Medicines and Healthcare products Regulatory Agency

DGAP-News: Eckert & Ziegler Announces Clinical Trial Notification for Joint Clinical Trial in Japan

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Friday, November 12, 2021
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Eckert & Ziegler (ISIN DE0005659700, TecDAX) announced that the Clinical Trial Notification (CTN) of a joint clinical study with Novartis Pharma K.K.

Key Points: 
  • Eckert & Ziegler (ISIN DE0005659700, TecDAX) announced that the Clinical Trial Notification (CTN) of a joint clinical study with Novartis Pharma K.K.
  • (Japan) was accepted by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for 68Ga-PSMA-11, an investigational radioligand imaging agent and 177Lu-PSMA-617, an investigational radioligand therapy in metastatic prostate cancer.
  • Eckert & Ziegler Radiopharma GmbH is manufacturer of the pharmaceutical 68Ge/68Ga generator GalliaPharm(R), which is used to produce the radiopharmaceutical 68Ga-PSMA-11 for 68Ga PET/CT diagnostics.
  • Eckert & Ziegler Strahlen- und Medizintechnik AG with more than 800 employees is a leading specialist for isotope-related components in nuclear medicine and radiation therapy.

Sio Gene Therapies Announces Successful Manufacture of Three GMP Batches of AXO-Lenti-PD Gene Therapy for Parkinson’s Disease

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Thursday, November 11, 2021
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NEW YORK and DURHAM, N.C., Nov. 11, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today provided a manufacturing and regulatory update for AXO-Lenti-PD, its clinical-stage gene therapy for Parkinson’s disease.

Key Points: 
  • With the successful manufacture of three batches, all of which achieved the target titer and have completed fill and finish, we now have a process that has generated sufficient clinical trial material for future clinical development.
  • The MHRA provided guidance on the:
    Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.
  • All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain.

Axcella Therapeutics Reports Third Quarter Financial Results and Provides Update on Long COVID, OHE and NASH Clinical Trials

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Wednesday, November 10, 2021
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The third quarter of 2021 was a time in which Axcella focused heavily on both execution and expansion, said Bill Hinshaw, President and Chief Executive Officer of Axcella.

Key Points: 
  • The third quarter of 2021 was a time in which Axcella focused heavily on both execution and expansion, said Bill Hinshaw, President and Chief Executive Officer of Axcella.
  • Progressed Screening and Enrollment in EMMPOWER and EMMPACT Clinical Trials: Patient screening and enrollment continues in Axcellas recently initiated EMMPOWER Phase 2 clinical trial in overt hepatic encephalopathy (OHE) and EMMPACT Phase 2b clinical trial in nonalcoholic steatohepatitis (NASH).
  • Published AXA1125 Data in The American Journal of Gastroenterology: The American Journal of Gastroenterology recently published results from Axcellas AXA1125-003 clinical study.
  • The companys unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials.

Dynacure Announces Strategic Collaboration and Option Agreement with Nippon Shinyaku

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Wednesday, November 10, 2021
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Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to pursue the development and commercialization of DYN101 in Japan upon analysis of interim data from the Phase 1/2 study, UNITE-CNM.

Key Points: 
  • Under the terms of the agreement, Nippon Shinyaku has secured opt-in rights to pursue the development and commercialization of DYN101 in Japan upon analysis of interim data from the Phase 1/2 study, UNITE-CNM.
  • "We look forward to leveraging the skills and network Nippon Shinyaku brings and advancing this important clinical program toward commercialization."
  • Nippon Shinyaku, established in 1919, has a long-history as a research-oriented pharmaceutical company in Japan.
  • Areas of specialty for both R&D and marketing by Nippon Shinyaku include hematology, intractable and rare diseases, urology and gynecology.

Rhythm Pharmaceuticals to Present at Stifel 2021 Virtual Healthcare Conference

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Tuesday, November 9, 2021
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Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare genetic diseases of obesity and is leveraging the Rhythm Engine and the largest known obesity DNA database -- now with approximately 37,500 sequencing samples -- to improve the understanding, diagnosis and care of people living with severe obesity due to certain genetic deficiencies. Rhythm’s headquarters is in Boston, MA.

Key Points: 
  • BOSTON, Nov. 09, 2021 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, today announced that David Meeker, M.D., Chair, President and Chief Executive Officer, will present at the Stifel 2021 Virtual Healthcare Conference on Monday, November 15, 2021 at 11:20 a.m.
  • A replay of the webcast will be available on the Rhythm website for 30 days following the presentation.
  • Rhythm is a commercial-stage biopharmaceutical company committed to transforming the treatment paradigm for people living with rare genetic diseases of obesity.
  • IMCIVREE is the first-ever FDA-approved and EC- and MHRA-authorized therapy for patients with these rare genetic diseases of obesity.

Galapagos reports commercial and operational progress at Q3 financial results

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Thursday, November 4, 2021
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Total sales of Jyseleca in Europe by both companies for the first nine months of 2021 is 15.8 million.

Key Points: 
  • Total sales of Jyseleca in Europe by both companies for the first nine months of 2021 is 15.8 million.
  • The decrease in revenue recognition was primarily due to a negative cumulative catch up of revenue triggered by the recent agreement under which Galapagos will assume operational and financial responsibility for the ongoing DIVERSITY clinical study.
  • Galapagos financial report for the first nine months ended 30 September 2021, including details of the unaudited consolidated results, is accessible via www.glpg.com/financial-reports .
  • Galapagos will conduct a conference call open to the public tomorrow, 5 November 2021, at 13:00CET/8AM ET, which will also be webcasted.

Pancreatic Cancer Action Network and CDISC Partnership Develops First Data Standards for World’s Toughest Cancer

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Thursday, November 4, 2021
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The Pancreatic Cancer Action Network (PanCAN) and CDISC have announced the release of a new Therapeutic Area User Guide setting the first-ever global data standards specifically for pancreatic cancer.

Key Points: 
  • The Pancreatic Cancer Action Network (PanCAN) and CDISC have announced the release of a new Therapeutic Area User Guide setting the first-ever global data standards specifically for pancreatic cancer.
  • The project was funded through a two-year grant awarded to CDISC by PanCAN and is designed to lead to greater efficiencies and data sharing among the pancreatic cancer scientific community.
  • Two years in the making, the User Guide for pancreatic cancer is now freely available via the CDISC website.
  • The Pancreatic Cancer Action Network (PanCAN) leads the way in accelerating critical progress for pancreatic cancer patients.

Pancreatic Cancer Action Network And CDISC Partnership Develops First Data Standards For World's Toughest Cancer

Retrieved on: 
Thursday, November 4, 2021
Pancreatic Cancer Action Network, Pancreatic cancer, Research, Volunteering, Disease, Medicines and Healthcare products Regulatory Agency, Global health, Drug development, Survival, PMDA, Partnership, U8 Global Student Partnership for Development, Patient, FDA, NMPA, Pharmaceuticals and Medical Devices Agency, VP, National Medical Products Administration, User guide, Development, CDISC, Doctor of Philosophy, Pharmaceutical industry, Medical device

LOS ANGELES and AUSTIN, Texas, Nov. 4, 2021 /PRNewswire/ -- The Pancreatic Cancer Action Network (PanCAN) and CDISC have announced the release of a new Therapeutic Area User Guide setting the first-ever global data standards specifically for pancreatic cancer.

Key Points: 
  • LOS ANGELES and AUSTIN, Texas, Nov. 4, 2021 /PRNewswire/ -- The Pancreatic Cancer Action Network (PanCAN) and CDISC have announced the release of a new Therapeutic Area User Guide setting the first-ever global data standards specifically for pancreatic cancer.
  • "Through this partnership with CDISC to standardize data submissions to regulatory agencies, we are creating opportunities to streamline drug development in the pancreatic cancer space," said Sudheer Doss, PhD, PanCAN's Chief Business Officer.
  • Two years in the making, the User Guide for pancreatic cancer is now freely available via the CDISC website.
  • The Pancreatic Cancer Action Network (PanCAN) leads the way in accelerating critical progress for pancreatic cancer patients.

Bluehole New Consumption Publishes a Feature Story on Recent Vaping Regulation Updates

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Thursday, November 4, 2021
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Chinas leading vaping media, Bluehole New Consumption, today published a feature story on recent vaping regulation updates in the UK, the US and Malaysia.

Key Points: 
  • Chinas leading vaping media, Bluehole New Consumption, today published a feature story on recent vaping regulation updates in the UK, the US and Malaysia.
  • As vaping accepted by more and more health regulators, the industry will witness a more concentrated and standardized market.
  • As BAT Malaysia states, regulation could positively accelerate healthy development of the vaping industry for setting higher quality requirements and safety standards for products.
  • Meanwhile, the whole industry and the supply chain will benefit from such a well-regulated and standardized market in turn.

GBT Wins Prestigious Prix Galien USA Award for Oxbryta® (voxelotor)

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Monday, November 1, 2021
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SOUTH SAN FRANCISCO, Calif., Nov. 01, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) announced today that Oxbryta® (voxelotor) tablets, a first-in-class oral therapy indicated for the treatment of sickle cell disease (SCD) in patients age 12 years and older in the United States, has been awarded the prestigious 2021 Prix Galien USA Award for Best Biotechnology Product. The Prix Galien award is among the global health industry's most renowned honors, recognizing outstanding product achievements that improve the human condition. Oxbryta was selected among 19 nominees by a committee of 12 leaders from the biotech industry and academia, including five Nobel Laureates.

Key Points: 
  • The Prix Galien award is among the global health industry's most renowned honors, recognizing outstanding product achievements that improve the human condition.
  • Receiving the prestigious Prix Galien Award is a tremendous honor.
  • Oxbryta has previously been recognized with the 2020 National Xconomy Award for Breakthrough Drug of the Year and the 2020 National Organization for Rare Disease (NORD) Rare Impact Award for Industry Innovation.
  • Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD.