--- title: "Ionis’ rare neurology drug approved; Pfizer inks an ADC deal" type: "News" locale: "en" url: "https://longbridge.com/en/news/298059860.md" description: "The FDA approved Ionis Pharmaceuticals' Zanvastro for Alexander disease, granting a priority review voucher. Pfizer and Medicus Pharma co-developed an ADC targeting melanotransferrin. Climb Bio reported positive Phase 1 data for CLYM116 in IgA nephropathy. Revolution Medicines published NEJM data supporting Phase 3 trials for Rasonque in lung cancer. Typewriter Therapeutics raised $56M Series A. Tscan Therapeutics cut 75% of staff to prioritize solid tumor programs." datetime: "2026-09-04T16:23:04.000Z" locales: - [zh-CN](https://longbridge.com/zh-CN/news/298059860.md) - [en](https://longbridge.com/en/news/298059860.md) - [zh-HK](https://longbridge.com/zh-HK/news/298059860.md) generator: "portal-rs" --- # Ionis’ rare neurology drug approved; Pfizer inks an ADC deal *Today, a brief rundown of news involving Ionis Pharmaceuticals and Pfizer, as well as updates from Climb Bio, Revolution Medicines, Typewriter Therapeutics and Tscan Therapeutics that you may have missed.* **The Food and Drug Administration** on Thursday approved the first medicine for an extremely rare, often fatal neurological condition known as Alexander disease. Developed by **Ionis Pharmaceuticals**, **Zanvastro** works by reducing the production of a protein that, in Alexander disease, forms toxic clumps that progressively damage the nervous system and lead to motor and cognitive dysfunction. The FDA based its decision on a study that found patients treated with Zanvastro were significantly more stable during a walking test. Ionis said the drug will become available in the U.S. “in the coming weeks.” It’s the first independent product launch from the company’s neurology pipeline. Its approval also earned Ionis a coveted regulatory fast pass known as a priority review voucher. *— Jacob Bell* **Medicus Pharma** and **Pfizer** have entered a co-development agreement to advance an antibody drug conjugate targeting melanotransferrin​​​​​, or CD228, the cell surface protein that binds iron and is expressed on melanomas and other tumor types. Medicus will retain an exclusive worldwide license to develop, manufacture and commercialize the ADC, while Pfizer will be responsible for funding and is eligible for development and regulatory milestone payments, according to the deal posted Wednesday. Medicus paid Pfizer $12 million up front, and is required to hand over an additional one-time payment of $15 million on the anniversary of the deal. *— Delilah Alvarado* Immune system specialist **Climb Bio** announced Thursday positive early data from an ongoing Phase 1 trial of its experimental anti-APRIL monoclonal antibody. Dubbed **CLYM116**, the drug is currently in development for the treatment of the kidney disease IgA nephropathy, or IgAN. Rather than binding to the cytokine APRIL like other medicines, Climb’s antibody is designed to block the cytokine’s activity and degrade it — which is implicated in B-cell-mediated diseases — as well as “recycle” the antibody to extend its half-life. The drug will continue to advance in a Phase 2 trial. *— Delilah Alvarado* **Revolution Medicines** said Wednesday data from a Phase 1/2 trial evaluating its drug **Rasonque**, otherwise known as daraxonrasib, against advanced RAS mutant non-small cell lung cancer were published in The New England Journal of Medicine. The drug has been high on companies’ radars after recently gaining speedy approval for the treatment of pancreatic cancer last month. It’s also been hailed as a paradigm-shifting treatment by Wall Street. Revolution has continued to study the drug in non-small cell lung cancer, and said the data published support an ongoing Phase 3 trial evaluating the drug against RAS-mutated lung cancer. *— Delilah Alvarado* **Typewriter Therapeutics** emerged from stealth mode Wednesday with $56 million from a Series A round led by **AN Venture Partners** and **RA Capital Management**. The Cambridge, Massachusetts-based company is built around a technology designed to insert whole genes into the genome to create better, re-dosable genetic medicines. Its initial focuses will be “in vivo” CAR-T and genetic liver diseases. Typewriter has non-human primate studies planned for later this year. The company also announced the appointment of **Matthew Stanton** as CEO. Stanton previously held leadership positions at **Moderna** and **Generation Bio**, and served as a venture partner at RA Capital’s healthcare incubator. *— Jacob Bell* **Tscan Therapeutics**, a Massachusetts-based biotechnology company, disclosed Wednesday plans to reduce its headcount by 75%. The job cuts are part of a strategic reorganization meant to prioritize the biotech’s in vivo solid tumor program — specifically, the advancement of two product candidates into studies that could pave the way to human testing. As its name suggests, Tscan focuses on T cell therapies for the treatment of cancer. The company had 142 full-time employees by the end of February, and had laid off around 30% of its staff last November. Over the first six months of this year, Tscan recorded a net loss of $59 million. *— Jacob Bell* ### Related Stocks - [RVMD.US](https://longbridge.com/en/quote/RVMD.US.md) - [IONS.US](https://longbridge.com/en/quote/IONS.US.md) - [CLYM.US](https://longbridge.com/en/quote/CLYM.US.md) - [TCRX.US](https://longbridge.com/en/quote/TCRX.US.md) - [MDCX.US](https://longbridge.com/en/quote/MDCX.US.md) - [PFE.US](https://longbridge.com/en/quote/PFE.US.md) - [MRNA.US](https://longbridge.com/en/quote/MRNA.US.md) - [GBIO.US](https://longbridge.com/en/quote/GBIO.US.md) - [RVMDW.US](https://longbridge.com/en/quote/RVMDW.US.md) - [MDCXW.US](https://longbridge.com/en/quote/MDCXW.US.md) ## Related News & Research - [Pelacarsen Trial Failure Might Change The Case For Investing In Ionis Stock (IONS)](https://longbridge.com/en/news/299511291.md) - [IONS: Breakthrough approvals and robust launches drive growth, with a deep pipeline advancing in CNS and metabolic diseases](https://longbridge.com/en/news/299192045.md) - [CLYM: CLYM116 and budoprutug advance with best-in-class data, Q12-week dosing, and pivotal trials ahead](https://longbridge.com/en/news/299196176.md) - [Health Canada authorizes Pfizer-BioNTech XFG-adapted COVID-19 vaccine](https://longbridge.com/en/news/299351665.md) - [IP Group (LON:IPO) Announces Quarterly Earnings Results](https://longbridge.com/en/news/299044088.md) --- > **Disclaimer: This article is for reference only and does not constitute any investment advice.**