Aes

Timber Pharmaceuticals Announces Publication of Sub-Analysis of Phase 2b CONTROL study in Clinical and Experimental Dermatology

Retrieved on: 
Monday, March 20, 2023
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BASKING RIDGE, NJ, March 20, 2023 (GLOBE NEWSWIRE) -- via NewMediaWire -- Timber Pharmaceuticals, Inc. ("Timber" or the “Company”) (NYSE American: TMBR), a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases, today announced the online publication of a sub-analysis of the Phase 2b CONTROL study in Clinical and Experimental Dermatology (CED), the educational journal of the British Association of Dermatologists. The CONTROL study evaluated TMB-001, a topical isotretinoin formulated using the Company’s patented IPEG™ delivery system, in subjects nine years of age and older with moderate to severe congenital ichthyosis (CI).

Key Points: 
  • The CONTROL study evaluated TMB-001, a topical isotretinoin formulated using the Company’s patented IPEG™ delivery system, in subjects nine years of age and older with moderate to severe congenital ichthyosis (CI).
  • CI is a group of rare genetic keratinization disorders that lead to dry, thickened, and scaling skin.
  • “In this sub-analysis, participants in the CONTROL study with ARCI and XLRI mostly showed comparable percentage differences in responses to treatment with a novel topical isotretinoin formulation versus vehicle.
  • The intent-to-treat (ITT) population consisted of all randomized participants who received one or more doses of the study medication.

Synlogic Announces Data Presentations at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting

Retrieved on: 
Monday, March 20, 2023
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In separate poster presentations, the company also presented clinical data and preclinical data related to its homocystinuria (HCU) program.

Key Points: 
  • In separate poster presentations, the company also presented clinical data and preclinical data related to its homocystinuria (HCU) program.
  • “We were delighted to review these encouraging findings from our two rare metabolic disease programs among the expert metabolic clinicians who attend the SIMD meeting,” said Dave Hava, Chief Scientific Officer and Head of Research and Development at Synlogic.
  • Clinical data presented included positive data from the Phase 1 study evaluating SYNB1353 in healthy volunteers using a dietary model of HCU.
  • Posters presented at the SIMD meeting are posted on the Publications page of the Synlogic website.

New Four-Year Data for Genentech’s Evrysdi Reinforce Long-Term Efficacy and Safety Profile in Some of the Most Severely Affected People With Types 2 and 3 Spinal Muscular Atrophy (SMA)

Retrieved on: 
Monday, March 20, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Clinical Trials, Muscular Dystrophy Association, SMA, PTC Therapeutics, Natural history, PTC, Aes, Roche, Facial muscles, Șaeș, ROG, RO, Patient, Episcopal conference, Upper respiratory tract infection, Phase, MDA, Headache, Therapy, Conference, Caregiver, Safety, Disease, Pharmaceutical industry, Genentech, Fever, Risdiplam

Participants also reported continuous improvement or stabilization when independently performing activities of daily living such as picking up and moving objects.

Key Points: 
  • Participants also reported continuous improvement or stabilization when independently performing activities of daily living such as picking up and moving objects.
  • The data were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 19-22, 2023.
  • “Preserving long-term independence and the ability to perform daily tasks is an important measure for people living with SMA and their caregivers.
  • Genentech is currently investigating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the Phase II/III trial MANATEE for the treatment of SMA.

LEO Pharma Presents Late-Breaking Positive Phase 2a Efficacy and Safety Results of LEO 138559 in Moderate-to-Severe Atopic Dermatitis at the 2023 AAD Annual Meeting

Retrieved on: 
Saturday, March 18, 2023
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LEO Pharma A/S, a global leader in medical dermatology, today announced that a Phase 2a trial evaluating the efficacy and safety of investigational agent LEO 138559 in adults with moderate-to-severe atopic dermatitis met its primary endpoint.

Key Points: 
  • LEO Pharma A/S, a global leader in medical dermatology, today announced that a Phase 2a trial evaluating the efficacy and safety of investigational agent LEO 138559 in adults with moderate-to-severe atopic dermatitis met its primary endpoint.
  • Results were shared as one of two LEO Pharma late breaker oral presentations at the 2023 American Academy of Dermatology (AAD) Annual Meeting.1 LEO 138559 is an investigational agent and its efficacy and safety are subject to further larger trials.
  • LEO Pharma and argenx BV jointly developed LEO 138559 under an exclusive option and research agreement.
  • LEO Pharma obtained the license to LEO 138559 in 2022 and now assumes the responsibility to develop and commercialize LEO 138559 for inflammatory skin disorders, such as atopic dermatitis.

LEO Pharma Presents New Adbry™ (tralokinumab-ldrm) Data in Adolescent Population from ECZTRA 6 and ECZTEND Trials at AAD 2023 Annual Meeting

Retrieved on: 
Friday, March 17, 2023
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The data was presented at the American Academy of Dermatology (AAD) 2023 Annual Meeting.

Key Points: 
  • The data was presented at the American Academy of Dermatology (AAD) 2023 Annual Meeting.
  • S. aureus is consistently found in the skin lesions of patients with AD and contributes to skin irritation and infections.
  • “In addition, the latest analysis of data from adolescents in the ECZTEND study shows the long-term safety and efficacy profile of Adbry.
  • Adtralza is approved for use in adults with moderate-to-severe AD in the U.S., United Arab Emirates, Switzerland, and Japan.

Mitsubishi Tanabe Pharma America Announces More Than 10,000 People with ALS Treated with RADICAVA ORS® (edaravone) and/or RADICAVA® (edaravone)

Retrieved on: 
Thursday, March 16, 2023
ALS, Edaravone, Average absolute deviation, RV, Fatigue, Pharmacy, Patient, Physician, Temperature, FDA, Disease, History, Weakness, Bruise, MTPA, Hypersensitivity, Headache, Aes, United States Air Force, HIV disease progression rates, Pharmaceutical industry

"This product milestone falls on MTPA's seventh anniversary as a U.S. company committed to helping those with ALS," said Atsushi Fujimoto, President, MTPA.

Key Points: 
  • "This product milestone falls on MTPA's seventh anniversary as a U.S. company committed to helping those with ALS," said Atsushi Fujimoto, President, MTPA.
  • "Addressing the unmet needs of people living with serious, debilitating diseases like ALS, is of the utmost importance to us, and we remain focused on delivering meaningful treatments to this community."
  • The initial treatment cycle starts with daily dosing of RADICAVA ORS for 14 days followed by a 14-day drug-free period.
  • RADICAVA and RADICAVA ORS are contraindicated in people with a history of hypersensitivity to edaravone or any of the inactive ingredients.4 See Important Safety Information below.

Direct Biologics Reports Compelling Results From Phase 2 Trial With ExoFlo™ in Hospitalized Patients With Respiratory Failure or Moderate-to-Severe Acute Respiratory Distress Syndrome (ARDS) Due to COVID-19

Retrieved on: 
Tuesday, March 14, 2023
COVID-19, Research, Clinical Trials, Stem Cells, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Trial of the century, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Șaeș, Respiratory failure, Ageing, Acute respiratory distress syndrome, Regenerative medicine, Aes, Seminal vesicles, Bone marrow, Disease, VFD, Patient, Death, MSC, Food, Perioperative, Microsoft Exchange Server, Incidence, EVS, FDA, DSMB, Multisite cloud, Mortality, Survival, Hospital, Pharmaceutical industry

“We are pleased to report compelling findings that provide hope to the thousands of patients around the world with respiratory failure and moderate-to-severe ARDS due to COVID-19.

Key Points: 
  • “We are pleased to report compelling findings that provide hope to the thousands of patients around the world with respiratory failure and moderate-to-severe ARDS due to COVID-19.
  • Findings demonstrated a significant survival benefit in ExoFlo-treated patients with respiratory failure due to COVID-19.
  • The FDA has granted Direct Biologics an allowance to proceed with a Phase 3 trial of ExoFlo-15 mL compared with placebo, randomized 1:1.
  • This multisite global trial will look at 60-day all-cause mortality in patients who meet Berlin criteria for moderate-to-severe ARDS.

RADICAVA ORS® (edaravone) Added to U.S. Department of Veterans Affairs National Formulary

Retrieved on: 
Friday, March 10, 2023
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JERSEY CITY, N.J., March 10, 2023 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) is proud to announce that as of June 2022, RADICAVA ORS® (edaravone) has been added to the U.S. Department of Veterans Affairs National Formulary (VANF), with a national prior authorization process in place, making the treatment accessible to U.S. veterans living with amyotrophic lateral sclerosis (ALS) who are within the VA's integrated healthcare system.

Key Points: 
  • "Given the devastating nature of ALS and the demand for additional treatment options, we are thrilled to see RADICAVA ORS added to the VA's National Formulary," said Atsushi Fujimoto, President, MTPA.
  • "MTPA is committed to helping U.S. veterans living with ALS have access to our treatments, and we applaud the VA for making RADICAVA ORS available as an option for them."
  • The Criteria for Use of RADICAVA ORS can be found at the VA website here: https://www.pbm.va.gov/apps/VANationalFormulary/ .
  • RADICAVA and RADICAVA ORS are contraindicated in people with a history of hypersensitivity to edaravone or any of the inactive ingredients.1 See Important Safety Information below.

Aclaris Therapeutics Announces Preliminary Topline Data from 12-Week Phase 2a Study of Oral Zunsemetinib (ATI-450) for Moderate to Severe Hidradenitis Suppurativa

Retrieved on: 
Monday, March 6, 2023
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WAYNE, Pa., March 06, 2023 (GLOBE NEWSWIRE) -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced preliminary topline results from a 12-week, Phase 2a, multicenter, randomized, placebo-controlled clinical study to investigate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of zunsemetinib (ATI-450), an investigational oral MK2 inhibitor, in patients with moderate to severe hidradenitis suppurativa (HS) (ATI-450-HS-201).

Key Points: 
  • The study did not meet its primary endpoint of change from baseline in inflammatory nodule/abscess count (AN) of zunsemetinib 50mg BID versus placebo at week 12.
  • The study also did not meet the secondary efficacy endpoints assessed in the topline data, including percentage of patients achieving HiSCR-50.
  • The placebo effect observed across all efficacy endpoints was higher than what has been observed in other published HS studies reported to date.
  • Thirty-seven patients discontinued study treatment (22 on zunsemetinib and 15 on placebo), with 15 patients discontinuing treatment due to AEs (11 on zunsemetinib and 4 on placebo).

BridgeBio Announces Positive Phase 2 Cohort 5 Results of Infigratinib in Achondroplasia Demonstrating Mean Increase in Annualized Height Velocity of 3.03 cm/year with No Treatment-related Adverse Events

Retrieved on: 
Monday, March 6, 2023
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Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source.

Key Points: 
  • Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source.
  • BridgeBio will also host an investor call on March 6, 2023, at 7:30 am ET to discuss the results from the Phase 2 study.
  • To date, the study has shown a well-tolerated safety profile, with no study drug related treatment emergent adverse events (TEAEs) in Cohort 5.
  • Based on the positive results to date, BridgeBio has started enrolling children in the run-in for a Phase 3 trial.