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I think this is more aimed at rare genetic diseases and malignancies, so CRISPR gene editing and CAR-T therapies and suchlike, but may include rare cancers, although the UK has an equivalent of the US's ODD (called Orphan Medicinal Product Designation), as does the EU: https://www.google.com/search?q=what+is+the+UK+equivalent+of+the+US's+orphan+drug+designation

Anyway, this is what the boss of the MHRA said:

"A significant set of reforms that we're signalling to take place for next year and this is all about giving hope to those many patients, as you say two and a half million patients in this country, who have one or more rare diseases and for whom in the past they've had relatively few treatment options. So the reforms we're signalling today, to come into force next year, will allow us to speed many of these new medicines that have been developed with the science we have today of personalised medicine and our ability to manipulate the cells and the genes of the individual to treat their rare diseases."

It's not a 'narrow therapeutic index', it's either a narrow therapeutic window or a low therapeutic index, you smug moron.

I'm not predicting anything. I'm just stating a fact. You should try it. It might improve your credibility, although you've probably gone too far down Twat Street for anyone to ever take you seriously.

Wtf is a "narrow therapeutic index"?

There hasn't been a head to head with standard doxorubicin in any indication you moron.

I don't know whether you are obtuse or just really, really thick.

There hasn't been a head to head with standard doxorubicin you moron.

Three things are needed for success: science that works; results that are statistically significant; and funds to get to the company's desired endpoint.

If the desired endpoint is short-term in the development-to-market process, a takeout will be at a low price as the risk will be taken on by the buyer.

If the desired endpoint is longer-term, the need for additional funding will dilute the equity but, on statistically reliable results, any takeout will be at a much higher price.

Essentially, it's really that simple. So what is Vlad's desired endoint? It's his intellectual property and his company - he is the brains of the organisation.

30 Sep 2025: https://www.lse.co.uk/rns/AVCT/interim-half-year-results-2025-xtgyescwh7u4pla.html

The RNS was issued on Monday 27 October at 07:00

https://www.londonstockexchange.com/news-article/AVCT/avacta-presents-preclinical-data-at-aacr-nci-eortc/17296010

The same announcement was released as a Globe Newswire press release (what passes for an RNS in the US), which was picked up and reproduced by the Financial Times, etc.

https://www.globenewswire.com/news-release/2025/10/25/3174179/0/en/Avacta-presents-first-preclinical-data-from-dual-payload-pre-CISION-medicines-at-the-2025-AACR-NCI-EORTC-International-Conference-on-Molecular-Targets.html

https://markets.ft.com/data/announce/detail?dockey=1330-1001135036en-0MVP9R4RGF54UDQ95KPC6A078M

https://uk.finance.yahoo.com/news/avacta-presents-first-preclinical-data-171400537.html

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I'm 100% sure they will be optimising the capping group and linker of the dual-payload AVA6207. Only Compound A, Compound B, etc. were presented on the poster but, if anything like with AVA6103, they will be deciding on the compound to take forward at some later time, maybe Compound X.

No, it's one of the national priorities specified in this CNPV program. CC said manufacture in the US applied to tariffs on marketted products only. AVA6000 is a research product, but large scale production for the marketted product will no doubt be done in the US.

Very much, I'd say. This week's update of the NCT04969835 record for AVA6000 removed the 'NO' answer from the Manufactured in the US prompt. We know that AVA6000 had previously been manufactured in Switzerland so I'd guess it is now being manufactured in the US, or will be when old stock has been used up or the Swiss contract expires. AVA6103 will surely be manufactured in the US as CC is Americanizing - see, I'm learning - everything.

It's clear that ArchBishopHolmes is a complete idiot ... "during Wednesday Avacta presentation their CEO said it was based on a TWO WEEK OLD discovery lol"... Er, no, the CEO said the patent was FILED two weeks ago, so patent life will be until 2045. And the rest of your post is just pure ignorant drivel.

I pity anyone who relies on that moron for facts about anything, especially Hemogenyx.

So... Bish, bash, bosh, in the green bin you go.

Phase 1a is by its very nature unpredictable, but for AVA6000 it was open-ended!

That FierceBiotech article documents at least a $1.5bn hit to GSK's finances. Such is cost of wrong steps and is the reason why big farmer is so cautious about The New.

Guys, guys, guys! Temu is your friend for when the handbag straps break.

Tis true.

The DSMB decision was obvious (clue: it was based on safety, not efficacy - that's the nature of a Phase 1a trial!) and the positive decision was much talked about here. ...It was baked into the share price, so no surprise then that the news caused a sell-off. I pity those that got spiked though but let that be a life lesson.

Is it us?

DanGB wrote: "From Big Pharma’s perspective: Would we want to licence AVA6000 and advance it knowing that AVA6103 and its potentially enhanced derivatives could blow it out of the water?"

I previously sort of answered this but to be more specific:

If Avacta were to offer exclusive licences for use of AVA6000 in STS and SGC (either to a single licensee or a licence each to two licensees) then it could be written into the licence agreement(s) that no other pre|CISION drug would be licensed for STS/SGC without the AVA6000 licensee(s) being offered first refusal, and this term in the agreement to be effective for x years, say 10 or 15. That would allow other companies to license or partner for AVA6103, AVA6207, etc. but not be able to take them to market approval for STS or SGC.

From a patient point of view I'd like to see multiple deals with multiple farmers for multiple drugs covering multiple indications as that would be the quickest way to make the tech available to humanity. I think in that scenario Avacta would sit at the centre, arranging deals and taking income payments from the deals. All 20 of them, plus a few in the new Finance department.

The options and combinations are myriad - multiple pre|CISION drugs with one or more warheads, multiple indications, and multiple geographic territories.

And the options for deals can go beyond simple partnering and licensing out with exclusive or non-exclusive, time-limited or -unlimited variations.

If I were a big farmer I would be interested in AVA6000 for the indications I'm interested in (SGC, STS and/orTNBC) when the efficacy data is convincing enough to make an offer because a) as first mover I'd likely get decent terms in return for the kudos of my validation, b) partnerhip in particular would give me close working access to Avacta for the technical aspects, and c) such a deal would not preclude deals for other drugs and indications in the future, for which I'd already have a foot in the door.