The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

FDA Approval 52

FDA Approval Disease FDA Treatment 52

The Pharma Data

OCTOBER 27, 2020

Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021. About Krabbe Disease.

Disease 40

Disease FDA Treatment Clinical Trials 40

The Pharma Data

AUGUST 3, 2021

Breakthrough results in the treatment of a type of heart failure, which is extremely difficult to manage Strong progress in mental health pipeline Good business performance in all areas, despite ongoing impact of COVID-19. The company’s success is based on its rich R&D portfolio with a focus on diseases with high unmet medical need.

Research 52

Research Clinical Trials Treatment Production 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

Disease 52

Disease Trials Treatment Clinical Trials 52

The Pharma Data

JULY 28, 2021

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa, a long-term enzyme replacement therapy for the treatment of people with Pompe disease. Avalglucosidase alfa has been granted Promising Innovative Medicine designation in the U.K.

Disease 52

Disease FDA Therapies Clinical Development 52

The Pharma Data

NOVEMBER 11, 2021

27 kg/m 2 ) with at least one weight-related comorbidity such as high blood pressure, heart disease or type 2 diabetes. The positive CHMP opinion is based on results from the STEP phase 3a clinical trial programme. Wegovy was launched in the US in June 2021 following approval by the U.S. Wegovy is a once-weekly semaglutide 2.4

Treatment 52

Treatment Clinical Trials Disease Trials 52

The Pharma Data

AUGUST 15, 2021

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the New Drug Application (NDA) for roxadustat for the treatment of anaemia of chronic kidney disease (CKD), in both non-dialysis dependent (NDD) and dialysis-dependent (DD) adult patients.

Disease 52

Disease Treatment FDA Clinical Trials 52

The Pharma Data

APRIL 4, 2022

The high rate of unvaccinated people will continue to put a strain on hospitals and healthcare systems around the world, furthering the need for effective treatments for patients hospitalised with COVID-19,” said Levi Garraway, M.D., Chief Medical Officer and Head of Global Product Development, Roche.

Treatment 52

Treatment FDA Approval Clinical Trials FDA 52

The Pharma Data

MAY 2, 2021

Holzkirchen, May 3, 2021 – Sandoz, a Novartis division, today announced progress in the late-stage clinical development program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. Accessed April 2021].

Biosimilars 40

Biosimilars Clinical Development Production Government 40

The Pharma Data

JANUARY 13, 2021

13, 2021 (GLOBE NEWSWIRE) — Harpoon Therapeutics, Inc. NASDAQ: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma.

Treatment 52

Treatment FDA Clinical Trials Trials 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.

Disease 40

Disease Treatment Therapies FDA 40

The Pharma Data

JANUARY 26, 2021

Novel chronic kidney disease (CKD) target from the partnership enters AstraZeneca’s portfolio. CKD is a complex, and debilitating disease which affects around 10% of the world’s population. LONDON , Jan. LONDON , Jan.

Disease 52

Disease Clinical Development Treatment Drug Development 52

The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. The purpose of the?Ph1

Trials 52

Trials Disease Clinical Trials Engineering 52

The Pharma Data

JANUARY 19, 2021

19, 2021 (GLOBE NEWSWIRE) — Fulcrum Therapeutics, Inc. The offering is expected to close on January 22, 2021, subject to customary closing conditions. The company has advanced losmapimod to Phase 2 clinical development for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and Phase 3 for the treatment of COVID-19.

Small Molecule 40

Small Molecule Clinical Development Treatment Engineering 40

The Pharma Data

JANUARY 14, 2021

15, 2021 /PRNewswire/ — Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX) (NASDAQ – CALT) today announced the program for the upcoming virtual R&D Day for investors, analysts and journalists on January 20, 2021 , which will take place between 1pm and 5pm CET. STOCKHOLM , Jan.

Clinical Trials 40

Clinical Trials Hospitals Disease International 40

Drug Discovery World

MARCH 7, 2023

She has managed multinational projects for charities and not-for-profits to find new treatments for Malaria and led Innovate UK grants through to successful completion. Here at FAB our proprietary approach aims to overcome this fundamental challenge to human health as we work to discover and develop new drugs for age-associated diseases.”

International 246

International Science Pharmaceuticals DNA 246

The Pharma Data

DECEMBER 18, 2020

18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).

Disease 52

Disease FDA FDA Approval Science 52

The Pharma Data

AUGUST 19, 2021

Oral presentation at the International Association for the Study of Lung Cancer’s (IASLC) 2021 World Conference on Lung Cancer (WCLC) shows evidence that the bispecific mechanism of action for RYBREVANT TM can provide anti-tumor activity against either EGFR-mutated or MET-mutated non-small cell lung cancer. 1 Treatment-related Grade ?3

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

JUNE 25, 2021

First-Time Disease-Free Survival Data to be Presented During Plenary Session at the 2021 ASCO Annual Meeting. 41.5), KEYTRUDA demonstrated a statistically significant and clinically meaningful reduction in the risk of disease recurrence or death by 32% compared to placebo (HR=0.68 [95% CI, 0.53–0.87]; KENILWORTH, N.J.–(BUSINESS

Disease 52

Disease Therapies Treatment Medical Schools 52

Drug Target Review

AUGUST 24, 2023

We will explore how GDF-15 impacts the tumour microenvironment and hinders the infiltration of T cells into the tumour, as well as the implications of neutralising GDF-15 to reverse its inhibitory effects and sensitise tumours to anti-PD-1 treatment. T-cell infiltration is a prerequisite for responses to checkpoint inhibitors.

Treatment 98

Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

JANUARY 25, 2021

– January 26, 2021) – NervGen Pharma Corp. The AD-SAB will work closely with NervGen as the Company plans its upcoming preclinical studies and clinical trials and in the analyses of the results from these studies. Vancouver, British Columbia–(Newsfile Corp. Dr.

Disease 40

Disease Clinical Development Molecular Biology Treatment 40

The Pharma Data

APRIL 8, 2021

Since its first approval in renal cell carcinoma nearly two years ago, KEYTRUDA has become an important first-line treatment option in combination with axitinib for patients with advanced renal cell carcinoma,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories.

Disease 52

Disease Therapies Research Laboratories Treatment 52

The Pharma Data

NOVEMBER 5, 2021

The data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting. With presently no approved treatments, there’s a major unmet need for curatives that can delay progression to order failure, “ said John Tsai, Head of Global Drug Development and Chief Medical Officer at Novartis.

Disease 52

Disease Pharmacokinetics Treatment FDA 52

The Pharma Data

JANUARY 3, 2021

04, 2021 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. 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. BOSTON, Jan.

Clinical Trials 52

Clinical Trials Trials Treatment FDA 52

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. In 2021, levels of investment hit an all-time high at $22.7 Cell and gene therapies.

Drugs 130

Drugs Therapies Clinical Trials Cell Based Assays 130

Drug Discovery World

JULY 2, 2024

OECD data places Switzerland second in the world for biotechnology R&D intensity 1 , and in 2021, the country had the seventh largest share of the global biotech market 2. The centre currently has 58 pharma/biotech assets that are in pre-clinical development, alongside 29 that are being tested in clinical trials.

Science 147

Science Drugs Clinical Trials Licensing 147

The Pharma Data

JANUARY 15, 2021

–(BUSINESS WIRE) January 15, 2021 — Daiichi Sankyo Company, Ltd. for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. mg/kg, interstitial lung disease occurred in 10% of patients.

Treatment 52

Treatment Trials Disease Therapies 52

The Pharma Data

JANUARY 18, 2021

Potential first-in-class disease-modifying biologic in late-stage clinical studies. Experienced executive development team from Gilead Sciences. 19, 2021 /PRNewswire/ — Vera Therapeutics, Inc. , SOUTH SAN FRANCISCO, Calif. ,

Disease 52

Disease Science Clinical Development Treatment 52

The Pharma Data

OCTOBER 1, 2021

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. Deucravacitinib also demonstrated durable efficacy in scalp psoriasis through 52 weeks of treatment (Poster Number: P1391).

Disease 52

Disease Laboratories Clinical Pharmacology Treatment 52

The Pharma Data

JANUARY 27, 2021

27 , 2021 /PRNewswire/ — Harbour BioMed (HBM) (HKEX: 02142.HK), HK), announced today that China Center for Drug Evaluation (CDE) has granted Breakthrough Therapy designation to Batoclimab (HBM 9161), a fully human anti-FcRn monoclonal antibody (mAb), for the treatment of adult patients with Myasthenia Gravis (MG).

Therapies 52

Therapies Treatment International Licensing 52

The Pharma Data

APRIL 11, 2021

The data were presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, held virtually April 10-15, 2021. In this cohort, the most common treatment-emergent adverse events of any grade (?20%) No patients in this cohort discontinued treatment due to treatment-related adverse events.

Research 52

Research Treatment Therapies Trials 52

The Pharma Data

JUNE 26, 2021

Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA), under Priority Review, for Port Delivery System with ranibizumab (PDS) for the treatment of neovascular or “wet” age-related macular degeneration (nAMD). 4 The FDA is expected to make a decision on approval by 23 October 2021.

Treatment 52

Treatment FDA Therapies Clinical 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

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) . United States and virtually from July 26 to 30, 2021 (Presentation No.:

Clinical Trials 52

Clinical Trials Treatment Clinical Development Disease 52

The Pharma Data

NOVEMBER 3, 2020

Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). Patient enrollment is expected to begin in early 2021. LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

FDA 52

FDA Treatment Clinical Trials Clinical Development 52

Drug Discovery World

JULY 11, 2022

Clinical trial sponsors need to coordinate the delivery of investigational medicinal products to clinical sites and the collection of samples from participants after they receive treatment. There are over 7,000 genetic diseases that could potentially be cured using gene therapy 10. 2021, February 23). Conclusion.

Therapies 130

Therapies Clinical Trials Production Trials 130

The Pharma Data

JUNE 28, 2021

mg, 5 mg, and 10 mg is approved for the treatment of patients with chronic heart failure who are receiving standard treatment for chronic heart failure. Heart failure is a life-altering disease which affects approximately 1.2 Vericiguat is the first sGC stimulator to be approved for the treatment of chronic heart failure.

Clinical Trials 52

Clinical Trials Treatment Disease Production 52