In response to a question in this morning’s webinar, Rohan pointed to two fairly recent deals (both in Dec 2022) of pre-clinical antisense drug candidates. I’ve written about these deals before but they’re probably worth revisiting. They provide insight into the value that has already been built into PYC's two lead assets.
Deal 1
The first deal was struck between Vertex and Entrada Therapeutics.
The focus of the deal was Entrada’s second asset, a CPP-conjugated PMO (ENTR-701) for the treatment of the rare disease, myotonic dystrophy type 1 (DM1), which has a US patient population of ~ 40,000. While there are no approved therapies for DM1, several companies lead Entrada in development. Nonetheless, the quality of the pre-clinical data package put together by Entrada convinced Vertex that it was worth backing ENTR-701. At the time of the deal, the asset was still approximately 12 months away from filing an IND submission – which is roughly 6 months behind where PYC-001 for ADOA is at.
Vertex paid an upfront of US$224m (AU$352m) and invested an additional US$26m (AU$40m) in Entrada. A further potential US$485m (AU$762m) in development and sales milestones was payable as well as tiered royalties in the mid to high single digits.
Vertex also assumed responsibility for funding of all further preclinical and clinical development, manufacturing, regulatory and commercialization of ENTR-701, plus additional DM1 research activities.
Entrada’s lead asset (in DMD) only entered the clinic late last month.
The company’s current market cap is US$520m (AU$817m).
https://www.fiercebiotech.com/biote...ease-drug-opening-new-front-assault-dystrophy
https://www.evaluate.com/vantage/ar...spotlight-vertex-flexes-its-dealmaking-muscle
Deal 2
The second deal was between GSK and Wave Life Sciences.
The companies agreed to a 4 year collaboration with two main components. The first part is a discovery collaboration which enables GSK to advance up to eight programs and Wave to advance up to three programs, leveraging Wave’s oligonucleotide discovery and drug development platform, PRISM. In addition. GSK received a global license for Wave’s preclinical program, WVE-006, a first-in-class RNA editing therapeutic designed to address both liver and lung manifestations of the inherited genetic disease, AATD.
Wave was to receive an upfront payment of US$170 m ($AU267m), which included a cash payment of US$120 m (AU$188m) and a US$50m (AU$78m) equity investment.
Under the terms of the deal for WVE-006, Wave is eligible to receive up to US$225 m (AU$353m) in development and launch milestone payments and up to US$300 m (AU$471m) in sales-related milestone payments, as well as tiered sales royalties. Wave was to take the asset to completion of the first in-patient study with GSK to assume all development and commercialization responsibilities thereafter. Dosing of the first patient is due to commence this quarter.
For each of GSK’s eight collaboration programs, Wave is eligible to receive up to US$130-$175 m (AU$204-275m) in development and launch milestones and $200 m (AU$314m) in sales-related milestones, along with tiered sales royalties for. Wave will take responsibility for all preclinical programs up to IND enabling studies at which point GSK is to take responsibility for all further development. The collaboration also included an option to extend the research term for up to 3 more years, expanding the number of programs available to both parties.
In comment at the time, GSK’s CSO said: “Oligonucleotide therapeutics are becoming a mainstream modality, and this collaboration will enable us to use our leading position in human genetics and genomics to advance novel oligonucleotide therapies.”
Wave Life Sciences current market cap is US$567 m (AU$891m).
https://www.fiercebiotech.com/biote...continue-ride-oligonucleotide-wave-years-come
Wave Life Sciences and GSK Announce Collaboration to Drive (globenewswire.com)
https://www.gsk.com/en-gb/media/pre...erapeutics-focusing-on-novel-genetic-targets/
(20min delay)