Member Info for onceaday

www.fiercebiotech.com/biotech/4dmt-will-regain-full-rights-to-blinding-disease-therapy-from-roche-to-initiative-new

"Another reason for Roche's exit may have been Adverum Biotechnologies' recent shaky safety results and a Biogen trial failure earlier this month. Those events have put a damper on the entire gene therapy field in eye diseases. "

Certainly seems like Gene therapy is challenging.

My experience of licensing deals is that, after the technology transfer phase is complete, the licensor may not have that much to do with the licensee's development program. In any case, you are absolutely right, RENE would be unable to disclose anything regarding FOSUN's development without explicit permission - not least because it would be market-sensitive information (for FOSUN).

I agree, neither study really matches. The first trial is using RPE (cells) rather than hRPC. The second is similar to the CTX trial but I would expect it to have explicitly stated if they were CTX cells.

Anyway, how's the food in the camp :-)

Edison research is paid for by the company so Vivien won't ever be asking difficult questions. However, based on PA's comments, seems she is buying a Porsche Taycan so the job pays well! :-)

Very interesting. I had a quick look back through the RNS catalogue and found this extract

JULY 20, 2020
Source: RNS
RNS Number : 4161T

"Clinical trial applications have recently been filed by Fosun Pharma to open clinical sites in the licensed territory to build on the Phase 2b clinical data already generated with the CTX cell therapy candidate for stroke disability...."

Assuming the regulator in China agreed, the CTX trial could have started at the beginning of this year. I'd estimate 3-4 years for headline results for a pivotal phase III. We could see approval in China as early as 2025.

Latest RNS out of hrpc

https://www.londonstockexchange.com/news-article/RENE/update-for-hrpc-therapy-candidate-study/15010809

https://www.bbc.co.uk/news/science-environment-57234610

Food for thought:

- switching to electric vehicles in the UK alone would require an entire year's world production of neodymium (to make electric motor magnets).
- replacing the estimated 1.4 billion internal combustion engine vehicles worldwide would need 40 times this quantity, and that's before the metal and mineral requirements of all the wind turbines and solar farms are considered.
- it takes a decade or more from finding material to actually mining the stuff.
- it isn't just about mines; it's the whole supply chain

Interesting article on RP on the BBC website

https://www.bbc.co.uk/news/health-57226572

Interesting article on jCyte's planned phase 3 - https://finance.yahoo.com/news/jcyte-inc-identifies-retinitis-pigmentosa-120000381.html

The RNS says the shares will be distributed and then shortly after listed on Nasdaq. I read that to mean it's the only exchange that the shares will be traded on...

With regard to TLS holders who have shares held in a SIPP or ISA that does not allow US investments then I'd expect the broker to either offer to sell them upon receipt or offer to transfer them into a brokerage account that allow foreign holdings. I use Interactive Investor which allows US holding is ISA, SIPP and their standard trading accounts.

Hopefully, they will make a few sales to large cancer centres in the US. I believe the last update was that several were in an evaluation phase.

It's quite a jump to go from medical imaging to drug development. I'm a little surprised the company was asked to be honest. Cancer developments are heavily backed by investors in the US - If early results are promising then I'd expect a partnership with big pharma or a US institutional investor(s) to follow.

The RNS mentions orphan designation which provides for an extended period of market exclusivity but arguably patent protection is more important because this can be for a longer period. It would be good to know who owns the IP (if it exists) for the compound . The very good news is that gallium nitrate has been evaluated for toxicity in animals and then for both toxicity and antineoplastic activity in humans in Phase I and II clinical trials. I would extrapolate from this that Gallium maltolate will have a similar safety profile in humans. This significantly de-risks its use as a drug - possibly why Trevor Brown decided to back it.

The RNS seems perfectly clear. They are not listing in London as originally intended, they are listing on NASDAQ. Accustem will need to raise further funds as I recall and NASDAQ is probably a better place to do this.

From the RNS "The board of directors of Accustem has resolved that the Nasdaq listing venue is more appropriate to the nature of Accustem's business."

I would consider an alternative provider. I use Interactive Investor and can hold US (and many other foreign) shares in my SIPP and ISA.

We should get a PDUFA date from the FDA in due course - that's on the proviso that they accept the NDA. I believe they have a month to decide whether to accept the submission.

Certainly very interesting stuff. His comment that it took 12 years to get from animal to human trials (in 2016) shows how long clinical development takes without a pandemic to move a program forward at pace! Also, interesting to note his view that repeat dosing may require immunosuppression - particularly relevant to jCell.

This is an review of stem cell research. The Foundation are very active in the funding of research.

Dr Mike Young's research is the basis of ReNe's treatment.. interesting to hear his objective, unbiased view of the trials.

www.youtube.com/watch?v=acbnWofwLJA

Does anyone know if Oxford has started recruiting? In early January, Olav stated that it was opening soon. It appears that's not the case based on the status showing on clinicaltrials.gov.

Olav also stated that Rene would be presenting 3 months RP data at the planned ASRS conference on July 24–26, 2021, in Stockholm, Sweden. However, this no longer seems to be showing on the ASRS site so I assume it has been canceled due to the Pandemic.

As a holder of the CVR's, I wasn't quite sure of the conditions attached. Found this in Amryt's half-yearly report. In case anyone is interested.

The terms of the CVRs are as follows:
• The total CVR payable is up to US$85,000,000
• This is divided into three milestones which are related to the success of FILSUVEZ (the Group’s lead development
asset, currently in Phase 3 clinical trials)
• FDA approval
o US$35,000,000 upon FDA approval
o 100% of the amount due if approval is obtained before December 31, 2021, with a sliding scale on a linear
basis to zero if before July 1, 2022
15
• EMA approval
o US$15,000,000 upon EMA approval
o 100% of the amount due if approval is obtained before December 31, 2021, with a sliding scale on a linear
basis to zero if before July 1, 2022
• Revenue targets
o US$35,000,000 upon FILSUVEZ revenues exceeding US$75,000,000 in any 12-month period prior to June 30,
2024
• Payment can, at the Board’s discretion, be in the form of either:
o 120-day loan notes (effectively cash), or
o Shares valued using the 30 day / 45-day VWAP

That's very good news for UK vaccine development. It's great to be a pioneer but It does beg the question of how long it will be before other countries follow suit? I imagine there are plenty of poorer countries where relatively small financial incentives would attract volunteers.

Sad but true. However, Krystal are using HSV-1 as the delivery mechanism for their EB trials.