Sodium phenylbutyrate

Pheburane® (sodium phenylbutyrate) coverage to reach ~80% of commercially insured US patients by January 1st, 2024

Retrieved on: 
Thursday, October 26, 2023
Government, Ammonia, History, Courage, Digestion, OTC, UCD, Health, DSM-IV codes, UCD School of Medicine, CPS, Disease, Pharmacy, Medicaid, Sodium phenylbutyrate, Patient, Government of Canada, Appetite, Pharmaceutical industry

Pheburane® is not indicated for the treatment of acute hyperammonemia which can be life-threatening and requires emergency medical treatment.

Key Points: 
  • Pheburane® is not indicated for the treatment of acute hyperammonemia which can be life-threatening and requires emergency medical treatment.
  • UCDs are rare, chronic, genetic conditions that can be fatal if left untreated, and can impact children from the time of birth.
  • It does not require any mixing or oral syringes and there is no weight restriction for patients.
  • UNIK Support offers specialized services including a copay savings program, patient care liaison services, mail order pharmacy and other support services.

Pheburane® Expanded Coverage with Commercial and Government (Medicaid) Payers

Retrieved on: 
Wednesday, May 17, 2023
Taste, Medicaid, Digestion, UCD, Pharmacy, Appetite, History, Tablet, PBM, WAC, UCD School of Medicine, Patient, Ammonia, CPS, OTC, DSM-IV codes, Health, Pharmaceutical industry, Sodium phenylbutyrate

Pheburane® is an innovative, palatable formulation of sodium phenylbutyrate (NaPB) with a special coating designed to mask the awful taste of NaPB.

Key Points: 
  • Pheburane® is an innovative, palatable formulation of sodium phenylbutyrate (NaPB) with a special coating designed to mask the awful taste of NaPB.
  • Pheburane® is not indicated for the treatment of acute hyperammonemia which can be life-threatening and requires emergency medical treatment.
  • Medunik USA has also worked diligently since that time to secure commercial and government reimbursement while increasing physician and patient awareness.
  • “In fact, the list price (WAC) of Pheburane® is approximately one third that of the most commonly prescribed UCD treatment.

Relief and Acer Therapeutics Sign Collaboration and License Agreement for Worldwide Development and Commercialization of ACER-001 for the Treatment of Urea Cycle Disorders and Maple Syrup Urine Disease

Retrieved on: 
Monday, March 22, 2021
Maple syrup urine disease, Acer Inc., Sodium phenylbutyrate, Health, Companies

Relief will also pay Acer up to $20 million in U.S. development and commercial launch costs for the UCDs and MSUD indications.

Key Points: 
  • Relief will also pay Acer up to $20 million in U.S. development and commercial launch costs for the UCDs and MSUD indications.
  • As part of its pipeline diversification strategy, In March 2021, Relief entered into a Collaboration and License Agreement with Acer Therapeutics for the worldwide development and commercialization of ACER-001.
  • ACER-001 is a taste-masked and immediate release proprietary powder formulation of sodium phenylbutyrate (NaPB) for the treatment of Urea Cycle Disorders and Maple Syrup Urine Disease.
  • On March 19, 2021, Acer entered into a Collaboration and License Agreement with Relief Therapeutics for worldwide development and commercialization of ACER-001.

Acer Therapeutics to Participate in March Virtual Investor Conferences

Retrieved on: 
Monday, March 1, 2021
Branches of biology, Health, Rare diseases, Collagen disease, Maple syrup urine disease, Celiprolol, Acer, Collagen, Sodium phenylbutyrate, Ehlers–Danlos syndromes, Ehlers, Maple

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 programs: ACER-001 (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; ACER-801 (osanetant) for the treatment of induced Vasomotor Symptoms (iVMS); and ACER-2820 (emetine), a host-directed therapy against a variety of infectious diseases, including COVID-19.
  • 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 .

Acer Therapeutics Announces Topline Results from its Bioequivalence Trial of ACER-001 Compared to BUPHENYL® Under Fed Conditions

Retrieved on: 
Thursday, February 11, 2021
Health sciences, Health, Life sciences, Prodrugs, Orphan drugs, Nitrogen cycle, Sodium phenylbutyrate, Bioequivalence, Maple syrup urine disease, Food and Drug Administration, Pharmacokinetics

The single-center, single-blind, randomized, single-dose crossover trial evaluated bioequivalence (BE) of ACER-001 compared to BUPHENYL when administered under fed conditions in 36 healthy adults.

Key Points: 
  • The single-center, single-blind, randomized, single-dose crossover trial evaluated bioequivalence (BE) of ACER-001 compared to BUPHENYL when administered under fed conditions in 36 healthy adults.
  • The topline data from this trial showed ACER-001 to have similar pharmacokinetic (PK) profiles for both phenylbutyrate (PBA) and phenylacetate (PAA) compared to BUPHENYL under fed conditions.
  • If ACER-001 is approved by the FDA, we believe its unique formulation will provide clinicians with an alternative to existing sodium phenylbutyrate-based treatments.
  • ACER-001 is an investigational product being studied for the treatment of patients with UCDs and MSUD and has not been approved by FDA for any indication.

Relief Therapeutics and Acer Therapeutics Sign Option Agreement for Exclusivity to Negotiate a Collaboration and License Agreement for the Worldwide Development and Commercialization of ACER-001 for the Treatment of Urea Cycle Disorders and Maple Syrup

Retrieved on: 
Monday, January 25, 2021
Maple syrup urine disease, Health, Branches of biology, Sodium phenylbutyrate, Maple syrup, Maple, Acer Inc.

ACER-001 (sodium phenylbutyrate) powder is a taste-masked, immediate release proprietary formulation in development for the treatment of urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).

Key Points: 
  • ACER-001 (sodium phenylbutyrate) powder is a taste-masked, immediate release proprietary formulation in development for the treatment of urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).
  • Further, in connection with entering into the Option Agreement, Relief will make a $4.0 million loan to Acer.
  • Further, Acer will retain development and commercialization rights in the U.S., Canada, Brazil, Turkey and Japan.
  • Acer could also receive a total of $6 million in milestones based on the first European (EU) marketing approvals for UCDs and MSUD.

EQS-News: Relief Therapeutics and Acer Therapeutics Sign Option Agreement for Exclusivity to Negotiate a Collaboration and License Agreement for the Worldwide Development and Commercialization of ACER-001 for the Treatment of Urea Cycle Disorders and Mapl

Retrieved on: 
Monday, January 25, 2021
Maple syrup urine disease, Health, Branches of biology, Sodium phenylbutyrate, Maple syrup, Maple, Acer Inc.

ACER-001 (sodium phenylbutyrate) powder is a taste-masked, immediate release proprietary formulation in development for the treatment of urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).

Key Points: 
  • ACER-001 (sodium phenylbutyrate) powder is a taste-masked, immediate release proprietary formulation in development for the treatment of urea cycle disorders (UCDs) and Maple Syrup Urine Disease (MSUD).
  • Further, in connection with entering into the Option Agreement, Relief will make a $4.0 million loan to Acer.
  • Further, Acer will retain development and commercialization rights in the U.S., Canada, Brazil, Turkey and Japan.
  • Acer could also receive a total of $6 million in milestones based on the first European (EU) marketing approvals for UCDs and MSUD.

Acer Therapeutics Announces Full Enrollment of Pivotal Bioequivalence Trial of ACER-001 for Urea Cycle Disorders

Retrieved on: 
Tuesday, December 22, 2020
Nitrogen cycle, Health sciences, Branches of biology, Health, Urea, Sodium phenylbutyrate, Adherence

Acer is developing ACER-001 for the treatment of urea cycle disorders (UCDs).

Key Points: 
  • Acer is developing ACER-001 for the treatment of urea cycle disorders (UCDs).
  • The single-center, single-blind, randomized, single-dose crossover trial is designed to demonstrate the BE of ACER-001 compared to BUPHENYL under fed conditions, in approximately 36 healthy adults.
  • Suggested guidelines for the diagnosis and management of urea cycle disorders.
  • Barriers to drug adherence in the treatment of urea cycle disorders: Assessment of patient, caregiver and provider perspectives.Mol Genet Metab.2016;8:43-47.

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 .

Acer Therapeutics Announces Administration of ACER-001 in a Fasted State Increased Systemic Exposure of Phenylbutyrate in Healthy Volunteer Food Effect Study

Retrieved on: 
Wednesday, July 8, 2020
Drugs, Prodrugs, Nitrogen cycle, Sodium phenylbutyrate, Tolerability, Health care, Chemistry

Currently approved therapies for UCDs, including BUPHENYL2 and RAVICTI3 (glycerol phenylbutyrate), are required to be administered with food.

Key Points: 
  • Currently approved therapies for UCDs, including BUPHENYL2 and RAVICTI3 (glycerol phenylbutyrate), are required to be administered with food.
  • Based on the results from the food effect study within the ACER-001 BE trial, Acer commissioned Rosa & Co. LLC to create a PhysioPD PK model to evaluate the potential food effect on exposure, tolerability and efficacy of ACER-001 in UCDs patients.
  • For nearly a quarter century, phenylbutyrate has been prescribed to UCD patients with food while its effect on phenylbutyrate absorption was never determined.
  • This taste-masked formulation may result in better patient tolerability allowing for administration in a fasted state, and likely prior to a meal.