• Argenica granted US FDA’s Rare Pediatric Disease Designation
  • Orthocell completes Remplir study, targets US$1b+ nerve repair device market
  • LTR Pharma progresses Erectile Dysfunction study


Argenica gets FDA approval

Argenica Therapeutics (ASX:AGN) jumped +15% this morning on the back of an FDA-related announcement.

The brain tissue focused biotech says the US FDA has granted its neuroprotective drug ARG-007 a Rare Pediatric Disease Designation (RPDD) for the treatment of Hypoxic Ischaemic Encephalopathy (HIE) in newborn term infants.

An RPDD is given by the FDA to those drugs that show promise in preventing, diagnosing or treating a rare disease in the pediatric population (children 18 years and younger).

The granting is a key stepping stone for Argenica as it could lead to the FDA awarding it a Priority Review Voucher (PRV), provided that HIE is the first indication for which the drug is approved.

Argenica says a PRV voucher can also be sold or transferred to a third party, with an estimated resale price of a PRV often in the tens of millions of dollars.

HIE occurs when the brain does not receive enough oxygen or blood flow for a period of time. It may occur at any time prior to labour, during labour and delivery, or immediately following delivery. The initial injury that is caused by a loss or reduction of oxygen supply is followed by progressive brain cell death.

Argenica’s CEO, Dr Liz Dallimore, says there are currently no therapeutic drugs available to treat this devastating condition.

“This RPDD will provide the company with potential significant upside at the end of a clinical program in HIE to receive a priority review to get the drug on the market quickly, or the option to sell the voucher to a third party.”


Orthocell to target US$1bn+ nerve repair market

Orthocell (ASX:OCC) has successfully completed the first stage of its pivotal Remplir US market authorisation study.

Remplir is Orthocell’s nerve repair device that reduces the need for damaging sutures and facilitates nerve regeneration.

Remplir has been shown, in the previous pilot study, to be superior to the existing FDA-approved comparator device for nerve repair, restoring the sciatic nerve to its pre-injured state with no adverse reactions.

In the latest completed study, all nerve repair surgeries were performed, also with no adverse events reported.

The study will now progress to the final two stages, in which safety and effectiveness of Remplir will be evaluated, and is expected to complete in Q3 of this year.

According to data, the US addressable market in peripheral nerve repair is estimated to be worth more than US$1.1 billion annually, with an estimated 700,000 procedures where Remplir could be used, completed each year.


LTR Pharma progresses Erectile Dysfunction study

Meanwhile, LTR Pharma (ASX:LTP) announced that all patients recruited for its pivotal bioequivalence clinical study of SPONTAN nasal spray have now received their second and final dose, completing the recruitment and dosing stage of the clinical study.

Data read-out is expected in mid 2024.

Results from this study will be used for pre-submission meetings with the FDA and Australia’s early access scheme as a path to initial sales.

SPONTAN is a world-first, on-demand nasal spray treatment for ED (Erectile Dysfunction).

“We are the first and only nasal spray coming to market for the treatment of Erectile Dysfunction, and are extremely excited to bring this key innovation to men worldwide,” said LTR Pharma chairman, Lee Rodne.

“We believe SPONTAN represents a large paradigm shift in the treatment for Erectile Dysfunction and is a disruptor to the global blockbuster PDE5 (Viagra, etc) market.”


At Stockhead we tell it like it is. While LTR Pharma is a Stockhead advertiser, it did not sponsor this article.