The Pharma Data

JUNE 18, 2025

By combining Neoclease’s AI-designed gene-editing platform with our technologies, regulatory expertise, and process support, we aim to help accelerate new treatments that minimize side effects and maximize effectiveness for patients.”

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The Pharma Data

JUNE 9, 2025

Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients aged seven years and older with narcolepsy. of participants reported treatment-emergent adverse events (TEAEs), all of which were mild or moderate and consistent with previous safety data on Xywav. Approximately 32.8%

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The Pharma Data

JUNE 11, 2025

argenx Unveils Positive Phase 2 Results for Efgartigimod in Myositis and Sjogren’s Disease at EULAR 2025 argenx SE a global immunology company dedicated to advancing treatments for severe autoimmune diseases, presented encouraging new clinical data at the 2025 European Congress of Rheumatology (EULAR), held June 11–14 in Barcelona, Spain.

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The Pharma Data

JUNE 23, 2025

This study explored the effects of beginning treatment with lower starting doses of MariTide to improve tolerability while maintaining clinical benefit. These results were evaluated using the efficacy estimand, a statistical model that accounts for treatment adherence and protocol deviations.

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The Pharma Data

JUNE 12, 2025

Together, these data reflect a significant step forward in the treatment of hematologic malignancies — a group of aggressive, complex disorders for which many patients still face limited treatment options and poor outcomes — and demonstrate the potential for these novel mechanisms of action to make a profound difference in patient care.

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Drug Target Review

JULY 3, 2025

It is becoming increasingly evident that generative artificial intelligence (GenAI) is a resourceful tool for helping pharmaceutical companies reduce manual tasks required by clinical trials. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Highlighting data integration. This is an AI generated image.

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Broad Institute

APRIL 5, 2025

David Liu receives Breakthrough Prize in Life Sciences By Corie Lok April 5, 2025 Breadcrumb Home David Liu receives Breakthrough Prize in Life Sciences Liu is honored for the development of base editing and prime editing, two gene editing technologies transforming medicine.

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The Pharma Data

JUNE 22, 2025

The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The trial is evaluating NXT007 in people with hemophilia A without factor VIII inhibitors who have not previously been treated with Hemlibra.

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The Pharma Data

JUNE 9, 2025

The COACH study is the first clinical investigation to assess the combined effects of two once-weekly treatments—TransCon® CNP (navepegritide) and TransCon® hGH (lonapegsomatropin)—in children with achondroplasia. TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia,” Dr. Shu stated.

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The Pharma Data

JUNE 22, 2025

The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. In the trial, participants were directly switched to Mim8 without a washout period.

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New Drug Approvals

JULY 9, 2025

Sunvozertinib , sold under the brand name 舒沃哲, among others is an anti-cancer medication used for the treatment of non-small-cell lung cancer. [2] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]

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SugarCone Biotech

JUNE 19, 2025

Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. months (versus 20% ORR and 8.3

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The Pharma Data

JUNE 6, 2025

has announced encouraging interim findings from the pivotal Phase 3 VISIONARY trial evaluating sibeprenlimab in adults with Immunoglobulin A nephropathy (IgAN), a rare and progressive autoimmune kidney disorder. Treatment-emergent adverse events (TEAEs) occurred in 76.3% in collaboration with Otsuka Pharmaceutical Co.,

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The Pharma Data

JUNE 25, 2025

In March 2025 , Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinical trial for farabursen. Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD.

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New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] Fitusiran 1711.0g/mol,

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Drug Target Review

JUNE 28, 2025

DARPin therapeutics were a prominent theme at AACR 2025 , with Molecular Partners presenting a trio of preclinical programmes spanning radiopharmaceuticals and immune cell engagers. ” With an IND filing and first-in-human trials expected in 2025, MP0712 is moving steadily towards clinical evaluation.

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The Pharma Data

JUNE 12, 2025

The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.

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Drug Target Review

JULY 8, 2025

mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As The dual approach represents an effort to expand treatment efficacy across a broader subset of tumours, potentially improving outcomes. “In

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The Pharma Data

JUNE 26, 2025

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.

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The Pharma Data

JUNE 11, 2025

The findings were presented as part of a late-breaking abstract session (#LB0001) at the European Alliance of Associations for Rheumatology (EULAR) Congress 2025 , held June 11–14 in Barcelona, Spain. No new safety signals emerged, and the treatment continued to be well tolerated. of patients achieving an ACR20 response versus 39.4%

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The Pharma Data

JUNE 12, 2025

The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.

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The Pharma Data

JUNE 13, 2025

The data were presented during the 2025 European Hematology Association (EHA) Congress (Abstract #S239). Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Director of Lymphoma Research at Sarah Cannon Research Institute and principal study investigator for the trial.

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The Pharma Data

JUNE 9, 2025

A New Treatment Option for a Debilitating Joint Tumor TGCT is a rare, debilitating condition that primarily affects young and middle-aged adults, often striking individuals in their prime working years. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

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SugarCone Biotech

JUNE 27, 2025

A post-ASCO update on tumor targets DLL3, B7H3 and Her3 June 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and Her3 are compelling tumor antigens to target with cancer therapeutics. As we’ll see, ASCO 2025 was a breakout conference for B7-H3-targeted therapies. That trial is in second-line NSCLC.

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New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment.

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Broad Institute

NOVEMBER 25, 2024

Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.

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The Pharma Data

JUNE 6, 2025

The studies shared at ECFS 2025 encompassed a broad range of clinical outcomes, including pulmonary function, exacerbation frequency, nutritional metrics, and health-related quality of life. These included randomized, controlled, and open-label studies in both adolescent and adult populations, as well as in children aged 6 to 11 years.

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The Pharma Data

JUNE 16, 2025

Currently, the medial survival for patients with MZL after multiple lines of treatment falls within the range of 3–5 years, reflecting a significant and persistent need for more effective therapeutic options. The data further highlight liso-cel’s superiority over standard-of-care treatments in this patient population. Source link

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The Pharma Data

JUNE 15, 2025

The data were presented today in a late-breaking oral presentation at the 2025 European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress in Glasgow, UK. This is crucial information for guiding both patients and physicians in their treatment decisions.”

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The Pharma Data

JUNE 11, 2025

Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.

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The Pharma Data

JUNE 6, 2025

Sanofi Highlights Robust Pipeline of Innovations in Rare Blood Disorders at ISTH 2025 Sanofi is poised to take center stage at the 33rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), set to be held in Washington, D.C., from June 21 to 25, 2025. Christopher Corsico, Global Head of Development at Sanofi.

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BioPharma Drive: Drug Pricing

JULY 8, 2025

Published July 8, 2025 Gwendolyn Wu Senior reporter post share post print email license Proteasomes are cellular machines for breaking down proteins. Dupixent, which blocks IL-4, alone brought in more than $4 billion in sales for Sanofi in the first quarter of 2025.

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The Pharma Data

JUNE 15, 2025

The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.

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Alta Sciences

MARCH 4, 2025

Forging a Path Forward for Substance Abuse Potential Studies With the Cross-Company Abuse Liability Council By Beatrice Setnik, PhD pmjackson Tue, 03/04/2025 - 20:10 Many of my colleagues and I are members of the Cross-Company Abuse Liability Council (CCALC), where I have been a co-chair for the past seven years, and a member for 15.

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New Drug Approvals

JUNE 29, 2025

Taletrectinib CAS 1505514-27-1 as salt: 1505515-69-4 , Taletrectinib adipate FDA 6/11/2025, Ibtrozi, To treat locally advanced or metastatic ROS1-positive non-small cell lung cancer AB-106, DS-6051a 405.5 1] Taletrectinib was approved for medical use in the United States in June 2025. [3] April 2025. Retrieved 14 June 2025.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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