PTC Previews Key Spring Readouts in Full-Year 2022 Report

 PTC Therapeutics reported Q4 and full-year 2022 results Tuesday ahead of a pivotal 2023 featuring three late-stage readouts in registration-directed trials.

In an investor call, Stuart Peltz, Ph.D., founder and CEO, said the company is anticipating results from the Phase III APHENITY trial evaluating sepiapterin in phenylketonuria (PKU) in May 2023. APHENITY is comparing the candidate’s efficacy against placebo in 178 PKU patients. The study was initially scheduled to return results by the end of Q4, 2022.

PTC gained access to sepiapterin in 2020 when it bought Censa Pharmaceuticals in a cash-and-stock deal valued at $51 million upfront.

The New Jersey biotech also expects key data this spring from vatiquinone, an investigational small molecule inhibitor of the 15-lipoxygenase enzyme, which is central to inflammatory response and oxidative stress pathways in neurological diseases.

PTC is testing vatiquinone in a Phase II/III trial for mitochondrial disease-associated seizures, for which results are expected in the second quarter of 2023.

The candidate is also being trialed for Friedreich’s ataxia in the Phase III study MOVE-FA. Findings from this study are also anticipated in the second quarter of this year.

The company also expects to complete enrollment in the Phase II PIVOT-HD trial of PCT518 in Huntington’s disease. Data from the first 12 weeks of PIVOT-HD are also expected to roll in during the second quarter of 2023, Peltz said.

Shooting for 30% Growth in ‘23

On Tuesday, Peltz set the company’s goals for the coming year, saying that PTC expects to “continue to accelerate” its growth in 2023 and is gunning for total revenue between $940 million and $1.0 billion, “which would represent a more-than 30% year-over-year growth.”

In 2022, the New Jersey biotech recorded $740 million in revenue. At constant exchange rates, this represents a 37% year-over-year increase.

Much of PTC’s strong performance was driven by its Duchenne muscular dystrophy (DMD) franchise. Translarna (ataluren), an oral DMD therapeutic approved in the European Union, earned the company $288.6 million.

PTC’s U.S.-approved DMD drug is Emflaza (deflazacort), an oral corticosteroid greenlit by the FDA in 2017. Emflaza recorded $218.3 million in net product revenues in 2022.

Outside of its DMD business, PTC continues to earn royalties from spinal muscular atrophy drug Evrysdi (risdiplam), which it developed in collaboration with Roche. Evrysdi’s royalty revenue for 2022 was $113.5 million.

https://www.biospace.com/article/ptc-previews-key-spring-readouts-in-full-year-2022-report/

JPMorgan Curbs Employee's Use Of AI-Powered ChatGPT

 Since ChatGPT became available for public use late last year, it's created numerous questions for employers about use cases and how to integrate the tool into the workplace while not breaching compliance. 

ChatGPT is an artificial intelligence language platform where workers can write emails, produce code or even write reports in minutes, if not seconds. But the use in the workplace could be coming to a close for some employers. 

JPMorgan Chase & Co. is one of the first and highest-profile companies to ban the use of ChatGPT in the workplace, according to Bloomberg, citing a person with direct knowledge.

The person said the reason for the move stemmed from the use of using third-party software and wasn't tied to any specific incident.

We wonder just how many JPMorgan employees used the artificial intelligence language platform to create stock portfolios, write market reports, and construct emails for clients. 

Here's ChatGPT's response when asked to write a stock market outlook for 2023: 

"In the short-term, the market can be affected by many unpredictable events such as natural disasters, geopolitical tensions and so on. But in the long-term, it tends to be driven by the underlying health of the economy." 

But the technology isn't perfect. And some tests found ChatGPT's responses were factually incorrect or had grammatical errors, to name a few -- all of which could've been problematic for JPMorgan employees. 

Although ChatGPT can introduce workplace efficiencies, third-party software has many legal risks. JPMorgan might spark a trend among other companies to restrict chatbots in the office.

https://www.zerohedge.com/markets/jpmorgan-curbs-employees-use-ai-powered-chatgpt

Cognition, ACTC OKd for Phase 2 START Study of Oral CT1812 in Early Alzheimer’s

  Cognition Therapeutics, Inc. (NASDAQ: CGTX) announced that it has cleared the U.S. Food and Drug Administration comment period and may now proceed with the 540-patient Phase 2 START study of CT1812 in adults with mild cognitive impairment or early Alzheimer’s disease. This randomized placebo-controlled trial will be conducted at approximately 50-to-60 sites in North America including those premier institutions in the Alzheimer's Clinical Trials Consortium (ACTC). CT1812 is an investigational oral small molecule that binds to a receptor on synapses and is designed to prevent the toxic effects of soluble beta amyloid (Aβ) oligomers on neurons, which drive the progression of neurodegenerative diseases such as Alzheimer’s disease.

https://finance.yahoo.com/news/cognition-therapeutics-actc-receive-fda-130000668.html

AstraZeneca wins three approvals in EU for cancer drugs

 AstraZeneca PLC on Wednesday said its Imfinzi and Imjudo were approved in the European Union for the treatment of advanced liver and lung cancers. Its Calquence was also approved to treat chronic lymphocytic leukaemia.

The Cambridge, England-based pharmaceutical firm said its Imfinzi and Imjudo immunotherapy combinations were approved by the European Commission based on positive results from the Himalaya Phase III trial.

Astra said the approvals authorise Imfinzi, also known as durvalumab in combination with Imjudo, whose generic name is tremelimumab, for first-line treatment of adult patients with advanced hepatocellular carcinoma. Moreover, Imfinzi combined with Imjudo and platinum-based chemotherapy were authorised for the treatment of adult patients with metastatic non-small cell lung cancer.

Dave Fredrickson, executive vice president of AstraZeneca's Oncology business unit said: "Lung cancer is one of the most prevalent and deadly cancers in Europe, and rates of liver cancer are rising steadily across the region. These approvals for Imfinzi and Imjudo bring critically needed, novel immunotherapy-based treatment regimens that will potentially deliver life-extending benefits for European patients with advanced liver and non-small cell lung cancers."

Imfinzi is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, while Imjudo is a human monoclonal antibody that targets the activity of cytotoxic T-lymphocyte-associated protein.

Separately, AstraZeneca said its tablet formulation of Acalabrutinib, sold under the brand name of Calquence, has been approved in the EU for the treatment of adult patients with chronic lymphocytic leukaemia.

Chronic lymphocytic leukaemia is a rare type of cancer that affects the blood and bone marrow.

The company said the approval was based on results from the Elevate-Plus trials, conducted in 116 healthy subjects.

Fredrickson added: "The Calquence tablet formulation underscores our commitment to understanding the needs of the chronic lymphocytic leukaemia community and providing patient-oriented treatment solutions. Today’s approval offers physicians and patients in the EU more flexibility to determine the right treatment plan and enables more patients to potentially benefit from this medicine."

Back in August, Calquence was approved in the US. Calquence is a selective inhibitor of Bruton’s tyrosine kinase. Calquence binds covalently to BTK, thereby inhibiting its activity. In B cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis and adhesion.

https://www.marketscreener.com/quote/stock/ASTRAZENECA-PLC-4000930/news/AstraZeneca-wins-three-approvals-in-EU-for-cancer-drugs-43049151/

Sage: Euro orphan status for Treatment of Huntington’s Disease

 Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the European Medicines Agency (EMA) granted Orphan Drug Designation to SAGE-718 for the treatment of Huntington’s disease (HD). SAGE-718 is in development as a potential oral therapy for cognitive disorders associated with NMDA receptor dysfunction. Multiple clinical studies are ongoing with SAGE-718 across several disease areas, including two placebo-controlled Phase 2 studies and a Phase 3 open-label safety study in the lead indication of HD-related cognitive impairment, and additionally Phase 2 placebo-controlled studies in mild cognitive impairment (MCI) associated with Parkinson’s disease (PD) and MCI and mild dementia due to Alzheimer’s disease (AD).

https://finance.yahoo.com/news/sage-therapeutics-announces-european-medicines-113000814.html

Pfizer’s Elranatamab Receives FDA and EMA Filing Acceptance

Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) has granted Priority Review for the company’s Biologics License Application (BLA) for elranatamab, an investigational B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb), for the treatment of patients with relapsed or refractory multiple myeloma (RRMM). Priority Review is intended to direct attention and resources from regulatory authorities toward drugs that, if approved, could offer significant improvements over existing options for serious conditions in order to make these drugs available to patients faster. The FDA’s decision on the application is expected in 2023. The European Medicines Agency (EMA) has also accepted elranatamab’s marketing authorization application (MAA). The company is working closely with the EMA to facilitate their review and will provide updates on timing as appropriate.

https://finance.yahoo.com/news/pfizer-elranatamab-receives-fda-ema-114500184.html

Axsome in License Agreement with Pharmanovia to Expand Narcolepsy Therapy

 Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing and delivering novel therapies for the management of central nervous system (CNS) disorders, today announced that it has entered into an exclusive license agreement with Pharmanovia to commercialize and further develop Sunosi® (solriamfetol), the first and only dual-acting dopamine and norepinephrine reuptake inhibitor shown to improve wakefulness in adults living with excessive daytime sleepiness (EDS) due to narcolepsy or obstructive sleep apnea (OSA), in Europe and certain countries in the Middle East and North Africa (MENA).

Under the terms of the agreement, Pharmanovia will be responsible for marketing Sunosi in Europe and MENA regions and will assume responsibility for all local clinical and regulatory activities and requirements including studies in pediatric patients. Axsome will receive an upfront payment of $66 million and is eligible to receive sales-based and other milestones totaling up to $101 million. Axsome will receive a royalty percentage in the mid-twenties on net sales.

https://finance.yahoo.com/news/axsome-therapeutics-enters-license-agreement-120000176.html