The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc. .

Therapies 52

Therapies Virus Disease Licensing 52

The Pharma Data

DECEMBER 21, 2020

Legend will receive $75 million from J&J as a clinical development-based milestone payment, per a 2017 collaboration pact. Cilta-cel also won a Breakthrough Therapy designation in China in August.

Therapies 40

Therapies Licensing Licensing Clinical Development 40

The Pharma Data

MARCH 5, 2021

Biogen (Nasdaq: BIIB) today announced it will host webcasts of its pre-recorded presentations and live discussions related to its Alzheimer’s disease investigational therapy, aducanumab, at the upcoming AD/PDTM 2021 Virtual Conference. Biogen licensed aducanumab from Neurimmune under a collaborative development and license agreement.

Licensing 52

Licensing Licensing Biosimilars Disease 52

The Pharma Data

DECEMBER 17, 2020

Partnering with Merck, a world leader in immuno-oncology, provides us with important expertise and resources in developing next-generation T cell engager therapies that will make immunotherapy work for more cancer patients.”. Janux, which was founded in 2017, is still largely a preclinical company. million per target, the company said.

Immune Response 52

Immune Response Pharmacokinetics Engineering Licensing 52

The Pharma Data

MARCH 2, 2021

Boehringer Ingelheim and Gubra today announced a new research and licensing agreement focused on the identification and validation of targets and innovative peptide compounds for the treatment of obesity. Our mission is to create breakthrough therapies that change lives. Financial terms of the new agreement are not disclosed.

Treatment 52

Treatment Bioinformatics Clinical Development Therapies 52

The Pharma Data

MARCH 22, 2021

Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. In December 2017, Roche licensed the investigational molecule from Ionis Pharmaceuticals. About Roche in Neuroscience.

Disease 52

Disease Clinical Trials Pharmacokinetics Treatment 52

The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. CEO and founder Min Li knows a thing or two from his time running GSK’s neuro R&D group before the company shuttered the program in 2017.

RNA 52

RNA Therapies Disease Protein Expression 52

The Pharma Data

MARCH 1, 2022

“There is a major unmet need for new therapies that can help improve cognitive function in patients suffering from difficult-to-treat neurologic diseases,” said Tom Hudson, M.D., The lead molecule, SDI-118, was discovered by UCB before being out-licensed to Syndesi as of 2018. About Syndesi Therapeutics.

Small Molecule 52

Small Molecule Licensing Licensing Disease 52

New Drug Approvals

OCTOBER 24, 2023

“Etrasimod as induction and maintenance therapy for ulcerative colitis (ELEVATE): two randomised, double-blind, placebo-controlled, phase 3 studies” Lancet. Peyrin-Biroulet L, Christopher R, Behan D, Lassen C (May 2017). Retrieved 13 October 2023. ^ [link] ^ World Health Organization (2017). 401 (10383): 1132–1133.

FDA 57

FDA FDA Approval International Pharmacokinetics 57

The Pharma Data

JANUARY 10, 2021

About MorphoSys MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for patients suffering from cancer and autoimmune diseases. In 2017, Tremfya (R) , developed by Janssen Research & Development, LLC and marketed by Janssen Biotech, Inc.,

Clinical Trials 52

Clinical Trials Clinical Development Clinical Supply Trials 52

The Pharma Data

DECEMBER 30, 2020

NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced positive results from the long-term, open-label Phase 3 MOVEMENT trial of AXS-07, Axsome’s novel, oral, multi-mechanistic investigational medicine in the acute treatment of migraine. Ann Neurol.

Trials 52

Trials Treatment Clinical Trials Therapies 52

The Pharma Data

JANUARY 5, 2021

PWS is an orphan disease with major endocrine and behavioral manifestations and no FDA-approved therapies for the treatment of hyperphagia. RAD011 was granted Fast Track Designation in 2017 and Orphan Drug Designation in August 2020 for the treatment of hyperphagia behavior and weight loss in patients with PWS. Disease Highlights.

Disease 52

Disease FDA Approval Clinical Trials Trials 52

Agency IQ

AUGUST 16, 2024

However, the agency can describe a concomitant therapy in labeling for a product it has authority to regulate if it is essential for the therapeutic effect. While the ability to describe and specify aspects of the therapy is limited, the agency can specify the number and licensure of therapists who would participate in the MDMA treatment.

FDA 40

FDA Trials Therapies Regulations 40

The Pharma Data

NOVEMBER 30, 2020

I n-licensing of four clinical-stage product candidates to treat orphan neurological disorders. From the outset, Noema has been leveraging the latest scientific discoveries in neuroscience to identify and pursue promising new indications, and to elaborate a strategy around the four product candidates it licensed from Roche.

Licensing 40

Licensing Licensing Packaging Production 40

The Pharma Data

DECEMBER 15, 2020

The clinical responses were sustained by maintenance therapy with belimumab, an antibody to B-cell activating factor. In October, Johnson & Johnson successfully completed its acquisition of Momenta Pharmaceuticals , which develops therapies for immune-mediated diseases, in an all cash transaction for approximately $6.5

Disease 52

Disease Clinical Trials Small Molecule Trials 52

The Premier Consulting Blog

JANUARY 17, 2023

However, the FDA Reauthorization Act of 2017 (FDARA) amended PREA to require timely pediatric investigations of certain new active ingredients intended for the treatment of adult cancers, even if the indication does not occur in children and/or orphan designation has been granted.

Production 52

Production Clinical Trials FDA Disease 52

The Pharma Data

JANUARY 3, 2021

We are initially focused on developing a novel therapy to treat ENPP1 and ABCC6 deficiencies. Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. Demetrios Braddock, M.D.,

Clinical Trials 52

Clinical Trials Trials Treatment FDA 52

FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

FDA-2017-E-5106 ), and concerns FDA’s October 19, 2016 Accelerated Approval of BLA 761038 for LARTRUVO (olaratumab). 156 or case law that would support extension of the ‘929 patent that claims the product despite revocation of the biologics license application. 8,128,929 [] for [PTE] request under 35 U.S.C. §

FDA 59

FDA Production FDA Approval Licensing 59

The Pharma Data

AUGUST 5, 2020

Ofatumumab is a targeted B-cell therapy that delivers superior efficacy with a similar safety profile when compared with teriflunomide, a commonly prescribed oral treatment for multiple sclerosis 1 . Ofatumumab was originally developed by Genmab and licensed to GlaxoSmithKline. 7. Savelieva M, Kahn J, Bagger M, et al.

Trials 52

Trials Treatment Disease Therapies 52

The Pharma Data

NOVEMBER 8, 2020

R&D pipeline continues to drive future value through innovation and differentiated therapies including expected upcoming approval of Tirbanibulin. In 2017 we bet on refocusing our strategy on medical dermatology to best address patients’ unmet needs. Financial highlights (€ million). .

Licensing 52

Licensing Licensing Marketing Production 52

The Pharma Data

NOVEMBER 5, 2020

Velders is presently chairman of HollandBIO, the association of Dutch biotech companies, and provides years of experience, specifically in the cell and gene therapy areas. Taveras will lead all research and non-clinical development functions supporting the company’s pipeline of investigational therapies.

Analytical Chemistry 40

Analytical Chemistry Pharmaceuticals Small Molecule Therapies 40

The Pharma Data

NOVEMBER 9, 2020

Food and Drug Administration Breakthrough Therapy Designation granted to tezepelumab in Sept. Amgen Inflammation Amgen brings therapies to millions of people with inflammatory diseases, with a focus on serving unmet patient needs. In doing so, we have introduced and evolved novel therapies that have changed the lives of patients.

Trials 52

Trials Production Clinical Trials Government 52

Agency IQ

JANUARY 12, 2024

These windows depend on whether the new drug application (NDA) or biologics license application (BLA) is a new molecular entity, and if the application has received standard review (10 months, plus 2 months of administrative time) or priority review (6 months, plus 2 months of administrative time). before any other country.

FDA 40

FDA Drugs Production Licensing 40

The Pharma Data

NOVEMBER 15, 2020

This prestigious group of professionals will help lead the Company as it prepares for potential commercialization of its investigational cell therapy, MultiStem ®. Most recently, she was a senior executive at Celgene Corporation, where she led corporate strategy from 2012 to 2017. She earned a BA from Durham University, U.K.,

Clinical Trials 40

Clinical Trials Trials Production Therapies 40

The Pharma Data

DECEMBER 21, 2020

In September 2018 , the US Food and Drug Administration granted Breakthrough Therapy Designation for tezepelumab in patients with severe asthma, without an eosinophilic phenotype. Amgen Inflammation Amgen brings therapies to millions of people with inflammatory diseases, with a focus on serving unmet patient needs.

Trials 40

Trials Production Clinical Trials Therapies 40

The Pharma Data

DECEMBER 15, 2020

A total of 43 patients initially diagnosed with unresectable stage IV metastatic PDAC, who had progressed following first-line gemcitabine-based therapy, were enrolled in the triple combination arm. Disease control rate (DCR). 63.2%. 29-52% 2,4.

Clinical Trials 40

Clinical Trials Trials Treatment Therapies 40

The Pharma Data

OCTOBER 18, 2020

David’s proven track record of success and deep experience with the launch and growth of new oncology therapies will play an important role in the success of our commercial strategy and team. “We are excited to welcome David to the AVEO team as we continue to prepare for the potential commercial launch of tivozanib in the U.S.,”

Clinical Trials 40

Clinical Trials FDA Treatment Trials 40

Agency IQ

MARCH 25, 2024

As explained in a 2013 Government Accountability Office report, the intent of the CPG was to “identify those circumstances under which the agency believed establishments with retail pharmacy licenses were engaged in ‘manufacturing, distributing, and promoting unapproved new drugs’ in a manner outside the traditional pharmacy practice of compounding.”

FDA 40

FDA Pharmacy Drugs Production 40

The Pharma Data

OCTOBER 28, 2020

There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. About Ultragenyx.

Protein Expression 40

Protein Expression Therapies Treatment Disease 40

The Pharma Data

NOVEMBER 11, 2020

Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a biologics license application (BLA) for a rare pediatric disease product application may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product.

Clinical Trials 40

Clinical Trials Trials Disease FDA 40

Agency IQ

AUGUST 2, 2024

September 30, 2024 PDUFA Novel Approaches to Development of Cell and Gene Therapy Convene a public meeting to solicit input on methods and approaches (e.g., use of RWE, registries) for capturing post-approval safety and efficacy data for cell and gene therapy products.

FDA 40

FDA Science Regulations Biosimilars 40

The Pharma Data

OCTOBER 27, 2020

License-related fees (5). License-related fees (5). Medtronic), the premarket approval application (PMA) for the ISR was approved by the FDA in December 2017. Three Months Ended September 30, . 2020.

FDA 40

FDA Production Licensing Licensing 40

The Pharma Data

JANUARY 12, 2021

Marianne De Backer, Head of Business Development & Licensing in Bayer’s Pharmaceuticals Division, pictured above. In an interview with BioSpace, De Backer outlined Bayer’s thought process for opening its purse strings and diving into the deep end of the cell and gene therapy space. Photo courtesy of Bayer.

Therapies 52

Therapies Licensing Licensing Pharmaceuticals 52

The Pharma Data

NOVEMBER 29, 2020

1 Meanwhile, these MDM2 inhibitors present some challenges, including dose-limiting hematological toxicities, thus the urgent need to develop new generation of MDM2-targeting therapies in the treatment of cancer. 2017, 16 , 101-114. SUZHOU, China and ROCKVILLE, Md. , 29, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK),

Licensing 40

Licensing Licensing Small Molecule Clinical Trials 40

The Pharma Data

AUGUST 21, 2020

RELATED: Roche pens $2B biobucks pact with Innovent Biologics for bispecific and cell therapies. Lilly first licensed Tyvyt from Innovent in 2015. The original deal included rights outside of China, but in 2017, Lilly licensed back those rights while retaining downstream economics, Taylor said. Source link.

Licensing 52

Licensing Licensing FDA Therapies 52

The Pharma Data

MARCH 4, 2021

Under the 2017 collaboration agreement, Takeda received equity in Ovid and was eligible to receive up to $85 million in payments for regulatory milestones, including the initiation of Phase 3 clinical trials. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines.

Treatment 52

Treatment Pharmaceutical Companies Clinical Trials Trials 52

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

Drug Development 52

Drug Development Disease Therapies Drugs 52