Acer Therapeutics Inc. (ACER - Free Report) , along with its partner Relief Therapeutics Holding AG, announced that the FDA has approved Olpruva, a formulation of sodium phenylbutyrate for oral suspension for the treatment of patients with urea cycle disorders (“UCDs”). The drug is approved for patients with UCDs involving deficiencies of carbamylphosphate synthetase (“CPS”), ornithine transcarbamylase (“OTC”), or argininosuccinic acid synthetase (“AS”).
The approval marks the first FDA nod to the company for any of its pipeline candidates.
Per the company, UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, which may lead to brain damage and neurocognitive impairments if not controlled.
The FDA approved Olpruva under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act (FDCA), a regulatory pathway that allows applicants to rely, at least in part, on third-party data for approval. Acer mentioned preclinical and clinical safety and efficacy data from the reference listed drug, Buphenyl powder, in its new drug application or NDA for Olpruva.
Buphenyl powder is also approved as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of CPS, OTC or AS.
Shares of Acer were down 33.3% on Tuesday following the announcement of the news. The stock has gained 8.8% in the year-to-date period against the industry’s decline of 29.7%.
We remind investors that in June 2022, the FDA issued a complete response letter (“CRL”) to Acer’s NDA for Olpruva to treat patients with UCDs.
The CRL was issued as Acer’s third-party contract packaging manufacturer facility was not ready for inspection. Hence, the FDA’s field investigator was unable to complete the inspection of this facility. The CRL cited that a satisfactory inspection was required to be carried out by the regulatory body before approving the drug in its current form.
The resubmitted NDA for Olpruva was accepted by the FDA in July. The regulatory body set had set an action date of Jan 15, 2023, but the decision has now come much earlier
Apart from UCD, Acer is also developing Olpruva to treat various other inborn errors of metabolism, including maple syrup urine disease.
Other than Olpruva, the company currently has other candidates in clinical development.
Edsivo (celiprolol) is being developed for vascular Ehlers-Danlos syndrome (“vEDS”) in patients with a confirmed type III collagen (COL3A1) mutation, while ACER-801 (osanetant) is being developed for the treatment of induced vasomotor symptoms (“iVMS”) and post-traumatic stress disorder (“PTSD”). Acer’s other candidate, ACER-2820 (emetine) is a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19.
Successful development of the pipeline candidates will be a big boost for the company.
Zacks Rank & Other Stocks to Consider
Acer currently carries a Zacks Rank #1 (Strong Buy). Other top-ranked stocks in the biotech sector are Syndax Pharmaceuticals, Inc. (SNDX - Free Report) , Celularity Inc. (CELU - Free Report) and Immunocore Holdings plc (IMCR - Free Report) , all carrying the same Zacks Rank #1 at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Loss per share estimates for Syndax Pharmaceuticals have narrowed 5.9% for 2022 and 14.5% for 2023 in the past 60 days.
Earnings of Syndax Pharmaceuticals surpassed estimates in three of the trailing four quarters and met the same on the other occasion. SNDX witnessed an earnings surprise of 95.39% on average.
Loss per share estimates for Celularity have narrowed 57.1% for 2022 and 7.7% for 2023 in the past 60 days.
Earnings of Celularity surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. CELU witnessed an earnings surprise of 51.01% on average.
Loss per share estimates for Immunocore have narrowed 56.1% for 2022 and 56.8% for 2023 in the past 60 days.
Earnings of Immunocore surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. IMCR witnessed an earnings surprise of 68.34% on average.
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Acer Therapeutics Inc. (ACER - Free Report) , along with its partner Relief Therapeutics Holding AG, announced that the FDA has approved Olpruva, a formulation of sodium phenylbutyrate for oral suspension for the treatment of patients with urea cycle disorders (“UCDs”). The drug is approved for patients with UCDs involving deficiencies of carbamylphosphate synthetase (“CPS”), ornithine transcarbamylase (“OTC”), or argininosuccinic acid synthetase (“AS”).
The approval marks the first FDA nod to the company for any of its pipeline candidates.
Per the company, UCDs are a group of rare, genetic disorders that can cause harmful ammonia to build up in the blood, which may lead to brain damage and neurocognitive impairments if not controlled.
The FDA approved Olpruva under section 505(b)(2) of the Federal Food, Drug and Cosmetic Act (FDCA), a regulatory pathway that allows applicants to rely, at least in part, on third-party data for approval. Acer mentioned preclinical and clinical safety and efficacy data from the reference listed drug, Buphenyl powder, in its new drug application or NDA for Olpruva.
Buphenyl powder is also approved as adjunctive therapy in the chronic management of patients with UCDs involving deficiencies of CPS, OTC or AS.
Shares of Acer were down 33.3% on Tuesday following the announcement of the news. The stock has gained 8.8% in the year-to-date period against the industry’s decline of 29.7%.
We remind investors that in June 2022, the FDA issued a complete response letter (“CRL”) to Acer’s NDA for Olpruva to treat patients with UCDs.
The CRL was issued as Acer’s third-party contract packaging manufacturer facility was not ready for inspection. Hence, the FDA’s field investigator was unable to complete the inspection of this facility. The CRL cited that a satisfactory inspection was required to be carried out by the regulatory body before approving the drug in its current form.
The resubmitted NDA for Olpruva was accepted by the FDA in July. The regulatory body set had set an action date of Jan 15, 2023, but the decision has now come much earlier
Apart from UCD, Acer is also developing Olpruva to treat various other inborn errors of metabolism, including maple syrup urine disease.
Other than Olpruva, the company currently has other candidates in clinical development.
Edsivo (celiprolol) is being developed for vascular Ehlers-Danlos syndrome (“vEDS”) in patients with a confirmed type III collagen (COL3A1) mutation, while ACER-801 (osanetant) is being developed for the treatment of induced vasomotor symptoms (“iVMS”) and post-traumatic stress disorder (“PTSD”). Acer’s other candidate, ACER-2820 (emetine) is a host-directed therapy against a variety of viruses, including cytomegalovirus, Zika, dengue, Ebola and COVID-19.
Successful development of the pipeline candidates will be a big boost for the company.
Zacks Rank & Other Stocks to Consider
Acer currently carries a Zacks Rank #1 (Strong Buy). Other top-ranked stocks in the biotech sector are Syndax Pharmaceuticals, Inc. (SNDX - Free Report) , Celularity Inc. (CELU - Free Report) and Immunocore Holdings plc (IMCR - Free Report) , all carrying the same Zacks Rank #1 at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Loss per share estimates for Syndax Pharmaceuticals have narrowed 5.9% for 2022 and 14.5% for 2023 in the past 60 days.
Earnings of Syndax Pharmaceuticals surpassed estimates in three of the trailing four quarters and met the same on the other occasion. SNDX witnessed an earnings surprise of 95.39% on average.
Loss per share estimates for Celularity have narrowed 57.1% for 2022 and 7.7% for 2023 in the past 60 days.
Earnings of Celularity surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. CELU witnessed an earnings surprise of 51.01% on average.
Loss per share estimates for Immunocore have narrowed 56.1% for 2022 and 56.8% for 2023 in the past 60 days.
Earnings of Immunocore surpassed estimates in three of the trailing four quarters and missed on the remaining occasion. IMCR witnessed an earnings surprise of 68.34% on average.
Just Released: Zacks Top 10 Stocks for 2024
Hurry – you can still get in early on our 10 top tickers for 2024. Hand-picked by Zacks Director of Research, Sheraz Mian, this portfolio has been stunningly and consistently successful. From inception in 2012 through November, 2023, the Zacks Top 10 Stocks gained +974.1%, nearly TRIPLING the S&P 500’s +340.1%. Sheraz has combed through 4,400 companies covered by the Zacks Rank and handpicked the best 10 to buy and hold in 2024. You can still be among the first to see these just-released stocks with enormous potential.
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