ASX Health Stocks: Kazia Therapeutics gets crucial designation status from the US FDA
Health & Biotech
Health & Biotech
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Oncology company Kazia Therapeutics (ASX:KZA) says its lead drug paxalisib has been awarded an Orphan Drug Designation (ODD) by the US FDA.
An ODD is a special status accorded to drugs which are considered promising as potential treatments for rare (or orphan) diseases, which in the US is defined as those which affect less than 200,000 cases per year.
The ODD status provides drug developers with up to seven years of Orphan Drug Exclusivity (ODE), during which competitors may not use Kazia’s data to develop generic versions of paxalisib.
This protection effectively extends the life of a commercial product, and also provides opportunities for grant funding.
Kazia’s paxalisib is a brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway, which is being developed to treat glioblastoma – the most common and most aggressive form of primary brain cancer in adults.
A Phase 2 clinical trial of multiple drug therapies, including paxalisib, is ongoing with initial results anticipated in 2023.
Following the ODD designation, the FDA will waive fees relating to a future regulatory filing in AT/RT (atypical rhabdoid/teratoid tumors), potentially saving more than US$3 million if Kazia was to seek approval for this disease.
“This represents an important new opportunity for paxalisib, and one that we continue to explore enthusiastically with our collaborators and advisors,” said Kazia CEO, Dr James Garner.
Lumos Diagnostics (ASX:LDX) has secured a contract with US-based Aptatek to develop a product to assist with screening for phenylketonuria (PKU).
PKU is a rare inherited disorder which causes an amino acid called phenylalanine to build up in the body and affects approximately 1 in 12,000 newborns.
Left untreated, this build-up can result in intellectual disabilities, seizures, behavioural problems and mental disorders.
The Aptatek product, developed under contract with Lumos, aims to provide a point-of-care/at-home screening test to allow PKU patients to directly measure their phenylalanine levels in real-time.
The initial phase of the partnership is expected to generate at least US$500,000 in revenue for Lumos.
“Aptatek’s objective of replacing a centralised PKU diagnostic test with a POC test that can be used in the home highlights the broad shift towards decentralised testing that has the potential to deliver better outcomes for patients,” said Sam Lanyon, Lumos’ interim CEO.
Trajan Group (ASX:TRJ) has signed a binding agreement to acquire 100% of Kentucky-based Chromatography Research Supplies (CRS) for US$43.3 million (around $61.9 million).
Having operated for over 25 years, CRS is a leading global manufacturer of high-quality analytical consumables.
Its products are used in analytical laboratories and various other industries worldwide, and are known for their quality according to Trajan.
Trajan believes the acquisition is highly complementary to its existing product portfolio, and has forecast that it will contribute US$14.1 million for FY22, and earnings per share accretion of more than 31%.
The acquisition is to be funded via a fully underwritten $29.7 million institutional placement, a $20 million in acquisition debt financing through a facility with HSBC, and $13.4 million from existing cash.
The TRJ stock price is currently under suspension pending the cap raise.