Originally posted by hottod
CEO Update 09/07/18
The recent investment by Sarepta Therapeutics in a pipeline of PMO therapies all joined to their CPP (known as PPMO or peptide-PMO therapies and within the class of cargoes known as ‘ASO’s – AntiSense Oligonucleotides) is testament to the increasing preference for CPP based delivery vehicles in certain settings. The lead therapy in this pipeline is currently in Phase1/2A human clinical trials and has led to the observation that ‘we will see more CPP-ASO candidates move towards clinical drug development. The opportunity for Phylogica is to clearly demonstrate the competitive advantage of our CPPs in this context……..
On 1 June 2018 we held the first formal review of our platform with our newly appointed Scientific Advisory Board. It was clear from this discussion that Phylogica has a near-term opportunity to improve the commercial attractiveness of our platform by showing that we can deliver a cargo class of interest into a target cell of interest more efficiently than the relevant competitive technology. The outcome of these comparative studies and progression into clinical evaluation are the two primary drivers of platform validation and therefore the value of the company’s assets. Realisation of this competitive assessment requires selection of a cargo since joining the CPP to a cargo has the ability to change the properties of both elements of the ‘conjugate’ (CPP and cargo). The extension of our 2017 strategic review is to now move from ‘proof of concept’ to therapeutic cargoes and to:
obtain a competitive read-out with the existing delivery modality for that cargo class to show greater efficacy (more cargo delivered) and/or lower toxicity; and to do so
in primary cells (ex vivo) or in a relevant in vivo setting
Annual Report 21/09/18
Forward View of 2019
The cargo classes that have been identified as being of particular interest to Phylogica in progressing into the clinic include:
- Anti-Sense Oligonucleotides;
- CRISPR/Cas9; and
- Peptide vaccines.
We will deliver the competitive read-out relevant to an exemplar cargo from within each class in FY19. We will also initiate IND-enabling studies for CPP-cargoes in which we can demonstrate out-performance of the current gold standard putting us within reach of the clinic…..
Phylogica currently has a number of Materials Transfer Agreements (MTAs) in place with commercial counter-parties (including for the cargoes identified above) that enable the counter-parties to evaluate the ability of our CPPs to deliver their cargoes into their target tissues of interest in their own hands. These agreements are set move to the next stage in CY19 with successful outcomes expected to translate into licensing agreements as described in the former development category outlined above.
AGM Presentation 16/11/18
Sarepta have taken promising preclinical results driven by Cell Penetrating Peptides (CPPs)…
…into the clinic.
- CPP-PMO (PPMO) is now in Phase 1 trials
- multiple follow on CPP-PMO programs are scheduled for IND in 2019
Efficiency of delivery remains the rate-limiting step in the field...
Phylogica has a major competitive advantage in CPPs...
Our CPPs deliver more cargo inside cells than competitor CPPs...
We will soon realise a major milestone in validating our platform...
We will achieve this objective with an Anti-Sense Oligonucleotide (ASO) cargo.
Summarising the above statements made by the Company over the past few months:
- PYC’s lead program will deliver an Anti-Sense Oligonucleotide (ASO) cargo inside cells.
- PYC will deliver a “competitive read-out” of an ASO cargo in FY 2019 aiming to demonstrate outperformance (more efficient delivery) compared with “the relevant competitive technology”.
- Sarepta Therapeutics’ PPMO technology is currently the most relevant competitive technology within the cargo class of ASOs.
- Therefore, PYC will need to obtain a competitive readout of FPP-PMO delivery with Sarepta’s proprietary CPP-PMO (PPMO) delivery.
- PYC will need access to Sarepta’s PMOs for its competitive read-out study.
- Phylogica has stated that it has a Material Transfer Agreement in place with a commercial counter-party with respect to relevant ASOs.
- To access Sarepta’s PMOs or PPMOs for its competitive read-out study, PYC would need an MTA with Sarepta.
Therefore, in my opinion, it is reasonable to assume that Sarepta is already aware of PYC and its connection to PYC involves more than just “sending an invoice for a PMO.”
There is a sequence of steps to arrive at the following statement, "to access Sarepta’s PMOs or PPMOs for its competitive read-out study, PYC would need an MTA with Sarepta. Of course, one of the steps could be misspoken. It is however, also a logical deduction given the reference to outperformance compared with “the relevant competitive technology”.
My question.
Why would Sarepta give access to its PMOs under an MTA in order for Phylogica to use the data to begin and/or advance negotiations with competitor biotechs?
Of course the premise could be wrong and no MTA is in place. Perhaps it is possible to access a molecule under alternative scenarios as in the case with OmoMyc. Perhaps there is no intention to run competitive readouts with Sarepta's PMOs. If, however, an MTA is in place what are the commercial implications?
(20min delay)