LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. A permanent gene edit to produce a difficult-to-titrate supply of GLP1 in the liver?

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The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions. About Eisai Co.,

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The Pharma Data

JANUARY 13, 2021

14, 2021 /PRNewswire/ — MindMed (NEO: MMED), (OTCQB: MMEDF), (DE: MMQ), a leading psychedelic medicine biotech company today announced the addition of Robert Barrow , an accomplished pharmaceutical executive, as Chief Development Officer. We are excited to attract such top tier talent from the psychedelic drug development community.

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The Premier Consulting Blog

OCTOBER 11, 2022

In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases. Key Takeaway RWD and RWE play an important role in rare diseases, where capturing relevant data to support drug development is challenging.

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LifeSciVC

AUGUST 21, 2024

Drug development is a long process, and patients are waiting. An adverse event or serious adverse event (SAE) that turns out NOT to be related to the drug may take a while to sort out. We will have to support an evolving clinical development plan and to keep manufacturing off the critical path.

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The Pharma Data

DECEMBER 20, 2020

In the first study, a phase 1/2 ‘basket’ trial of GPS in combination with the checkpoint inhibitor pembrolizumab (Keytruda), which is conducted under a Clinical Trial Collaboration and Supply Agreement with Merck & Co., Kenilworth, N.J., with a median follow-up of 9.4 Kenilworth, N.J., with a median follow-up of 9.4

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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. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process.

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The Pharma Data

DECEMBER 17, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron and lead author of the publication. patients under an Emergency Use Authorization, and we also continue a robust clinical development program.” In the initial 275 patients, rates of adverse events (AEs) were similar among groups.

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The Pharma Data

DECEMBER 3, 2020

No serious adverse events related to the vaccine candidates studied were reported. The majority of adverse events were mild and transient. We are proud to be partnered with Clover in our endeavors to develop a safe and effective COVID-19 vaccine that will be readily accessible around the world to combat this ongoing pandemic.”.

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The Pharma Data

MAY 31, 2022

The adverse event more commonly observed with Dupixent was conjunctivitis. The FDA grants priority review to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Additional regulatory filings outside of the US are also planned in 2022.

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The Pharma Data

JANUARY 4, 2021

. chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T). Barber’s work will give Kiromic a significant acceleration in the clinical development of its therapy platform and an even more significant advantage over its competitors.

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The Pharma Data

DECEMBER 9, 2020

The FURI study is evaluating oral ibrexafungerp as a salvage treatment in patients with a variety of difficult-to-treat mucocutaneous and invasive fungal infections that are refractory to, intolerant of current standards of care, or require a non-azole oral step-down therapy for treatment of azole-resistant species. Forward Looking Statement.

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NIH Director's Blog: Drug Development

JUNE 14, 2016

Several years ago, the Food and Drug Administration (FDA) recommended that drug developers take special care to show that potential drugs to treat diabetes don’t adversely affect the cardiovascular system [1]. Food and Drug Administration. 5] Clinical development success rates for investigational drugs.

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The Pharma Data

OCTOBER 25, 2020

Treatment choices are limited, and commonly used off-label therapies are not always effective or may result in prolonged, deep sedation. A therapy that quickly and effectively reduces agitation, without causing excessive sedation, is needed to speed up recovery time and improve patient outcomes. NEW HAVEN, Conn.,

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The Pharma Data

JANUARY 7, 2021

Cerecor intends to use the net proceeds of the offering for general corporate purposes and working capital, primarily to support the ongoing clinical development of key assets within its pipeline and for general and administrative expenses. The company is also developing two monoclonal antibodies, CERC-002, and CERC-007.

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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) . Chief Clinical Officer, Neurology Business Group, Eisai. aggregates (protofibrils).

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The Pharma Data

OCTOBER 27, 2020

NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that the U.S. There are currently no disease-modifying therapies for Krabbe disease, and we believe incidence may be 2.6 PHILADELPHIA, Oct. About PBKR03.

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The Pharma Data

JANUARY 31, 2021

No serious adverse events related to the vaccine candidates studied were reported. These findings give confidence that Clover’s COVID-19 vaccine candidates are suitable for further clinical development.” Dynavax is a commercial stage biopharmaceutical company developing and commercializing novel vaccines.

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Our rare disease team at Worldwide will be hosting a panel discussion webinar with patient advocates active in Rare Disease Day events to synthesize the most salient conversations taking place. Rob: UA first began donating funds to researchers through our annual event, the Young Investigator’s Draft, in 2018. So, I decided to pivot.

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The Pharma Data

DECEMBER 29, 2020

As in earlier outpatient trial, immune status when patients entered the trial was a strong predictor of viral load and clinical outcomes. First antibody therapy to demonstrate anti-viral effect in patients hospitalized with COVID-19. Senior Vice President and Head of Global Clinical Development at Regeneron. In the U.S.

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The Pharma Data

AUGUST 29, 2020

Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab. To date, after at least six months of stable LNP023 add-on therapy, and at the investigators’ discretion, seven patients (70%) have discontinued eculizumab and remained on LNP023 as monotherapy.

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The Premier Consulting Blog

JUNE 18, 2023

This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases.

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The Pharma Data

MARCH 23, 2023

Dupixent is the first and only biologic to demonstrate a clinically meaningful and highly significant reduction (30%) in moderate or severe acute exacerbations of COPD (rapid and acute worsening of respiratory symptoms), while also demonstrating significant improvements in lung function, quality of life and COPD respiratory symptoms.

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The Pharma Data

NOVEMBER 29, 2020

Antiviral therapies are used to help control disease, but they are limited by toxicity and the emergence of viral resistance. Three cases of human leukocyte antigen (HLA)-sensitization have been reported, two as serious adverse events. An archived replay will be accessible for 30 days following the event.

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The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

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The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

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The Pharma Data

DECEMBER 5, 2020

” In the trial, the 49 patients evaluated had a median of five prior lines of therapy (range: 2-17) with 100% being triple-refractory and 57% being penta-refractory; all patients were refractory to anti-CD38 therapy. With a median follow up of 2.6 74% of responders (n=14) remain on treatment. About Regeneron Pharmaceuticals, Inc.

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The Pharma Data

JULY 8, 2021

Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. Please see full Prescribing Information including Medication Guide. About Biogen. At Biogen, our mission is clear: we are pioneers in neuroscience.

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The Pharma Data

OCTOBER 28, 2020

As in the earlier analysis, patients with higher viral load at baseline and/or no detectable antibodies at baseline (suggesting their bodies had not yet mounted an effective immune response), derived greater benefit from REGN-COV2 therapy. Serious adverse events were numerically more frequent with placebo than REGN-COV2 treatment (0.8%

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Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

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Drug Target Review

JANUARY 15, 2024

Gaining access inside cells Fortunately, evolution has provided a mechanism, found in all higher vertebrates, for the immune system to monitor intracellular events. Previously, as a member of the faculty at Harvard Medical School , Dr Kavanagh was engaged in preclinical and clinical development of cellular immunotherapy and products.

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Drug Discovery World

AUGUST 7, 2023

In our current environment of political polarization, efforts to encourage diversity are unable to prevail, and more needs to be done to foster the trust that is necessary for broader participation in clinical trial recruitment. RA: How does a lack of patient diversity in trials impact how treatments work in certain populations?

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The Pharma Data

NOVEMBER 5, 2020

Lastly, we are proud that our novel antibody cocktail REGN-EB3 recently became the first FDA-approved treatment for Ebola, underscoring the potential of antibody therapies to address deadly infectious diseases.” Updates from the clinical pipeline include: Dupixent ® (dupilumab). Corporate and Business Development Update.

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The Pharma Data

OCTOBER 14, 2020

. “We are very pleased that the FDA has cleared our IND, and we plan to initiate clinical development of AP-PA02 by the end of this year, consistent with our original guidance notwithstanding disruptions to drug development timelines across the industry caused by the COVID-19 pandemic,” stated Todd R.

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Agency IQ

AUGUST 16, 2024

As opposed to the linear dose-response relationships of chemotherapies, the wide therapeutic indexes of many of these newer therapies means that doses can be increased without simultaneously increasing toxicities. Other issues also arise: Unlike conventional cytotoxic drugs where the relationship between early (e.g.,

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The Pharma Data

OCTOBER 28, 2020

Preliminary results from the first five patients treated indicate substantial improvements in all patients in at least two disease domains including communication, behavior, sleep, gross motor function, and fine motor function as measured by the Clinical Global Impression of Improvement Scale for Angelman Syndrome (CGI-I-AS) at day 128.

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The Pharma Data

OCTOBER 27, 2020

The collaboration aims to leverage Molecular Partners’ proprietary DARPin® technologies and Novartis’ broad expertise in global drug development, regulatory affairs, manufacturing and commercialization to rapidly advance the program in keeping with the unprecedented global urgency created by the pandemic.

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