Ureas

AbbVie Submits Regulatory Application to FDA for RINVOQ™ (upadacitinib) for the Treatment of Adults with Active Ankylosing Spondylitis

Retrieved on: 
Tuesday, August 25, 2020
Health, Medical specialties, Clinical research, Design of experiments, Pyrrolidines, Organofluorides, Upadacitinib, Ureas, Ankylosing spondylitis, Placebo-controlled study, Clinical trial, Tofacitinib

You should not breastfeed while taking RINVOQ and for at least 6 days after your last dose.

Key Points: 
  • You should not breastfeed while taking RINVOQ and for at least 6 days after your last dose.
  • RINVOQ can make you more likely to get infections or make any infections you have worse.
  • Efficacy and Safety of Upadacitinib in a Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 2/3 Clinical Study of Patients With Active Ankylosing Spondylitis.
  • A Study Evaluating the Safety and Efficacy of Upadacitinib in Subjects With Active Ankylosing Spondylitis (SELECT Axis 1).

Acer Therapeutics to Virtually Present at Upcoming Investor Conferences

Retrieved on: 
Tuesday, August 25, 2020
Branches of biology, Health, Clinical medicine, Rare diseases, Collagen disease, Celiprolol, Ureas, Maple syrup urine disease, Collagen, Sodium phenylbutyrate, Acer, Ehlers–Danlos syndromes

Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs.

Key Points: 
  • Acer is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs.
  • Acers pipeline includes four clinical-stage candidates: emetine hydrochloride for the treatment of patients with COVID-19; ACER-001 (a taste-masked, immediate release formulation of sodium phenylbutyrate) for the treatment of various inborn errors of metabolism, including urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD); EDSIVO (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation; and osanetant for the treatment of induced Vasomotor Symptoms (iVMS).
  • Each of Acers product candidates is believed to present a comparatively de-risked profile, having one or more of a favorable safety profile, clinical proof-of-concept data, mechanistic differentiation and/or accelerated paths for development through specific programs and procedures established by the FDA.
  • For more information, visit www.acertx.com .

Eisai: Application for Additional Indication of Anti Cancer Agent Lenvima for Unresectable Thymic Carcinoma Submitted in Japan

Retrieved on: 
Friday, July 31, 2020
Orphan drugs, Organic compounds, Chemical compounds, Chloroarenes, Ureas, Chemistry, Cyclopropanes, Lenvatinib, Protein kinase inhibitor, Thymic carcinoma

a subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A., announced today that Eisai has submitted an application in Japan for the additional indication of treatment of unresectable thymic carcinoma for multiple receptor tyrosine kinase inhibitor LENVIMA (generic name: lenvatinib mesylate).

Key Points: 
  • a subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A., announced today that Eisai has submitted an application in Japan for the additional indication of treatment of unresectable thymic carcinoma for multiple receptor tyrosine kinase inhibitor LENVIMA (generic name: lenvatinib mesylate).
  • In June 2020, LENVIMA received orphan drug designation in Japan for unresectable thymic carcinoma.
  • This application is based on the results of an open-label, single-arm, multicenter, investigator-initiated clinical phase II study (NCCH1508) conducted in Japan, evaluating LENVIMA as a single agent in 42 patients with thymic carcinoma previously treated with at least one platinum-based regimen.
  • This study met its endpoint as the lower value of the CI exceeded the pre-specified statistical criteria, a threshold ORR of 10%.

ACADIA Pharmaceuticals Announces U.S. FDA Accepted for Filing the Supplemental New Drug Application for NUPLAZID® (pimavanserin) for the Treatment of Hallucinations and Delusions Associated with Dementia-Related Psychosis

Retrieved on: 
Monday, July 20, 2020
Biotechnology, FDA, Health, Pharmaceutical, Clinical trials, Psychiatry, Psychopathology, Abnormal psychology, Chemical compounds, Psychosis, RTT, Pimavanserin, Ureas, Delusion, Hallucination, Acadia Pharmaceuticals

If approved, NUPLAZID would be the first therapy indicated for the treatment of hallucinations and delusions associated with dementia-related psychosis.

Key Points: 
  • If approved, NUPLAZID would be the first therapy indicated for the treatment of hallucinations and delusions associated with dementia-related psychosis.
  • NUPLAZID was approved in the U.S. in 2016 as the first and only treatment for hallucinations and delusions associated with Parkinsons disease psychosis.
  • Pimavanserin was granted Breakthrough Therapy Designation by the FDA for the treatment of hallucinations and delusions associated with DRP in October 2017.
  • ACADIA submitted a supplemental new drug application (sNDA) for pimavanserin for the treatment of hallucinations and delusions associated with dementia-related psychosis on June 3, 2020.

NMD Pharma Receives Approval to Start a combined Phase I/IIa Clinical Trial of NMD670 for the Treatment of Symptoms of Myasthenia Gravis

Retrieved on: 
Tuesday, July 14, 2020
Chloroarenes, Anticonvulsants, Ureas, Anorectics, RTT

NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel.

Key Points: 
  • NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel.
  • NMD Pharma has demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical safety and efficacy data for MG.
  • The combined Phase I/IIa clinical trial is a randomized, double-blind, placebo controlled, single and multiple dose escalation study designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NMD670 in male and female healthy subjects and patients with MG.
  • Thomas Holm Pedersen, Chief Executive Office of NMD Pharma, said: We are very pleased to receive this approval to start our first clinical trial in humans with NMD670.

NMD Pharma Receives Approval to Start a combined Phase I/IIa Clinical Trial of NMD670 for the Treatment of Symptoms of Myasthenia Gravis

Retrieved on: 
Tuesday, July 14, 2020
Chloroarenes, Anticonvulsants, Ureas, Anorectics, RTT

NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel.

Key Points: 
  • NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel.
  • NMD Pharma has demonstrated that ClC-1 inhibition can strengthen neuromuscular transmission and ultimately skeletal muscle function and this novel treatment approach has demonstrated compelling preclinical safety and efficacy data for MG.
  • The combined Phase I/IIa clinical trial is a randomized, double-blind, placebo controlled, single and multiple dose escalation study designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NMD670 in male and female healthy subjects and patients with MG.
  • Thomas Holm Pedersen, Chief Executive Office of NMD Pharma, said: We are very pleased to receive this approval to start our first clinical trial in humans with NMD670.

Shareholder Alert: Robbins LLP Is Investigating the Officers and Directors of Acer Therapeutics Inc. (ACER)

Retrieved on: 
Thursday, July 9, 2020
Legal, Professional services, Celiprolol, Ureas, Anatomy, Ehlers–Danlos syndrome, Collagen disease, Ehlers, Contortion, Acer Inc., Acer, Fiduciary, Organ systems, Medical specialties

Shareholder rights law firm Robbins LLP announces that it is investigating the officers and directors of Acer Therapeutics Inc. (NYSE: ACER) for breaches of fiduciary duty, waste of corporate assets, unjust enrichment, and violations of the Securities Exchange Act of 1934.

Key Points: 
  • Shareholder rights law firm Robbins LLP announces that it is investigating the officers and directors of Acer Therapeutics Inc. (NYSE: ACER) for breaches of fiduciary duty, waste of corporate assets, unjust enrichment, and violations of the Securities Exchange Act of 1934.
  • Acer is a pharmaceutical company that focuses on the development and commercialization of therapies for rare diseases.
  • One of its medications is EDVISO (celiprolol) for the treatment of vascular Ehlers-Danlos Syndrome ("vEDS").
  • To help fund this research, Acer conducted two secondary public offerings in December 2017 and August 2018.

Merck and Eisai Receive Complete Response Letter for KEYTRUDA® (pembrolizumab) plus LENVIMA® (lenvatinib) Combination as First-Line Treatment for Unresectable Hepatocellular Carcinoma

Retrieved on: 
Wednesday, July 8, 2020
Oncology, FDA, Health, Clinical trials, Pharmaceutical, Biotechnology, Clinical medicine, Medicine, Cancer, Chloroarenes, Orphan drugs, Ureas, Hepatology, Antineoplastic drugs, Merkel-cell carcinoma, Pembrolizumab, Sorafenib, Hepatocellular carcinoma, LENVIMA® (lenvatinib) Capsules, 10 mg and 4 mg, Merck, Eisai

As such, LEAP-002, the Phase 3 trial evaluating the KEYTRUDA plus LENVIMA combination as a first-line treatment for advanced HCC, is currently underway and fully enrolled.

Key Points: 
  • As such, LEAP-002, the Phase 3 trial evaluating the KEYTRUDA plus LENVIMA combination as a first-line treatment for advanced HCC, is currently underway and fully enrolled.
  • KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
  • KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.
  • KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).

Myovant Sciences Announces Positive Results from Second Phase 3 Study Evaluating Once-Daily Relugolix Combination Therapy in Women with Endometriosis

Retrieved on: 
Tuesday, June 23, 2020
Chemical compounds, Chemistry, Organic compounds, Ethers, Pyrimidines, Relugolix, Triketones, Ureas, Therapy

Relugolix combination therapy met its co-primary efficacy endpoints and all seven key secondary endpoints in the SPIRIT 1 study.

Key Points: 
  • Relugolix combination therapy met its co-primary efficacy endpoints and all seven key secondary endpoints in the SPIRIT 1 study.
  • In addition, relugolix combination therapy was generally well-tolerated and resulted in minimal bone mineral density loss over 24 weeks.
  • Relugolix combination therapy was generally well-tolerated with minimal bone mineral density loss over 24 weeks.
  • The only reported adverse events in at least 10% of women in the relugolix combination group were headache and hot flashes.

twoXAR Pharmaceuticals Presents Positive Preclinical Safety and Efficacy Data for its Novel Investigational Cancer Treatment TXR-311

Retrieved on: 
Monday, June 22, 2020
Health sciences, Health, Pharmaceutical sciences, Pharmaceutical industry, Chloroarenes, Pyridines, Sorafenib, Ureas, Drug discovery, Pre-clinical development, Drug development, In vivo

The data was presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting II.

Key Points: 
  • The data was presented at the American Association for Cancer Research (AACR) Virtual Annual Meeting II.
  • This study is another demonstration that our approach to drug discovery is effective in identifying novel molecules that have high likelihood of showing positive safety and efficacy signals in preclinical studies," stated Andrew A. Radin, co-founder and CEO of twoXAR.
  • "These early results are interesting and exciting because they showed safety and efficacy comparability against sorafenib but with a completely novel mechanism of action."
  • twoXAR saves years in drug development while generating 30x the hit rate at in vivo efficacy milestones over traditional methods.