Stepping on BioMarin's turf, PTC Therapeutics picks up PKU drug for a song
While industry-shaking M&A deals are still nowhere in sight, smaller deals by bargain shoppers continue to trickle in. PTC Therapeutics has joined the club, bagging Censa Pharmaceuticals and its rare metabolic disease drug.
The upfront cash comes in at just $10 million, though Censa investors might find more value in the equity portion of the deal: 850,000 shares of PTC $PTCT, worth a little over $40 million if you take the closing price on Wednesday. An additional $217.5 million is reserved for development and sales milestones for the two lead indications of CNSA-001.
With its oral spinal muscular atrophy therapy — jointly developed by Roche and the SMA Foundation — widely expected to be approved later this year, PTC now gets to launch a new Phase III program in phenylketonuria.
Caused by mutations in the phenylalanine hydroxylase gene, the disease is characterized by toxic buildup of the amino acid phenylalanine (Phe) in the brain.
As with SMA, even if it all goes well, PTC will be stepping on the turf of a much larger competitor. Cowen analyst Joseph Thome summarized it this way:
The PKU market is relatively competitive headlined by BioMarin’s Kuvan and Palynziq, with gene therapy approaches in early clinical development. In addition, generic versions of Kuvan are set to launch later this year, which may yield additional reimbursement hurdles. However, non-responders to generic Kuvan should be identified fairly quickly should treatment failure be required ahead of treatment with a branded agent.
Given that only roughly 30% to 40% of patients respond to Kuvan, there is room for a new option, he added.
The BioMarin drug replaces BH4, the PAH enzyme cofactor that’s deficient among patients with PKU. Censa’s approach goes a little upstream of the pathway, giving patients an oral formulation of synthetic sepiapterin, which is a precursor to BH4 in the cell.
In a Phase II trial where it was compared to Kuvan, CNSA-001 hit the statistical threshold for reducing blood Phe concentration.
PTC is now taking over the late-stage work from Censa, which was backed by Israel’s Arkin Bio Ventures, though it noted there is no obligation to hire any of their staffers.
“We believe that CNSA-001 has the potential to address the majority of PKU patients whose condition is not adequately managed by current treatments,” CEO Stuart Peltz said in a statement.