Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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The Pharma Data

OCTOBER 26, 2020

27, 2020 /PRNewswire/ — Synlogic, Inc. Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced the Company will release its third quarter 2020 financial results before the market opens on Thursday, November 5, 2020. CAMBRIDGE, Mass. ,

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The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

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The Pharma Data

MAY 8, 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. About aflibercept. source Link.

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The Pharma Data

DECEMBER 21, 2020

22, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK), 22, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK), UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. SUZHOU, China and ROCKVILLE, Md. , Ascentage Pharma (6855.HK)

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The Pharma Data

DECEMBER 10, 2020

GREEN BROOK, NJ, December 10, 2020 /24-7PressRelease/ — Gianluca Pirozzi, MD, PhD, has been included in Marquis Who’s Who. Dr Pirozzi subsequently was inspired to pursue a career in rare diseases after his daughter Valentina was diagnosed with a rare chromosomal syndrome, called Smith-Magenis Syndrome.

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The Pharma Data

NOVEMBER 10, 2020

Growing into a biopharmaceutical company, Innovent has built an integrated platform covering from drug discovery, CMC, clinical development and commercialization. Innovent 2020 Virtual R&D day was successfully held with a lot of attention from domestic and overseas investors. SAN FRANCISCO and SUZHOU, China , Nov.

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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).

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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. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

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The Pharma Data

MARCH 8, 2021

Valneva SE (“Valneva”), a specialty vaccine company focused on prevention of diseases with major unmet needs, and Pfizer Inc. As announced in December 2020 1 , VLA15-221 is a randomized, observer-blind, placebo-controlled Phase 2 study. We are excited to be part of the Lyme disease vaccine development program with Valneva.

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The Pharma Data

SEPTEMBER 16, 2020

16, 2020 /PRNewswire/ — Amgen (NASDAQ:AMGN) announced today that data from its oncology pipeline in solid tumors will be presented during the European Society of Medical Oncology (ESMO) Virtual Congress 2020, Sept. 19-21, 2020. 20, 2020 , from 2:25 p.m. – 21, 2020 , from 2:25 p.m. – 20, 2020, at?11:00

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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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The Pharma Data

NOVEMBER 10, 2020

The NCT03763149 study is a phase 1a clinical study evaluating the tolerability, safety, and PK/PD properties of IBI188 (letaplimab) as monotherapy for advanced malignancies that failed in standard treatments. . SAN FRANCISCO , U.S. and SUZHOU, China , Nov. A total of 20 subjects were enrolled in this study.

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The Pharma Data

OCTOBER 28, 2020

SIGNAL Phase 2 trial data continue to support cognitive benefit and reduced brain atrophy with pepinemab treatment in Huntington’s disease. Data provide strong rationale for continued development in Huntington’s and other slowly progressive neurodegenerative diseases, including Alzheimer’s. ROCHESTER, N.Y., Vaccinex, Inc.

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The Pharma Data

NOVEMBER 30, 2020

Kunwar Shailubhai will host a conference call on Wednesday, 2 December 2020 at 4:15 p.m. Dr. Shailubhai will provide updates on the Company’s proposed move from AIM to the Standard Segment of the Main Market of the London Stock Exchange, ongoing clinical trials, as well as other recent developments in Tiziana Life Sciences.

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The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

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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. Anaemia is particularly prevalent in patients with CKD.

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The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. Valneva and Pfizer entered into a collaboration agreement in April 2020 to co-develop VLA15 2.

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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. About aflibercept.

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The Pharma Data

DECEMBER 29, 2020

30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. an international research-focused healthcare Group (Chiesi Group), today announced final study results from the BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease. mL/min/ 1.73m 2 /year to -1.73

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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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).

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The Pharma Data

NOVEMBER 4, 2020

5, 2020 /PRNewswire/ — Alligator Bioscience (Nasdaq Stockholm: ATORX) today announced that its CD40 targeting antibody mitazalimab will be presented at the World Immunotherapy Congress, November 2-6, 2020. In addition, clinical biomarker data confirm proof of mechanism in patients. CET on November 5, 2020.

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NOVEMBER 11, 2020

. Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. 12, 2020 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. BOSTON, Nov.

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LifeSciVC

JANUARY 16, 2024

In lean times such as 2023, we saw a shift towards several “hot” areas driven by massive commercial potential, clinically (± commercially) validated mechanisms, and exits that encouraged further investing, even at the early stage. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

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The Pharma Data

DECEMBER 14, 2020

14, 2020 (GLOBE NEWSWIRE) — TG Therapeutics, Inc. NASDAQ: TGTX), a biopharmaceutical company developing medicines for patients with B-cell mediated diseases (“the Company”), today announced the pricing of an underwritten public offering of 6,320,000 shares of common stock at a public offering price of $43.50 NEW YORK, Dec.

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The Pharma Data

OCTOBER 22, 2020

The closing of the offering is expected to occur on or about October 27, 2020, subject to the satisfaction of customary closing conditions. Iterum Therapeutics is advancing its first compound, sulopenem, a novel penem anti-infective compound, in Phase 3 clinical development with an oral formulation and IV formulation.

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The Pharma Data

DECEMBER 17, 2020

The trial remains on-track to complete enrollment of all 500 patients in the United States and Australia before the end of 2020, despite the worsening of the COVID-19 pandemic. In its Phase II clinical trial, TLC599 significantly reduced pain at and through every scheduled visit through six months. SOUTH SAN FRANCISCO, Calif.

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The Pharma Data

OCTOBER 21, 2020

22, 2020 /PRNewswire/ — Synlogic, Inc. 22-25, 2020. 22-25, 2020. Title: Development of a Synthetic Biotic, SYNB8802, for the Treatment of Enteric Hyperoxaluria Poster #: PO0650. CAMBRIDGE, Mass. ,

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The Pharma Data

JULY 27, 2021

AbbVie (NYSE: ABBV) and Calico Life Sciences today announced an extension of their leading-edge collaboration to discover, develop and bring to market new therapies for patients with age-related diseases, including neurodegeneration and cancer. This is the second extension of the collaboration originally established in 2014.

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The Pharma Data

OCTOBER 18, 2020

19, 2020 06:00 UTC. ZURICH, Switzerland–( BUSINESS WIRE )– Versantis AG , a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. Versantis is a clinical-stage biotech company focused on the development of orphan drugs in liver and pediatric diseases.

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SEPTEMBER 16, 2020

New integrated analyses from our tumour agnostic Rozlytrek ® (entrectinib) clinical development programme. Roche’s Chief Medical Officer and Head of Global Product Development. “As Roche highlights featured at ESMO Virtual Congress 2020. Join this event on Roche’s LinkedIn page here.

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The Pharma Data

NOVEMBER 5, 2020

5, 2020 /PRNewswire/ — Third quarter 2020 revenues increased 32% to $2.29 Third quarter 2020 EYLEA ® U.S. Third quarter 2020 Dupixent ® global net sales (2) , which are recorded by Sanofi, increased 69% to $1.07 Third quarter 2020 Dupixent ® global net sales (2) , which are recorded by Sanofi, increased 69% to $1.07

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OCTOBER 26, 2020

27, 2020 (GLOBE NEWSWIRE) — Prevail Therapeutics Inc. Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. NEW YORK, Oct. Founder and Chief Executive Officer of Prevail. “FDA

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OCTOBER 29, 2020

30, 2020 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ: INCY) announced today new data for baricitinib (marketed as OLUMIANT ® ) will be presented at the annual Fall Clinical Dermatology meeting taking place virtually October 29-November 1, 2020. INDIANAPOLIS , Oct.

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DECEMBER 13, 2020

2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development.

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