During Rohan Hocking’s AGM presentation today he mentioned, I think in response to a gentleman’s question about probability of pharma interest, a number of deals that had been done for early-stage antisense therapy candidates.
While I posted about these deals at the time, the details might be of interest to those who have come to this stock more recently or those like me who forget the details!
In December 2022, Vertex acquired a preclinical oligonucleotide drug from Entrada for the treatment of myotonic dystrophy type 1 (DM1). The estimated US population with this condition is estimated to be ~40,000. Entrada’s oligonucleotide drug utilises their cell penetrating peptide platform to enhance intracellular delivery.
Vertex paid Entrada Therapeutics US$224 million upfront and also made a US$26 investment in the company. Entrada became eligible to receive up to US$485 million for the successful achievement of certain research, development, regulatory and commercial milestones, and tiered royalties on future net sales for any products that might result from the collaboration agreement.
The agreement included a four-year global research collaboration whereby Entrada would continue to advance and receive payments for certain research activities related to the DM1 drug, as well as additional DM1-related research activities. Vertex was to be responsible for global development, manufacturing and commercialization and any additional programs stemming from Entrada’s DM1 research efforts.
Almost a year on, Entrada is yet to file an IND to commence clinical trials.
Also in December 2022, GSK entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets.
The collaboration included two main components.
The first was a discovery collaboration which enabled GSK to advance up to eight programmes and Wave to advance up to three programmes, leveraging Wave’s PRISM platform and GSK’s expertise in genetics and genomics. The discovery collaboration had an initial four-year research term.
Under the terms of the agreement, Wave was to receive an upfront payment of US$170 million, which included a cash payment of US$120 million and a US$50 million equity investment.
For each of GSK’s eight collaboration programmes, Wave was eligible to receive up to US$130-$175 million in development and launch milestones and US$200 million in sales-related milestones, along with tiered sales royalties. Wave was to lead all preclinical research for GSK and Wave programmes up to IND enabling studies. GSK collaboration programmes were to transfer to GSK for IND-enabling studies, clinical development, and commercialisation. The collaboration included an option to extend the research term for up to three additional years, expanding the number of programmes available to both parties.
In addition to these programmes, GSK received the exclusive global license for Wave’s preclinical asset, WVE-006, a first-in-class RNA editing therapeutic designed to address both liver and lung manifestations of the inherited genetic disease, AATD.
For the WVE-006 programme, Wave was eligible to receive up to US$225 million in development and launch milestone payments and up to US$300 million in sales-related milestone payments, as well as tiered sales royalties. Development and commercialisation responsibilities were to transfer to GSK after Wave completed the first-in-patient study.
In January 2022, Acadia Therapeutics announced a collaboration with Stoke Therapeutics to discover, develop and commercialise antisense therapies targeting three rare neurodevelopmental diseases.
Acadia paid US$60m upfront and Stoke became eligible to receive up to US$907 million in milestones as well as royalties on future sales.
Stoke’s leading compound, which targets Dravet syndrome, was in a Phase 1/2 trial. Stoke had only recently released interim data from that trial, suggesting that the drug was safe and demonstrating efficacy signals at the earlier, lower doses. The US population for Dravet syndrome population appears to be less than 20,000.
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