BTX Sub Asterias Highlights (Conf Call).
-> Market Opportunity for OPC1:
"Previously published data from key opinion leaders in the spinal cord injury field have shown that the two motor level improvement in hand or in finger function that we're targeting in the SCiSTAR trial which as Mike mentioned earlier can be driven by only four-centimeter long restoration of conduction. Can drive substantial improvements for these patients in independence, quality of life, employability and lifetime cost of care. As a result, we estimate that the market potential for OPC1 is substantial and asset of $1 billion annually in the United States alone."
-> The 10 Million Cell AIS-A cohort is still stable and improving (safe and effective) (at 12 months target was to get at least two motor levels on at least one side, in practice results were 67% 4/6).
"Our previous efficacy data readout from the SCiSTAR show a promising motor function recovery at three months with maintained or additional improvement observed through six and nine months post-treatment for subjects in the AIS- A 10 million cell cohort or Cohort 2 of the study.
We are very pleased to see that the longer term data continues to convincing results. And we believe shows OPC1's potential will generate durable clinical benefit for patients with severe spinal cord injuries. In October, we reported positive 12-month follow-up data from the cohort. Specifically, the result showed, that previously reported meaningful improvements in arm, hand and finger function have been sustained and in some cases further improved at 12-month following administration of OPC1."
-> Cohorts 3+4 done, 5 - halfway to completion.
"During the third quarter, we completed enrollment and dosing Cohorts 3 and 4 and we're now more than halfway done with enrollment of the fifth and final cohort."
-> Cohorts 3+4 data is coming (Q1 - 6M update) and it is getting exciting (its here that we might see surprising results as the dosage is higher and the scope of injury is less severe than in cohorts 1+2).
"We expect to provide six months readout from Cohorts 3 and 4 in the first quarter of 2018 and 12 months readout during the third quarter of 2018. This is an important development for Asterias."
-> FDA Granted Regenerative Medicine Advanced Therapy (RMAT) to OPC1, is validating.
"The FDA granted OPC1 the Regenerative Medicine Advanced Therapy or RMAT designation is an expedited program established under 21st Century Cures Act to walk through the development and approval of important new regenerative medicine therapies intended for the treatment of serious of life-threatening diseases and conditions. We're gratified by the FDA's interest in the therapeutic potential of OPC1 and greatly appreciate their decision to provide support to accelerate its regulatory review and potential approval."
-> next step is to formulate a PII trial.
-> 12m data has sparked interest from Japan (where regulation only calls for Safety data and leaves efficacy for the free market). Discussions are in progress.
"those discussions are definitely continuing to progress on - and we're in conversations with multiple parties and even into confidential [indiscernible] for some of them, following the 12-month data readout we've also had some additional inbound interest from new parties as well. So those discussions are making good progress as you know any business development relationship does that sometimes to secure, but we will definitely provide an update as their timeline."
-> Cost Cutting and cash management (BTX being the boss - their interests align with all investor - have the Sub use as little cash and generate as much progress!
"our monthly cash burn has been a little over $2 million recently and so we're taking that 40% hair cut off with that. So we actually think our cash burn for 2018 on average will be about $1.3 million per month. So we're looking at 2018 annual burn rate of between $15 million and $16 million."
-> RMAT designation under the new law is new, and no one knows what the FDA will want for approval. OPC1 is leading the approval race.
" it's too early to say exactly what FDA would require to ultimately achieve approval".
Conclusion: Nice and steady. good progress is made and in Q12018 we might be in for a nice surprize and great PR. imagine another story similar to what we saw in ASI A quadriplegic moving his hands. Good solid progress.
