Here is the part about PYC in the Listcorp.com article "Movers & Shakers - Pharma & Biotech" 11-Sep-2024
PYC Therapeutics Limited (ASX: PYC)PYC Therapeutics share price was up 48.9% in August. During
the month, the company provided an update on the clinical
trials for its drug candidate VP-001 for the treatment of patients
with a blinding eye disease called Retinitis Pigmentosa type 11
(RP11). The trials include a Single Ascending Dose (SAD) clinical
trial with an open-label repeat dose extension arm and a
Multiple Ascending Dose (MAD). The clinical trials are designed
to establish a proof of concept for VP-001 to progress to a
registrational study in 2025.
The company has completed dosing in the SAD trial in 4 cohorts
of patients with an SDA trial extension at the two higher doses
and commenced the MAD trial. To date, the drug has been safe
and well tolerated in the small cohort of patients treated in the
Phase 1 trials with improvement reported in the vision in 2 out of
the 3 patients after a singe dose of VP-001 at 30 microgram dose
and 75 microgram dose was received providing the company
confidence to move forward with planning for the registrational
study.
RP11 is a childhood disease affecting 1 in 100,000 people which
is caused by a mutation in 1 copy of the PRPF31 gene leading
to a protein insufficiency in photoreceptor and Retinal Pigment
Epithelial cells. VP-001 is designed to increase expression of
PRPF31 back to unaffected levels.
VP-001 is the first drug candidate to progress to human clinical
trials for RP11 and has been grated fast track status by the
FDA. With an addressable market of more than $1 billion and
no available treatment means success of the clinical trials and
progress to approval would be significant for the company.
During the month, the company also announced that the FDA
had granted Rare Pediatric Disease (RPD) designation for its drug
candidate PYC-001, which seeks to treat a blinding eye disease
called Autosomal Dominant Optic Atrophy (ADOA). ADOA affects
1 in 35,000 people and there is currently no treatment option
available for patients with ADOA. The RPD designation means
that if the drug is approved the company would be eligible
for a Priority Review Voucher (PRV) providing the potential for
additional value to the company.
The company had $66.9 million in cash as at 30 June 2024
providing over 4 quarters of cash runway at the current quarterly
run rate with positive results from the clinical trials likely to
trigger a capital raise.
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