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.

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 AbbVie (NYSE: ABBV) reported that it will participate in the Barclays Global Healthcare Conference on Thursday, March 15, 2018. Bill Chase, executive vice president and chief financial officer, will present at 8 a.m. Central time (Press release, AbbVie, FEB 26, 2018, View Source [SID1234524160]).

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A live audio webcast of the presentation will be accessible through AbbVie’s Investor Relations website at investors.abbvie.com. An archived edition of the session will be available later that day.

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 Athenex, Inc. (Nasdaq:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that it will release fourth quarter and full year 2017 earnings results on March 26, 2018 (Press release, Athenex, FEB 26, 2018, View Source;p=RssLanding&cat=news&id=2334562 [SID1234524161]). The Company will host a conference call and audio webcast on Monday, March 26, 2018 at 9:00 a.m. Eastern Time.

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To participate in the call, dial (855) 227-0567 (domestic) or (612) 979-9912 (international) fifteen minutes before the conference call begins and reference the conference passcode 4156727. A replay will be available approximately one hour after the recording through Monday, April 2, 2018 and can be accessed by dialing (855) 859-2056. The live conference call and replay can also be accessed via audio webcast at the Investor Relations section of the Company’s website, located at www.athenex.com. An archive will be available at this website until April 26, 2018.