will begin at 3:30 p.m. Eastern Time on Monday, March 12, 2018.

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The presentation will be webcast live and can be accessed on Allergan’s Investor Relations website at www.allergan.com/investors. The webcast can also be accessed through the following URL: http://wsw.com/webcast/cowen46…

An archived version will be available immediately following the live presentation, and can be accessed at the same location for 90 days.