Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

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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

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

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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.:

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The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness.

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KIF1A

MARCH 16, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.

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The Pharma Data

JANUARY 10, 2021

Chief Executive Officer of MorphoSys, will present at the 39 th Annual J.P. EST. Live audio of the presentation can be accessed from the Media and Investors section under Conferences on MorphoSys’ website, www.morphosys.com. The presentation as well as a replay of the webcast will also be available on MorphoSys’ website.

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The Pharma Data

APRIL 11, 2023

The presentation highlights the synergistic anti-cancer effects of a PSMA (prostate-specific membrane antigen)-actinium-225 conjugate in combination with darolutamide in preclinical prostate cancer models. Data from all three areas of scientific focus will be showcased during this year’s meeting.

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The Pharma Data

APRIL 12, 2022

20% variant allele frequency reduction – At the time of analysis with > 2 year follow up the survival estimate was 100% in patients who had improvements in bone marrow fibrosis or variant allele frequency – Results were presented at the American Association for Cancer Research annual meeting.

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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek.

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.

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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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The Pharma Data

APRIL 26, 2022

NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme disease vaccine candidate, VLA15. Valneva and Pfizer plan to submit these data for publication and presentation at a future scientific congress. About VLA15 VLA15 is the only Lyme disease vaccine candidate currently in clinical development.

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The Pharma Data

JULY 28, 2021

The TAK-994 BTD was based, in part, on early phase and preliminary clinical data that indicates Takeda’s investigational oral orexin agonist may demonstrate substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients. 1 Currently, TAK-994 is being studied in an ongoing Phase 2 (TAK-994-1501) study.

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KIF1A

SEPTEMBER 30, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease? What’s a pre-print?

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The Pharma Data

NOVEMBER 14, 2020

The results were presented at The Liver Meeting Digital Experience (TLMdX), November 13–16, 2020 (Late Breaker #L02). These data offer new insights into potential therapeutic approaches to treating NASH, a disease which currently has limited treatment options.”. About NASH.

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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 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. The presentation will also summarize previously released clinical data from the Phase I study performed by Janssen Biotech Inc.

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Drug Target Review

APRIL 9, 2024

How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration.

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The Pharma Data

MARCH 9, 2021

New data from 10 studies will be presented by Eli Lilly and Company (NYSE: LLY) at the upcoming virtual International Conference on Alzheimer’s & Parkinson Diseases 2021 (AD/PD 2021), March 9-14, 2021. vice president of Alzheimer’s disease development unit, Eli Lilly and Company.

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The Pharma Data

OCTOBER 21, 2020

(Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced that it will present a poster on SYNB8802, an investigational Synthetic Biotic medicine for the treatment of Enteric Hyperoxaluria (HOX), during the American Society of Nephrology (ASN) Kidney Week, Oct.

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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

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The Pharma Data

SEPTEMBER 16, 2020

New integrated analyses from our tumour agnostic Rozlytrek ® (entrectinib) clinical development programme. Blueprint Medicines will present new data from the registrational phase I/II ARROW trial, investigating Gavreto TM (pralsetinib) for the treatment of people with RET-mutant medullary thyroid cancer.

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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. These data (Presentation #2857) are being presented today during the Late Breaking News Session from 3:45 – 4:45 p.m.

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Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. The rigors of training as a physician-scientist present their share of obstacles to overcome.

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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.

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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. We are excited to be part of the Lyme disease vaccine development program with Valneva. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick.

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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The Pharma Data

APRIL 8, 2021

Phase 3 Data from KEYNOTE-564 to be Presented at Upcoming Medical Meeting and Submitted to Regulatory Authorities. Results will be presented at an upcoming medical meeting and will be submitted to regulatory authorities. Most cases of RCC are discovered incidentally during imaging tests for other abdominal diseases. In the U.S.

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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

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. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick.

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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). About Krabbe Disease. Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021.

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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

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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. Therefore, atacicept may be uniquely positioned to be disease-modifying for these patients with no current treatment options.”

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

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The Pharma Data

OCTOBER 18, 2020

ZURICH, Switzerland–( BUSINESS WIRE )– Versantis AG , a clinical-stage company developing novel therapies for orphan liver diseases, today announced that the U.S. VS-01 is currently being evaluated in clinical trials in decompensated cirrhotic patients.

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PPD

OCTOBER 17, 2024

This expansion represents a significant opportunity for both drug developers and patients, while also presenting a new and diverse set of challenges for drug developers, particularly in clinical trial execution. Factoring in cultural dynamics is key to enabling new therapy treatment success.

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