AstraZeneca And Daiichi Sankyo To Jointly Commercialise MOVANTIK; Amicus Provides Positive Global Regulatory Updates for Fabry Monotherapy Print E-mail
By Marilyn Mullen   
Thursday, 19 March 2015 18:49
Below is a look at some of the headlines for companies that made news in the healthcare sector on March 19, 2015.  
Nektar Therapeutics Inc (NASDAQ: NKTR) reported that partner AstraZeneca today announced a co-commercialisation agreement with Daiichi Sankyo,
reported that partner AstraZeneca today announced a co-commercialisation agreement with Daiichi Sankyo, Inc. for MOVANTIK™ (naloxegol) in the US, in line with the Company's strategy of delivering value through its own development and commercial capabilities as well as through external collaboration. MOVANTIK is a first-in-class once-daily oral peripherally-acting mu-opioid receptor antagonist (PAMORA) for the treatment of opioid-induced constipation (OIC) in adults with chronic non-cancer pain.
MOVANTIK was approved by the US Food and Drug Administration in September 2014. It was descheduled by the US Drug Enforcement Administration in January 2015 and is no longer labelled as a controlled substance. The launch of MOVANTIK in the US is planned for early April 2015.  
Under the terms of the agreement, Daiichi Sankyo Inc. will pay a $200 million up-front fee and subsequent sales-related payments of up to $625 million. AstraZeneca will be responsible for manufacturing, will book all sales and will make sales-related commission payments to Daiichi Sankyo, Inc. Both companies will be jointly responsible for commercial activities. AstraZeneca's 2015 financial guidance, provided on 6 March 2015, is unaffected by today's announcement.
Paul Hudson, President, AstraZeneca US and Executive Vice President, North America, said: "We are delighted to collaborate with Daiichi Sankyo to expand  our  commercialisation efforts  in the US in order to get this important medicine to the large number of patients suffering with opioid-induced constipation. Our agreement reflects our evolving business model by creating value from our portfolio through externalisation activity. Together, we will grow the potential of this important treatment, while we retain our significant interest in the long-term commercial success of MOVANTIK in our largest market."
Ken Keller, President, US Commercial, Daiichi Sankyo, Inc., said: "We are proud to bring our proven primary care and specialty expertise to this collaboration with AstraZeneca. MOVANTIK represents an opportunity to help patients manage one of the most common conditions arising from widely used pain medications, as well as an opportunity to continue to build the Daiichi Sankyo US portfolio of medicines in this therapeutic area."
The agreement is in line with AstraZeneca's business model, which includes value creation from the strong science underpinning its pipeline and portfolio through externalisation activity. This approach aims to benefit patients by collaborating with subject matter experts that can help us to bring important treatments to market while delivering revenue. 
 
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Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced that it has met very recently with regulatory authorities in Europe and the U.S. to discuss the approval pathways for the oral small molecule pharmacological chaperone migalastat HCl ("migalastat") as a precision medicine monotherapy for Fabry patients who have amenable genetic mutations.
Meeting with European Rapporteurs -- The Company held a meeting with the assigned European Rapporteurs to discuss the upcoming marketing authorization application (MAA) for migalastat monotherapy. Amicus Chief Medical Officer Dr. Jay Barth led the Amicus delegation at the meeting in Europe last week. It was the first face-to-face meeting with the assigned Rapporteurs on the proposed dossier for migalastat monotherapy. The European Medicines Agency (EMA) assigns Rapporteurs to review the data in the MAA in detail and to provide guidance to sponsors during the regulatory approval process. The meeting was highly successful for Amicus. The key takeaways from the meeting include:
  • Acknowledgment from the Rapporteurs of the continued significant unmet need in Fabry disease, and the need for innovative treatment options such as migalastat monotherapy
  • The Rapporteurs commented favorably upon the quality and quantity of the migalastat data submitted, which included data from the two largest Phase 3 studies ever conducted in Fabry disease and over 400 patient years of data
  • The Rapporteurs also noted that the Amicus clinical development plan followed the prior scientific guidance from EMA
  • The Rapporteurs agreed that Amicus may submit its MAA for migalastat monotherapy under the Full Approval pathway in the EU
  • The Rapporteurs indicated support for Amicus to request Accelerated Assessment1 in view of the migalastat data and the major public health interest that exists for Fabry patients
  • Based on this feedback and other positive comments from the EU Regulatory officials, Amicus is accelerating the timing of the MAA submission for full approval for migalastat monotherapy from the middle of 2015 to the second quarter of 2015.
Type C Meeting with U.S. Food and Drug Administration (FDA) -- Amicus held a Type C meeting with the FDA earlier this week to review results from both Phase 3 Fabry clinical studies (Study 011 and Study 012) and to discuss the U.S. approval pathway for migalastat. Amicus and FDA officials discussed the migalastat monotherapy data, multiple potential approvable endpoints, and a path toward NDA submission under Subpart H. Dr. Barth also led the Amicus delegation for this meeting which also included one of the world's foremost Fabry nephrology experts. The key takeaways from the meeting include:
  • Amicus plans to submit an NDA for Accelerated Approval (Subpart H), which is only available to therapies for severe and life-threatening conditions that address significant unmet medical needs2
  • For Accelerated Approval, FDA is open to considering several potential surrogate endpoints evaluated in the clinical studies of migalastat, including:
  • Substrate reduction (interstitial capillary GL-3)
  • Reduction in cardiac mass (left ventricular mass index, LVMi)
  • Stabilization of kidney function (glomerular filtration rate)
A post-approval (Phase 4) confirmatory study is required under Subpart H, and several potential protocol designs were discussed
Amicus plans to schedule a pre-NDA meeting and to submit an NDA under Subpart H in the second half of 2015
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "We are very pleased with these recent regulatory interactions. These regulatory discussions demonstrate that health officials in both the U.S. and EU recognize the continued life threatening medical challenges that people living with Fabry face each day. It has been a stellar example of industry and regulators working together to evaluate data and to determine the best and fastest ways in which to bring novel therapies to people in need, especially in such a rare and devastating disease like Fabry. The Amicus team is working tirelessly to submit the highest quality submission to the EU in the next quarter and to the United States in the second half of this year. This is another great step forward for people living with Fabry and the potential for a new precision medicine to treat their disease."
Amicus previously reported positive Phase 3 data in both treatment naïve (Study 011, or FACETS) and enzyme replacement therapy (ERT) switch patients (Study 012, or ATTRACT). Results from these studies have shown that treatment with migalastat has resulted in reductions in disease substrate, stability of kidney function, reduction in cardiac mass, and a positive impact on patient-reported outcomes in patients with amenable mutations.]
 
Also Thursday:
 
Advaxis, Inc. (Nasdaq:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, announced that the first patient was dosed in a Phase 1/2 clinical trial evaluating higher doses and repeat cycles of ADXS-HPV (ADXS11-001), an investigational Lm-LLO immunotherapy, for the treatment of patients with recurrent cervical cancer.
Aldeyra Therapeutics, Inc. (Nasdaq:ALDX), a biotechnology company focused on the development of products to treat diseases related to free aldehydes, today announced its financial results for the full year ended December 31, 2014.
Anavex Life Sciences Corp. (OTCQX:AVXL), a clinical-stage biopharmaceutical company developing drug candidates to treat Alzheimer's and other central nervous system (CNS) diseases, pain and various types of cancer, is pleased to announce the upcoming presentation of new data for two of its Alzheimer's drug candidates, ANAVEX 2-73 and ANAVEX 3-71, at the 12th International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD 2015).
Assembly Biosciences, Inc. (Nasdaq:ASMB) announced today the pricing of an underwritten public offering of $75 million, or 5,555,555 shares of its common stock pursuant to an effective shelf registration statement that was previously filed with the Securities and Exchange Commission at a public offering price of $13.50 per share, before underwriting discounts. 
Athersys, Inc. (Nasdaq:ATHX) announced today that it will be participating in the 12th annual BIO Asia International Conference to be held on March 24-25, 2015 at the Grand Hyatt, Tokyo, Japan. Athersys is additionally a conference sponsor.
Auris Medical Holding AG (Nasdaq:EARS) today provided an update on the Company's business and announced financial results for the fourth quarter and financial year ended December 31, 2014.
 
Black Book Rankings™ named Allscripts (Nasdaq:MDRX) Sunrise platform as the top overall inpatient electronic health record (EHR) for large hospitals and academic medical centers in 2015. 
Cara Therapeutics, Inc. (Nasdaq:CARA), a biotechnology company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced that the Company will host a conference call and live audio webcast on Thursday, March 26, 2015, at 4:30 p.m. ET to report fourth quarter and full year 2014 financial results and provide a corporate update.
Cara Therapeutics, Inc. (Nasdaq:CARA), a biotechnology company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced that it is sponsoring and participating in a breakfast symposium titled "Old problems, new solutions. 
CombiMatrix Corporation (Nasdaq:CBMX), a molecular diagnostics company specializing in DNA-based testing services for pre- and postnatal developmental disorders and pre-implantation genetic screening, today announced it has entered into a contractual agreement with InterWest Health, LLC ("InterWest") for coverage of CombiMatrix's suite of diagnostic laboratory services. 
Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on cancer, today announced the appointment of Lewis W. Coleman to the company's board of directors.
Innocoll AG (Nasdaq:INNL), a global, commercial-stage, specialty pharmaceutical company that develops and manufactures a range of pharmaceutical products and medical devices using its proprietary collagen-based technologies, today announced financial and operating results for the three months and full year ended December 31, 2014.
The Joint Corp. (Nasdaq:JYNT), a national healthcare operator and franchisor of chiropractic clinics, today reported financial results for the fourth quarter and full year ended December 31, 2014.
Metabolix, Inc. (Nasdaq:MBLX), an advanced biomaterials company focused on sustainable solutions for the plastics industry, today reported financial results for the three months and full year ended December 31, 2014.
Minerva Neurosciences, Inc. (Nasdaq:NERV), a clinical-stage biopharmaceutical company focused on the development of innovative therapies to treat Central Nervous System (CNS) diseases and disorders, today announced that it will release fourth quarter and full year 2014 financial results after market close on Thursday, March 26, 2015. 
Pazoo, Inc. (OTCQB:PZOO) (German WKN#: A1J3DK) is pleased to report that MA & Associates, LLC general contractor, Ryan Taylor of All Pro Construction, just completed filming the 100th episode of Kitchen Crashers to be aired in the near future. Ryan Taylor's company, All Pro Construction, is the general contractor handling the build out of Pazoo's 40% owned subsidiary MA & Associates, LLC's medical marijuana testing laboratory in Las Vegas. Construction is expected be complete by mid-April of this year. 
Prothena Corporation plc (Nasdaq:PRTA), a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs, today announced positive results from a Phase 1 single ascending dose study of PRX002, a monoclonal antibody for the potential treatment of Parkinson's disease and other related synucleinopathies. PRX002 is the focus of a worldwide collaboration between Prothena and Roche.
Response Genetics, Inc. (Nasdaq:RGDX), a company focused on the development and sale of molecular diagnostic tests that help determine a patient's response to cancer therapy, will announce its fourth quarter and year-end financial results for 2014 and give an operational update in a press release to be issued after the market closes on Tuesday, March 31, 2015. 
VBL Therapeutics (Nasdaq:VBLT), a clinical-stage biotechnology company committed to the discovery, development and commercialization of first-in-class treatments for cancer, today announced that it will host a conference call and live audio webcast on Wednesday, March 25, 2015 at 8:30 a.m. ET to report fourth quarter and full year 2014 financial results and discuss recent business activities.
Vital Therapies, Inc. (Nasdaq:VTL), a biotherapeutic company developing ELAD®, a cell-based therapy targeting the treatment of acute liver failure, today announced results for the fourth quarter and fiscal year ended December 31, 2014 and provided a corporate update.
Wellness Center USA, Inc. (OTCQB:WCUI), today announced that it and JMJ Financial ("JMJ") have entered into a Note Termination Agreement terminating the $350,000 Promissory Note dated April 16, 2014 (the "Note") with JMJ. 
Zafgen, Inc. (Nasdaq:ZFGN), a biopharmaceutical company dedicated to significantly improving the health and well-being of patients affected by obesity and complex metabolic disorders, today announced its fourth quarter and full year 2014 financial results.
ZS Pharma, Inc. (Nasdaq:ZSPH), a biopharmaceutical company focused on the development and commercialization of highly selective, non-absorbed drugs to treat renal, cardiovascular, liver and metabolic disorders, today announced that data from Phase 3 trials of ZS-9 (sodium zirconium cyclosilicate), an investigational treatment for hyperkalemia, will be presented at the National Kidney Foundation 2015 Spring Clinical Meetings being held March 25 to 29, 2015 in Dallas, Texas. 



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