Conversations in Drug Development Trends

DECEMBER 20, 2023

Single-Arm Trial Design Single-arm trials have also become an increasingly common development strategy to support regulatory approval and allow patients expedited access to novel therapies, particularly in the accelerated access setting.

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The Pharma Data

NOVEMBER 17, 2021

Biogen moment blazoned that the European Commission (EC) has granted marketing authorization for VUMERITY ® (diroximel fumarate) to treat grown-ups with relapsing- remitting multiple sclerosis (MS). Encyclopedically, an estimated2.8 million people live with MS, with further than 1 million people in Europe living with the complaint. “

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The Pharma Data

JULY 6, 2021

New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. Sanofi’s two marketed extended half-life factor replacement therapies shifted a two-decades-old treatment paradigm when launched in 2014.

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The Pharma Data

MARCH 9, 2022

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi ® ) (STO:SOBI) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ? 12 years of age with severe hemophilia A.

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LifeSciVC

APRIL 24, 2024

with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., Consider anticipated time to market (not just time to the clinic), competitiveness of the mechanism itself and broader competition for the indication at large, and potential impact on patient lives should discovery and development be successful.

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Alta Sciences

OCTOBER 30, 2023

TOP NONCLINICAL SCIENTIFIC RESOURCES eBook : Safety Assessment for Ophthalmic Products Designing preclinical studies for ocular therapies take a lot of deliberation. The Altascientist : Issue No. Read or listen now. TOP MANUFACTURING AND ANALYTICAL RESOURCES The Altascientist : Issue No. Watch it now. The Altascientist : Issue No.

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Drug Target Review

JULY 1, 2024

Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? The global pain management market is projected to reach about $87 billion by 2025, driven by rising chronic diseases, an ageing population and more surgical procedures. South Rampart Pharma, Inc.

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes.

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DrugBank

FEBRUARY 26, 2025

This stagnation has created a perfect storm, threatening the effectiveness of current therapies and limiting the pipeline of new drugs. Advances in pharmacology and drug formulation have enabled researchers to refine these therapies and extend their utility.

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The Pharma Data

MARCH 23, 2021

We have designed PF-07321332 as a potential oral therapy that could be prescribed at the first sign of infection, without requiring that patients are hospitalized or in critical care. At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives.

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Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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The Pharma Data

MARCH 9, 2022

PAXLOVID™ (nirmatrelvir [PF-07321332] tablets and ritonavir tablets) is the first oral therapy specifically designed to combat COVID-19 to be evaluated in a pediatric clinical study PAXLOVID is currently authorized under U.S.

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DrugBank

APRIL 3, 2025

This increases costs and delays the introduction of potentially life-saving therapies. These studies typically include in vitro assays to evaluate cytotoxicity and in vivo models to study pharmacokinetics, pharmacodynamics, and toxicological profiles. Preclinical studies form the backbone of early safety assessment.

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The Pharma Data

DECEMBER 13, 2020

About 15 to 20% of breast cancers cases are HER2+, and antibody-based therapies targeting HER2 have significantly improved outcomes of patients with HER2+ breast cancer. Zai is strongly positioned to take advantage of a growing pharmaceutical market in this region.”. We look forward to working with the FDA to bring ABP 798 to market.”.

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The Pharma Data

APRIL 4, 2023

Biosimilars help patients to gain broader access to effective and high-quality treatments that improve their disease therapies,” said Rebecca Guntern, Head of Region Europe, Sandoz. The study met all its primary objectives, demonstrating comparable pharmacokinetics and showing similar safety and immunogenicity between the two concentrations.

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The Pharma Data

APRIL 7, 2021

Nasdaq: BIIB) today announced that the European Commission (EC) has granted marketing authorization for a subcutaneous (SC) injection of TYSABRI® (natalizumab) to treat relapsing-remitting multiple sclerosis (MS). TYSABRI is a trusted high-efficacy therapy with a well characterized safety profile for patients living with MS.

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The Pharma Data

NOVEMBER 11, 2020

We now have compelling data showing SLV213 has potent antiviral activity against SARS-CoV-2 as well as data on the safety, tolerability, and pharmacokinetics and pharmacodynamics of SLV213 in several preclinical model systems, including nonhuman primates,” said Felix Frueh, Ph.D., Cofounder and Chief Scientific Officer of Selva Therapeutics.

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The Pharma Data

APRIL 7, 2022

“At Pfizer, we have a strong heritage in, and commitment to, fighting infectious diseases, most recently evidenced by our delivery of the first authorized vaccine and oral therapy to combat COVID-19,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. Sisunatovir has been granted Fast Track designation by the U.S.

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The Pharma Data

JUNE 28, 2021

Current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurrence of blood clots with standard anticoagulation therapy which requires injections, dietary restrictions, and regular laboratory monitoring. Xarelto is marketed outside the U.S. Study The randomized, open-label phase III EINSTEIN-Jr.

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The Pharma Data

NOVEMBER 8, 2020

The active ingredient is violaxazine hydrochloride, which has an extensive safety record in Europe, where it was previously marketed as an antidepressant. Celgene acquired it in 2018 from bluebird bio, who originally developed the therapy. SPN-812 is a serotonin norepinephrine modulating agent (SNMA).

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The Pharma Data

DECEMBER 27, 2020

phase I trial is a randomized, double-blind, placebo-controlled, single and multiple dose escalation study to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics (biomarkers – FGF19 and C4) of ASC42 in healthy subjects. Phase I Trial. This trial also investigates the food effect on ASC42 exposure.

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The Pharma Data

FEBRUARY 25, 2022

The study met its primary goal by demonstrating pharmacokinetic equivalence in patients who switched multiple times between treatment with the two medicines. They have the ability to help increase patient access to essential medicines and provide value to the healthcare system by driving market competition that can lower the cost of care.

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The Pharma Data

AUGUST 9, 2021

Since 40% of people with DLBCL relapse after initial therapy, achieving meaningful treatment effects in the front-line setting has the potential to be transformative,” said Levi Garraway, M.D., Safety outcomes were consistent with those seen in previous trials. Roche’s Chief Medical Officer and Head of Global Product Development.

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The Pharma Data

APRIL 27, 2021

Mobocertinib is the first oral therapy specifically designed to selectively target EGFR Exon20 insertion mutations. The Phase 1/2 trial aims to evaluate the safety, pharmacokinetics and anti-tumor activity of oral mobocertinib in patients with non-small cell lung cancer (NSCLC). and around the globe.”. About the Phase 1/2 Trial.

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The Pharma Data

DECEMBER 29, 2020

Of the 22 patients enrolled in the BRIDGE study, the majority of treatment emergent adverse events were mild or moderate in severity, with two patients (9.1%) withdrawing from the therapy due to hypersensitivity reaction that was resolved. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea.

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The Pharma Data

JANUARY 26, 2021

Previous human pharmacokinetic (PK) study showed significantly better PK profile when compared to market leading product. US market for overdose rescue medications. Patent protection until 2039. UPPSALA, Sweden , Jan. Tel: +46 (0)18 780 88 00. About Orexo.

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The Pharma Data

JANUARY 17, 2021

Syngeneic tumor animal models play a critical role because they use standard inbred mice that have a competent immune system, which is required to evaluate immune-modulating therapies. Because the Jh mouse cannot produce ADA, there is no neutralizing or pharmacokinetic impact on the therapeutic or anaphylaxis risk.

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The Pharma Data

AUGUST 19, 2021

3] While MET inhibitors have recently received accelerated approval in this setting in some regions, the vast majority of patients eventually acquire resistance to these therapies, thus underscoring the need for new treatment options. [4] METex14 mutations are found in approximately three percent of patients with NSCLC. [2] 4] , [5] , [6].

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The Pharma Data

APRIL 11, 2023

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients: next-generation Immuno-Oncology, Targeted Radionuclide Therapies and Precision Molecular Oncology. Data from all three areas of scientific focus will be showcased during this year’s meeting.

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The Pharma Data

DECEMBER 9, 2020

The first trial will be conducted as a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and pharmacokinetics of the intravenous liposomal formulation of ibrexafungerp in healthy subjects. The study will be conducted in South Africa. For more information, visit www.scynexis.com.

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The Pharma Data

DECEMBER 23, 2020

Marketing Authorization was based on the pivotal phase III ATLAS (Antiretroviral Therapy as Long-Acting Suppression), FLAIR (First Long-Acting Injectable Regimen) and ATLAS-2M studies. Ilya Pharma – Sweden’s Ilya Pharma announced it extended its research collaboration in immunophysiology with Uppsala University.

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The Pharma Data

MARCH 17, 2021

Verzenio (abemaciclib) : Abstract # P008 : Patients’ quality of life and side effect perceptions in monarchE, a study of abemaciclib plus endocrine therapy in adjuvant treatment of HR+, HER2-, node-positive, high risk, early breast cancer (Sara M. Accepted as ePoster – Adjuvant Systemic Therapy. Selected for one of the three St.

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The Pharma Data

OCTOBER 14, 2020

Food and Drug Administration granted Breakthrough Therapy designation to France-based Inventiva’s experimental NASH treatment lanifibranor. The funds will be used to bring Ori’s innovative manufacturing platform to the market. Ori Biotech – London-based Ori Biotech closed a $30 million Series A financing round. Source link.

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The Pharma Data

NOVEMBER 26, 2020

Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Chiesi researches, develops, and markets innovative drugs in the respiratory therapeutics, specialist medicine, and rare disease areas.

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The Pharma Data

FEBRUARY 8, 2021

IBRANCE is indicated for the treatment of adult patients with HR+, HER2- advanced or metastatic breast cancer in combination with an aromatase inhibitor as initial endocrine based therapy in postmenopausal women or in men; or with fulvestrant in patients with disease progression following endocrine therapy. 2,3 In the U.S.,

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The Pharma Data

MARCH 16, 2021

1,2 TREMFYA is the first and only IL-23 inhibitor therapy approved in the U.S. Efficacy, safety, pharmacokinetic, immunogenicity and biomarker evaluations were performed in the study on a defined schedule. The Janssen Pharmaceutical Companies of Johnson & Johnson maintain exclusive worldwide marketing rights to TREMFYA®.

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Genkyotex SA , a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset. STOCKHOLM , Jan.

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