Syntara – it’s a name that biotech investors will be hearing a lot in the years to come with the Company set to accelerate clinical development of its novel drugs to treat blood cancers. 

Syntara* will be the new name for Pharmaxis (ASX: PXS) which has sold its mannitol respiratory business and is pursuing an exciting, advanced clinical program of drug development. The mannitol sale is expected to save $14 million annually allowing the company to focus on key assets including cornerstone drug PXS-5505 which has reported final interim data and is on the cusp of a valuation growth lever. 

The sale of the mannitol business has now been settled, with Arna Pharma having taken over the day-to-day operations setting the path for Syntara to accelerate PXS-5505 to commercialisation. Syntara will continue to receive ongoing royalties from Arna Pharma’s Sydney-based business, which includes products like Bronchitol, an approved cystic fibrosis treatment, and Aridol, an asthma diagnostics test.

By focusing its resources towards clinical drug development, Syntara is poised to tackle a substantially larger commercial market in blood cancer treatments and leverage its established clinical expertise. The revenue of the mannitol business which was $5.8 million in FY 2023 has not been sufficient to cover the fixed operating costs resulting in a loss before one-off items of over $5 million for the year. Expected growth from the US market had not as yet eventuated so the decision was made to sell the mannitol business in order to lower costs and better leverage infrastructure.

In contrast, PXS-5505 has shown the potential to modify bone marrow cancer in a way no other drug in the world can and addresses a market where current treatments generate more than USD $1 billion per annum. 

When queried by a shareholder on an investor call in April about how much of that market PXS-5505 could attract, CEO Gary Phillips expressed confidence in targeting all of it.  He remarked on the disease modification potential and good safety profile of the drug in data to date and explained how it can be added to the existing standard of care. Myelofibrosis patients are in need of new treatments and currently have an average life expectancy of just five years from the time of diagnosis. 

“We’ve had three Nature publications published over the last two years on the role of Lysyl Oxidases in many different diseases. This is an outstanding achievement for a small group to achieve, representing global leadership in this field,” said Phillips. 

“We’ve now proven beyond doubt I think, that inhibiting lysyl oxidase enzymes does produce a strong antifibrotic effect in two diseases; myelofibrosis and also skin scarring

“The new company starts with a really strong scientific base with a proven technology and a drug that’s already on its way to generating Phase 2 clinical data, which will be fundamental to a much increased valuation of the asset going forward.”

With Phase 2a clinical data that has shown to reduce fibrosis score in patients, PXS-5505 has drawn substantial interest from medical researchers around the world. Many of those researchers will be in attendance at the American Society of Hematology Conference in December where Syntara will present final data from its Phase 2a clinical trial with PXS-5505 as a stand alone treatment for patients with myelofibrosis. 

This market has grown notably in recent years with Syntara drawing attention to M&A activity around the last six companies that generated Phase 2b or 3 data for their myelofibrosis drugs, all exited with deals worth over USD $1 billion at the time of acquisition.

Further addressing shareholder speculation, Phillips shut down any prospect of selling PXS-5505 in the near-term future with the company keen to extract the full potential of the drug, a value that could be realised by clinical data from the Phase 2 combination clinical trial about to commence. 

Cognisant of the commercial pathway, the Company opened dialogue with the FDA in June 2023 which will lead PXS-5505 to an FDA-agreed study design that combines it with a JAK inhibitor, the current standard of care. The program is set to enrol its first patient in the fourth quarter of 2023, with interim data expected by the end of 2024. 

Further leveraging PXS-5505’s value in haematological malignancies and blood-related cancers, Syntara has also flagged plans to advance clinical trials to treat Myelodysplastic Syndrome (MDS). Discussions around a clinical program for MDS are ongoing with the program firmly on Syntara’s agenda. 

Preclinical data targeting MDS has already elicited excitement from clinical and industry thought leaders, with the data published in esteemed medical journal, Nature Communications. 

In addition to PXS-5505, Syntara will advance other programs, including oral and topical pan-LOX inhibitors in Phase 2 trials for scar prevention and scar modification. These programs are part of a collaboration with Professor Fiona Wood and the University of Western Australia, which reported positive results in May 2023. Furthermore, Syntara will continue a trial with PXS-4728, a novel anti-inflammatory drug candidate for early-stage neurodegenerative diseases, which is significantly funded by Parkinson’s UK.

Syntara welcomes investors to register for updates on their latest developments and industry research by joining their mailing list here.

*The formal renaming to Syntara from Pharmaxis is subject to shareholder approval on 28 November 2023.