As per the special someone who isn't posting rn."Once CTA is...

As per the special someone who isn't posting rn.

"Once CTA is approved for Australia IXC will start recruiting Australian patients so first patient could be as soon as CTA approved then CTAs for other jurisdictions takes around 60-80 days to get approved so once applications have been submitted for UK US and EU expect around 60-80 days so if apps are submitted within the next month all CTAs approved by June Then its a matter of getting patients through the door but the good thing to note considering its been 4 months for AU is that the review of the application must be soon and if approved first patient can be recruited thats huge"



IXC - Invex Therapeutics LimitedI recently caught up with Tom Duthy from Invex (IXC.asx) to discuss the commencement of a major Phase 3 clinical trial in an orphan disease called Idiopathic Intracranial Hypertension (IIH).IXC is currently trading at 60c to hand them a market cap of $45m and an EV of around $15m, as of their last annual report the top 20 shareholders owned over 58% of the shares on issue, some of whom are clients of mine and will happily read this tonight. IXC continues to make substantial progress in its preparations for a Phase 3 trial in an orphan disease called Idiopathic Intracranial Hypertension (IIH).This disease has no FDA or European approved drugs for treatment, and Invex values the annual opportunity at $1b in the UK and Europe and $600m in the US. Their drug, exenatide, has been re-purposed from its current use in type II diabetes to specifically treat IIH patients, who suffer from debilitating headaches and in some bases permanent vision loss through uncontrolled pressure effects in their brains, IXC calls this drug Presendin.Executive Director and Chief Scientific Officer Alexandra (Alex) Sinclair (whom I met and was very impressive) she has increased her commitment to IXC by entering into an employment agreement with their UK subsidiary, Invex Therapeutics Ltd.She is the world’s leading experts in IIH and has written a number of clinical guidelines on how to treat IIH, which has exploded in cases as we Westerners become fatter. Prof Sinclair is no doubt attending the 48th Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) on 12-17 February 2022 in Austin, Texas – where she and colleagues are reporting on their Phase 2 trial from 2020 and other IIH information. You can register to the annual meeting by clicking on the link below:https://bit.ly/3HPLk04Great way to woo an audience and get your doctors on board for the big trial......or just a write up in Rat's Rant IXC closed the December quarter in a strong financial position with cash and cash equivalents of $31.4 million, with overall cash outflows for the quarter of $0.6m (at that rate it would take well over 12 years to burn through that Tin).IXC is super light on internal infrastructure, preferring to outsource key elements of the trial prep and execution. Importantly, they are fully funded to complete the registration of Presendin which is a major inflexion point.Particularly in light of the market valuing this asset at a mere $15m or so on this asset. Presendin is a long acting (once weekly) formulation of Exenatide, which the Invex has entered into a multi year manufacturing and supply agreement with Peptron, Inc. in Korea.This deal was signed in September 2021, with Invex paying a fixed cost per unit of Presendin for clinical trials and commercial supply.The price has not been disclosed, however, Invex is confident its pricing structures for Presendin in IIH if approved, will see pharmaceutical gross margins preserved – which are typically 80-90%.IXC’s strategy is to secure market approvals for Presendin in IIH in the UK, Europe and Australia. Invex is intending on recruiting patients in the US, however, at this stage they are not betting on an FDA approval without requiring a second trial that meets the FDA’s advice on an improvement in vision as the primary endpoint.The current trial design is based on scientific advice from the Europeans, which the company believes is a lower risk pathway to clinical success relative to a single trial that attempts to meet both FDA and European requirements together.The Trial is expected to commence in earnest this half and patient recruitment over 24 months.The next Clinuvel ? ? ? Let's hope so for those that own itThe strategy employed by IXC is similar to another listed orphan drug developer, Clinuvel Pharmaceuticals (CUV.asx market cap $1.2B). It’s drug, Scenesse, is a long-acting implant to treat a rare skin disease called EPP, Scenesse was approved in Europe in 2014.Scenesse only received FDA approval in late 2019, some five years after European approval and filed for Australian approval in late 2019. In FY19 (before FDA approval) Clinuvel recorded revenues of $31M in Europe (three full years post launch), NPAT of $8.1M and also paid a dividend. In FY21, CUV revenues were $48.5M (EU/USA combined). For the period FY16-FY21, total shareholder return (TSR) was 651% - providing a clear indication on growth in value achieved through success in Europe (noting the potential for replication of this effect to Invex).The market is valuing Invex at a ~99% discount on an enterprise value (EV) basis (~$1.2Bn v $15M) – although Invex has not yet received market approvals, the difference is substantial.



None of this is my work it is the other guy