The Pharma Data

DECEMBER 17, 2020

18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.

Treatment 52

Treatment Clinical Trials International Pharmaceutical Companies 52

The Pharma Data

APRIL 7, 2022

It may also limit coverage for any future approved treatment in the class. When additional data from this new class of treatments become available, Biogen urges CMS to reconsider today’s decision for all FDA-approved amyloid-beta targeting therapies. ADUHELM is indicated for the treatment of Alzheimer’s disease.

Therapies 52

Therapies Treatment Disease FDA Approval 52

Drug Target Review

JANUARY 25, 2024

These findings offer a novel strategy for developing targeted treatments for triple-negative breast cancer, which currently has no approved options. The team also observed that the drug prevented in vitro cancer stem cells from self-renewing, which is a characteristic of aggressive cancer cells that supports treatment resistance.

Treatment 106

Treatment Clinical Trials Research Science 106

The Pharma Data

MARCH 27, 2023

Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen met its co-primary endpoints through 66 weeks. The results were consistent with the positive 35-week findings announced in June 2022. Sami Khella, M.D.,

Trials 40

Trials Disease Treatment Science 40

The Pharma Data

NOVEMBER 12, 2020

The DETOUR1 trial is the first-in-man trial of the FDA-designated Breakthrough Device, The Detour System, evaluating the safety and effectiveness of the novel procedure and device system for treating long, complex lesions in the superficial femoral artery (SFA). This press release features multimedia. About the Company: PQ Bypass, Inc.

Clinical Trials 52

Clinical Trials Trials FDA Treatment 52

New Drug Approvals

NOVEMBER 13, 2023

WO 2017/208267A1, WO 2020/250133 Al and WO 2022/024072A1, which are hereby incorporated by reference, disclose methods of use of the compound of Formula I for the treatment of Parkinson’s disease, synucleinopathies and Alzheimer’s disease (AD) respectively. amcrasto@amcrasto /////////// Ref [1].

Treatment 62

Treatment Disease Therapies Production 62

The Pharma Data

NOVEMBER 24, 2020

25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).

Treatment 52

Treatment DNA Trials Therapies 52

Drug Target Review

OCTOBER 24, 2023

Live Bacterial Products (LBPs) will provide patients with a desirable, credible, safe and effective treatment option. The human microbiome, a complex ecosystem of microorganisms residing within our bodies, has emerged as a promising frontier in the quest for innovative medical treatments.

Treatment 122

Treatment Pharmacokinetics Pharmaceuticals Production 122

The Pharma Data

JANUARY 3, 2021

Anecdotal feedback from Foralumab-treated patients was positive and suggests that the treatment was well-tolerated. This trial is the first to evaluate nasally administered Foralumab to improve the immune system’s fight against coronaviruses. NEW YORK and LONDON, Jan.

Clinical Trials 40

Clinical Trials Science Treatment Trials 40

Agency IQ

JUNE 28, 2024

New guidance provides definition for orphan device, offers alternative trial designs New guidance from the European Commission outlines alternatives for full pre-market clinical trials for orphan devices, defined by the Commission for the first time. Appendix A.2

Trials 40

Trials Marketing Clinical Trials Regulations 40

Codon

JANUARY 21, 2024

yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops. Human challenge trials were an indispensable part of the development of the malaria vaccine, R21/Matrix-M, endorsed by the World Health Organization last October.

Vaccine 111

Vaccine Trials Disease Treatment 111

PPD

JUNE 20, 2024

Despite advances in technology and innovation, pharmaceutical leaders say that trials are increasingly complex, particularly when it comes to patient recruitment and site engagement. Major hurdles include: Clinical trial volume: Since 2020, registered studies have surged by 50% , intensifying the competition for resources, patients and sites.

Clinical Research 52

Clinical Research Research Clinical Trials Pharmaceuticals 52

The Pharma Data

JULY 21, 2021

(NYSE: PFE), today announced that they have completed recruitment for the Phase 2 trial, VLA15-221, of Lyme disease vaccine candidate, VLA15. The trial builds on previous positive Phase 2 trials and includes both adult and pediatric participants with the aim to support acceleration of the vaccine candidate’s pediatric program.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. The recruitment period for the study spanned from December 2015 to July 2017. 6] ERLEADA ® is being studied in five Phase 3 registrational clinical trials. . ‡

Treatment 52

Treatment Pharmaceutical Companies Therapies Pharmaceuticals 52

Broad Institute

OCTOBER 4, 2023

A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy. AbbVie and Calico discovered the molecule, called ABBV-CLS-484 , after TIDE researchers at Broad identified the PTPN2 gene as a promising cancer immunotherapy target in 2017.

Small Molecule 128

Small Molecule Clinical Trials Engineering Trials 128

The Pharma Data

JANUARY 25, 2021

agree to place handheld Ceribell Rapid Response EEG systems in hospitals in connection with its Phase 3 RSE clinical trial. Within the RAISE trial, it will allow clinicians to rapidly establish a diagnosis of nonconvulsive SE in the absence of immediate availability of conventional EEG. About the RAISE Trial. RADNOR, Pa.–(

Clinical Trials 52

Clinical Trials Trials Hospitals Pharmaceutical Companies 52

The Pharma Data

JANUARY 11, 2021

The trial plans to enroll 99 boys aged 4 to 7 years across 55 sites in 15 countries. The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. Meanwhile, Sarepta Therapeutics reported disappointing top-line results from a phase 2 trial evaluating SRP-9001, its gene therapy for DMD.

Therapies 52

Therapies Trials FDA Treatment 52

Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. Early-phase clinical trials have shown the safety and efficacy of using stem cells for cardiac repair, encouraging further research and development.

Therapies 52

Therapies Disease Engineering RNA 52

Drug Target Review

JUNE 19, 2024

Our goal is to stand on the shoulders of giants and advance this exciting and new field of medicine, so that it can deliver valuable treatments to patients. That would provide wonderful information as we think about then going into a first-in-human clinical trial. SVRA) in April 2017. from May 2017 until December 2022.

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

Treatment 52

Treatment Pharmaceutical Companies Therapies Virus 52

Agency IQ

AUGUST 16, 2024

Many commenters took issue with the guidance’s statement that, “The contribution of the psychotherapy component to any efficacy observed with psychedelic treatment has not been characterized. Throughout 2017, Lykos sought feedback on the protocol for its first Phase 3 study, MAPP1, via the Special Protocol Assessment (SPA) process.

FDA 40

FDA Trials Therapies Regulations 40

The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease. In June 2021, the U.S.

Disease 52

Disease Clinical Trials Trials Treatment 52

The Pharma Data

MARCH 4, 2021

Together we generated positive Phase 2 ELEKTRA study data, and as a result, soticlestat is poised to enter two pivotal trials,” said Andy Plump, M.D., Our goal is to one day bring new treatment options that provide greater seizure control, tolerability and function to DS and LGS patients around the world.”.

Treatment 52

Treatment Clinical Trials Pharmaceutical Companies Trials 52

PPD

OCTOBER 12, 2023

These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type. There are over 2,000 CGTs currently being evaluated in clinical trials, with more than 200 in Phase III and 10-20 per year estimated to come to market by 2025.

Laboratories 52

Laboratories Protein Expression Clinical Trials Pharmacokinetics 52

The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

Disease 40

Disease FDA Treatment Therapies 40

The Pharma Data

NOVEMBER 17, 2021

Tokyo, Japan) blazoned moment an update on the ongoing review of the Marketing Authorization Operation for aducanumab 100 mg/ mL concentrate result for infusion for the treatment of Alzheimer’s complaint by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). As of October 2017, Biogen and EisaiCo.,Ltd.

Clinical Trials 52

Clinical Trials Biosimilars Treatment Trials 52

The Pharma Data

JUNE 25, 2021

protofibril antibody for the treatment of Alzheimer’s disease (AD). The FDA’s Breakthrough Therapy designation for lecanemab is based on the recently published results of a Phase 2b clinical trial (Study 201) of 856 patients with mild cognitive impairment (MCI) due to AD and mild AD with confirmed presence of amyloid pathology.1

Disease 40

Disease Treatment Therapies FDA 40

The Pharma Data

AUGUST 9, 2020

In the HIBISCUS I induction study, in people without prior anti-tumour necrosis factor (anti-TNF) treatment, etrolizumab met the primary endpoint. In contrast, the HIBISCUS II induction study, which also included people without prior anti-TNF treatment, did not meet its primary endpoint.

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

The Pharma Data

MARCH 16, 2022

The data also show that in both Phase 3 trials, at 78 weeks, patients with reduced levels of plasma p-tau181 had less clinical decline than those whose plasma p-tau181 levels were not reduced. These findings point to the potential of continued benefit of treatment in the longer term with continued reduction of amyloid beta plaques.

Disease 52

Disease Clinical Trials Treatment Trials 52

Drug Target Review

OCTOBER 26, 2023

1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Wang D, Tai PW, Gao G.

Engineering 105

Engineering Virus Therapies Immune Response 105

The Pharma Data

AUGUST 25, 2020

While we are disappointed by the outcome of the IDHENTIFY study, we remain confident in IDHIFA ’s established role as a treatment option for patients with relapsed or refractory AML with an IDH2 mutation and are grateful to all those who participated in the study,” said Noah Berkowitz, M.D., Food and Drug Administration (FDA)-approved test.

Trials 52

Trials FDA Approval Therapies Treatment 52

New Drug Approvals

OCTOBER 24, 2023

Molecular Weight: 631.700 FDA APPROVED, To treat moderately to severely active ulcerative colitis in adults, 10/12/2023 Velsipity Etrasimod , sold under the brand name Velsipity , is a medication that is used for the treatment of ulcerative colitis (UC). [1] Peyrin-Biroulet L, Christopher R, Behan D, Lassen C (May 2017). 2017.03.007.

FDA 57

FDA FDA Approval International Pharmacokinetics 57

The Pharma Data

APRIL 28, 2022

currently on ADUHELM avoid any treatment interruptions following the final national coverage determination by Centers for Medicare & Medicaid Services (CMS) that could result in a loss of coverage for Medicare beneficiaries. For those already on ADUHELM, this meant a potential halt in their treatment plans.

Clinical Trials 52

Clinical Trials Treatment Biosimilars Disease 52

The Pharma Data

NOVEMBER 10, 2020

The trial is a dose-ranging, open-label study that will enroll 60 patients in the U.S. We believe the scientific rationale for oral treatment with Foralumab is logical to facilitate topical action at the inflamed sites in the gastrointestinal tract. NEW YORK and LONDON, Nov. NEW YORK and LONDON, Nov. and Europe. Dr. Howard L.

Clinical Trials 40

Clinical Trials Science Disease Trials 40

The Pharma Data

AUGUST 5, 2020

Ofatumumab is a targeted B-cell therapy that delivers superior efficacy with a similar safety profile when compared with teriflunomide, a commonly prescribed oral treatment for multiple sclerosis 1 . 10% of patients 1,3. ” These data were published in the August 6, 2020 issue of The New England Journal of Medicine.

Trials 52

Trials Treatment Disease Therapies 52

Advarra

AUGUST 16, 2022

Orphan drugs have historically faced a number of barriers, such as limited research and development (R&D) investment due to an expected lack of profitability as well as challenges in clinical trial design and recruitment. Before 1983, only 38 orphan drugs had received U.S. Food and Drug Administration (FDA) approval.

Disease 52

Disease Clinical Trials FDA Approval FDA 52

The Pharma Data

APRIL 4, 2022

living with eosinophilic esophagitis who are currently treated, of whom approximately 48,000 have failed multiple treatments. The safety results of these trials were generally consistent with the known safety profile of Dupixent in its approved indications. If approved, Dupixent would be the first medicine available in the U.S.

FDA 52

FDA Treatment Disease FDA Approval 52