Drug Target Review

JUNE 21, 2023

Strikingly, a strong genetic link between target and disease biology is emerging as a predictor for success in clinical trials. 2,3 This is exemplified by drugs that target disease-specific genes or genetically distinct patient subsets which are more likely to succeed in demonstrating efficacy in clinical development.

Disease 111

Disease Clinical Trials Therapies Science 111

The Pharma Data

JANUARY 3, 2021

Nasdaq: INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced that the U.S. Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., BOSTON, Jan.

Clinical Trials 52

Clinical Trials Trials Treatment FDA 52

The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

Disease 40

Disease FDA Treatment Therapies 40

Drug Target Review

SEPTEMBER 26, 2023

1-4 This approach saves time, money, and resources, and ultimately leads to safer medicines going forward to the clinic. CNS-related issues account for nearly a quarter of failures during clinical development, a phase where consequences are high in terms of resources and patient impact. 2017 Jan;155(1):234–47.

Drugs 111

Drugs FDA Disease Animal Testing 111

The Pharma Data

DECEMBER 18, 2020

Roxadustat is also in clinical development for anemia associated with MDS and for chemotherapy-induced anemia. Roxadustat is approved in China, Japan (under the name Evrenzo), and Chile for the treatment of anemia in CKD in non-dialysis dependent (NDD) and dialysis-dependent (DD) adult patients. Am J Kidney Dis. Bikbov B et al.

Disease 52

Disease FDA FDA Approval Treatment 52

The Pharma Data

JUNE 25, 2021

protofibril antibody for the treatment of Alzheimer’s disease (AD). Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. and clinical decline. Eisai serves as the lead in the co-development of lecanemab.

Disease 40

Disease Treatment Therapies FDA 40

The Pharma Data

JANUARY 10, 2021

Based on its leading expertise in antibody, protein and peptide technologies, MorphoSys, together with its partners, has developed and contributed to the development of more than 100 product candidates, of which 27 are currently in clinical development.

Clinical Trials 52

Clinical Trials Clinical Development Clinical Supply Trials 52

The Pharma Data

JANUARY 3, 2021

Anecdotal feedback from Foralumab-treated patients was positive and suggests that the treatment was well-tolerated. The scientific approaches underlying this clinical study could potentially be effective against SARs, MERS, and all variants of coronaviruses. NEW YORK and LONDON, Jan.

Clinical Trials 40

Clinical Trials Science Treatment Trials 40

The Pharma Data

NOVEMBER 18, 2020

Kai-Uwe Schässburger Director Clinical Development & Medical Affairs +49 (0)151 688 092 41 or + 46 (0)762 386 153 e-mail: kai-uwe.schassburger@neodynamics.com. With around 55,000 cases, it represented 15% of all new cancer cases in 2017. NeoNavia is evaluated at leading clinics in UK, Germany and Sweden.

Hospitals 52

Hospitals Clinical Development Treatment Regulations 52

The Pharma Data

JULY 29, 2021

Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . 42/40 ratio, brain amyloid by PET and treatment Low values of plasma A?42/40

Clinical Trials 52

Clinical Trials Treatment Clinical Development Disease 52

The Pharma Data

APRIL 4, 2022

living with eosinophilic esophagitis who are currently treated, of whom approximately 48,000 have failed multiple treatments. FDA granted Breakthrough Therapy designation to Dupixent for the treatment of patients aged 12 years and older with EoE. If approved, Dupixent would be the first medicine available in the U.S. About Dupixent.

FDA 52

FDA Treatment Disease FDA Approval 52

The Pharma Data

MAY 31, 2022

The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. Food and Drug Administration in 2017.

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

The Pharma Data

MARCH 1, 2022

The mechanism is currently being evaluated for the potential treatment of cognitive impairment and other symptoms associated with a range of neuropsychiatric and neurodegenerative disorders, such as Alzheimer’s disease and major depressive disorder. senior vice president, R&D, chief scientific officer, AbbVie.

Small Molecule 52

Small Molecule Licensing Licensing Disease 52

The Premier Consulting Blog

JULY 9, 2023

Formally, both of these documents apply solely to marketing approval; informally, however, they are often used as general guidelines during clinical development to avoid obstacles that could result in extensive process changes, product reformulation, or safety qualification efforts. evaluating the safety of) impurities and degradants.

Small Molecule 52

Small Molecule DNA Drugs Clinical Development 52

The Pharma Data

APRIL 29, 2022

The data showed an estimated 91% of infants (n=58) treated with Evrysdi were alive after three years of treatment. The Evrysdi-treated infants continued to improve or maintain motor functions, including the ability to swallow, sit without support, stand with support and walk while holding on, between two and three years of treatment.

Treatment 52

Treatment Clinical Trials Trials Clinical Development 52

The Pharma Data

NOVEMBER 11, 2020

McChesney is the founder and chief executive officer of Cloaked Therapeutics and Arbor Therapeutics, through which he has drawn on more than five decades of research in natural products chemistry to identify and develop novel cancer treatments. a consulting and research firm where he acted as the principal until 2017.

Organic Chemistry 52

Organic Chemistry Science Pharmacy Pharmaceuticals 52

The Pharma Data

JANUARY 12, 2021

The roots of Bayer’s platform began in 2014, when the company stepped into the gene therapy space through a collaboration for hemophilia A with Dimension Therapeutics, which was later acquired by Ultragenyx in 2017. An ongoing partnership with Recursion Pharmaceuticals is expected to lead to new development for fibrotic disease treatments.

Therapies 52

Therapies Licensing Licensing Pharmaceuticals 52

The Pharma Data

DECEMBER 3, 2020

Monti is a pioneer in the research and development of neuroactive steroids known as pherines, which includes VistaGen’s intranasal treatment candidate PH94B for anxiety disorders and PH10 for depression. Zai Lab – Alan Sandler was appointed to the newly created role of president and head of Global Development in Oncology.

Pharmaceuticals 52

Pharmaceuticals Clinical Development Medical Schools Pharmacy 52

The Pharma Data

DECEMBER 17, 2020

Cadavid will be responsible for the strategy, direction and execution of the company’s clinical development programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinical development group.

Small Molecule 52

Small Molecule Engineering Clinical Development Pharmaceuticals 52

The Pharma Data

JANUARY 25, 2021

The co-primary endpoints for the RAISE trial are (1) proportion of patients with SE cessation within 30 minutes of treatment initiation without other medications for the treatment of SE, and (2) proportion of patients with no progression to IV anesthesia for 36 hours following treatment initiation. About Status Epilepticus (SE).

Clinical Trials 40

Clinical Trials Trials Hospitals Pharmaceutical Companies 40

The Pharma Data

JUNE 30, 2021

Food and Drug Administration (FDA) whereby the trial design, clinical endpoints and statistical analysis were agreed in advance with the Agency. Safety results from the study were consistent with or lower than the known safety profile of Yescarta for the treatment of LBCL in the third-line setting.

Therapies 52

Therapies Treatment Virus Vaccine 52

The Pharma Data

APRIL 1, 2021

Food and Drug Administration (FDA) for Tecartus ® (brexucabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). In 2017, Tecartus was granted Breakthrough Therapy Designation by the FDA for relapsed or refractory adult B-cell precursor ALL.

Treatment 52

Treatment Virus Therapies FDA 52

The Pharma Data

DECEMBER 8, 2020

Now poised to advance a robust therapeutics pipeline to clinical development, Nuance will use the funds for ongoing R&D of existing products and business development of potential new assets. Sigilon Therapeutics . Now live on the ticker, Sigilon hopes to sell 7 million shares for a $126 million raise. Noema Pharma .

RNA 52

RNA Therapies Disease Protein Expression 52

Metabolite Tales Blog

JANUARY 23, 2024

Patented active metabolites Gepirone’s metabolites have been patented for “the manufacture of a medicament for the treatment of psychological disorders” [9]. Veozah (Fezolinetant): A Promising Non-Hormonal Treatment for Vasomotor Symptoms in Menopause. link] [5] FDA prescribing information for fezolinetant. Health Sci Rep. 6(10):e1610.

Small Molecule 52

Small Molecule FDA Approval FDA Pharmacokinetics 52

The Pharma Data

DECEMBER 15, 2020

Treatment induced sustainable clinical responses and reduced systemic inflammation. Daratumumab already is approved for the treatment of multiple myeloma. The company is developing its platform for the treatment of chronic, inflammation-mediated autoimmune diseases, and is initially focused on the treatment of RA.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

The Pharma Data

NOVEMBER 10, 2020

We believe the scientific rationale for oral treatment with Foralumab is logical to facilitate topical action at the inflamed sites in the gastrointestinal tract. 2017) Oral treatment with foralumab, a fully human anti-CD3 monoclonal antibody, prevents skin xenograft rejection in humanized mice. Clin Dev Immunol 2012, 425021.

Clinical Trials 40

Clinical Trials Science Disease Trials 40

The Pharma Data

JULY 21, 2021

Positive top-line results have already been reported for two Phase 2 clinical trials of VLA15 in over 800 healthy adults. VLA15 is the only active Lyme disease vaccine candidate in clinical development today, and covers six serotypes that are prevalent in North America and Europe. About VLA15.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

Advarra

AUGUST 10, 2023

Most rare diseases do not have treatments. The Orphan Drug Act creates incentives for developing orphan drugs to treat such diseases. FDA CBER began issuing approvals for gene therapies in 2015 and issued the first approval for a rare disease gene therapy in 2017, with the second rare disease approval issued in 2019.

Drug Development 52

Drug Development Disease Therapies Engineering 52

The Pharma Data

NOVEMBER 1, 2020

A patent application was filed in July 202 0 to protect the potential use of nasally administered Foralumab for the treatment of COVID-19 either alone or in combination with other anti-viral drugs. Foralumab is the only fully human anti-CD3 monoclonal antibody (mAb) in clinical development in the world.

Clinical Trials 40

Clinical Trials Trials Medical Schools Hospitals 40

The Pharma Data

NOVEMBER 8, 2020

In 2017 we bet on refocusing our strategy on medical dermatology to best address patients’ unmet needs. All Almirall’s production sites continue to operate at full capacity in order to be able to ensure the supply of medicines to all patients requiring treatment. In key markets such as Germany , Ilumetri ® gained c.

Licensing 52

Licensing Licensing Production Treatment 52

The Pharma Data

JULY 23, 2021

Opdivo was first granted this indication in 2017 under the FDA’s accelerated approval program, making it the first immunotherapy agent to be approved for use in this setting. This included a meeting of the Oncologic Drugs Advisory Committee in April and subsequent discussion with the FDA.

Treatment 52

Treatment Trials Therapies Disease 52

The Pharma Data

MARCH 11, 2021

development lead, gastrointestinal cancers, Bristol Myers Squibb. “In In HCC, despite evolution of the treatment landscape over the past few years, we believe Opdivo continues to address an unmet medical need for patients in the post-sorafenib setting, and we appreciate the opportunity to discuss this in more depth with the Committee.”.

Treatment 52

Treatment Trials Therapies FDA Approval 52

The Pharma Data

NOVEMBER 5, 2020

has been appointed to the additional role of president and will oversee the growth of the company’s technology portfolio for the treatment of ischemic and hemorrhagic stroke. 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

OCTOBER 28, 2020

NASDAQ: RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. The UBE3A antisense transcript targeted by GTX-102 is a viable target for treatment,” stated Scott Stromatt, M.D.,

Protein Expression 40

Protein Expression Therapies Treatment Disease 40

The Pharma Data

NOVEMBER 11, 2020

Submitted CTA for INZ-701 for the treatment of ENPP1 deficiency to United Kingdom regulatory agency. Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. . BOSTON, Nov.

Clinical Trials 40

Clinical Trials Trials Disease FDA 40

The Pharma Data

DECEMBER 21, 2020

Since 2017, Captor has raised approximately €40 million from private investors and non-dilutive funding sources, allowing it to create an advanced TPD platform and establish five pipeline programs in cancer and autoimmune diseases. ” About Sosei Heptares.

Targeted Protein Degradation 40

Targeted Protein Degradation Small Molecule Structure Based Drug Design (SBDD) Drugs 40

The Pharma Data

NOVEMBER 15, 2020

Most recently, she was a senior executive at Celgene Corporation, where she led corporate strategy from 2012 to 2017. Prior to Celgene, she held leadership roles in marketing, sales and new business development from 2002 to 2011 at Johnson & Johnson. She earned a BA from Durham University, U.K., Before that, Ms. About MultiStem ®.

Clinical Trials 40

Clinical Trials Trials Production Therapies 40