The company said (22/02) that a CNS drug candidate targeting a high unmet need neurodegenerative condition was expected to be named in H1.
I was thinking about PYC’s neurodegenerative disease drug candidate just yesterday. This was prompted by media coverage of two infants, one identified at birth via a heel prick test to have Spinal muscular atrophy (SMA) and one who wasn't tested and whose condition wasn’t picked up before four months. (SMA is an autosomal recessive neurodegenerative disease and one of the most common genetic causes of infant death.)
The early-tested infant commenced treatment with Biogen’s Spinraza, an antisense oligonucleotide therapy licensed from Ionis Pharmaceuticals and approved in the treatment of SMA by the FDA in 2016 and EMA in 2017. The Spinraza-treated infant is meeting milestones at eight months. At five months, the other infant is already on a feeding tube and receiving breathing support.
Biogen released long-term follow-up data on Spinraza mid-last year. This is from their announcement at the time
In infants genetically diagnosed with SMA, new data demonstrate that early and sustained treatment with SPINRAZA for up to 4.8 years enabled unprecedented survival. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease….
As of February 2020, all patients treated (n=25; median age of 3.8 years old) were alive and remained free of permanent ventilation. In the absence of treatment, the majority of children with SMA Type 1 would, on average, not reach their second birthday. Additionally, all children who achieved the motor milestone of being able to walk independently (many within a normal timeframe) have maintained that ability from the first occurrence until the last visit.
Spinraza’s stunning success (financial as well – 2019 and 2020 sales were US$2bn+) highlights the potential of antisense therapies for the treatment of neurodegenerative disease.
I’m looking forward to PYC’s announcement of its chosen drug candidate.
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