Welcome back to part 4 of our Clinical Trials 101 series.  In the first of our series we looked at what exactly are clinical trials, the three different trial stages, protocol and key organisations.

In Part 2 we looked at the review and drug approval proof process and how to spot a company which has a good chance of actually getting its drug to market and passing the hallowed Phase III or pivotal trial status.

In Part 3 we continued on the theme of getting a drug to market. Now in part 4,  the final of our series we look at some of the companies on the ASX who have jumped the hurdles to make it to the home stretch of Phase III pivotal trials, a crucial step in getting a drug to market.

Neuren share price up ~285% in past year as it awaits FDA drug approval

Even one step ahead of the pivotal Phase III trial is Neuren Pharmaceuticals (ASX:NEU), which recently announced the US Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) of  trofinetide for the treatment of Rett syndrome, that was submitted by its US partner Acadia Pharmaceuticals (Nasdaq:ACAD).

It will be the first ever drug for Rett syndrome, a rare genetic neurological and developmental disorder that affects the way the brain develops.

Neuren said it expects to earn revenue over 2022 and 2023 for Rett syndrome in the US alone of A$118 million plus double-digit percentage royalties on net sales.

Furthermore, Neuren has four Phase 2 trials of lead drug NNZ-2591 to treat serious neurological disorders that emerge in early childhood.

Neuren has attracted the interest of investors and been rewarded for its trial success with its share price rising ~285% in the past year.

The company’s higher market cap saw it recently being admitted to the elite S&P/ASX 300 index following the September re-balancing.


Companies in Phase III (pivotal) or about to start

Opthea (ASX:OPT) 

Eye disease specialist Opthea is conducting not one but two concurrent Phase III clinical trials with OPT-302 for the treatment of wet AMD, a chronic eye disorder that causes blurred vision or a blind spot in your visual field.

According to Opthea’s site, the Phase III trials, referred to as ShORe and COAST, are double-masked, sham-controlled trials that enrol treatment-naïve patients and assess the efficacy and safety of 2.0mg OPT-302 in combination with anti-VEGF-A therapy, compared to standard of care anti-VEGF-A monotherapy.

Dimerix (ASX:DMX) 

Dimerix is undertaking a Phase III trial for its lead product DMX-200 in the study of rare kidney disease Focal Segmental  Glomerulosclerosis  (FSGS).

The ACTION 3 Phase III study is being performed across 75 sites with part one data outcome expected to be known by mid-2023.

DMX orphan drug designation in US, Europe and the UK and that comes with advantages including an accelerated pathway for approval and extended exclusivity period affecting all patents.

The company said it’s within 12 months of a major outcome for the Phase III clinical study.  A second analysis is expected about 12 months later where, if the data looks good with 144 total patients, they can go to the FDA and potentially to market.

Telix Pharmaceuticals (ASX:TLX)

Oncology company Telix is a complex business with multiple trials and diagnostics. The company this week announced that the Chinese National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) had approved an investigational new drug (IND) application to commence a pivotal Phase III registration study of TLX591-CDx.

Known as Illuccix TLX591-CDx is a radiopharmaceutical cold kit for the preparation of 68Ga-PSMA-11 injection, used for prostate cancer imaging using Positron Emission Tomography (PET).

Telix said the bridging study was required to provide data obtained in a Chinese population to establish that the diagnostic efficacy of TLX591-CDx is equivalent in Chinese and Western populations.

The company this week also released preliminary data from two separate studies of TLX250-CDx (89Zr-DFO-girentuximab) in triple negative breast cancer (TNBC) and non-muscle-invasive bladder cancer (NMIBC).

You can find more information on the Telix trial pipeline spanning across various stages for prostate, kidney, lung, ovarian and glioblastoma cancers along with bone marrow conditioning on their website.

The TLX share price is up ~19% this week on the positive trial announcements.


Invex Therapeutics (ASX:IXC) 

IXC announced in August it had received Investigational New Drug Application (IND) approval for lead candidate Presendin from the FDA to start the IIH EVOLVE Phase III clinical trial in the US.

Presendin is a potential drug used to treat Idiopathic Intracranial Hypertension (IIH).  IXC plans to open up 40 sites globally for the randomised, placebo-controlled, double-blind trial that will randomise 240 patients with newly diagnosed IIH.

The trial aims to determine the efficacy and safety of Presendin versus placebo, administered once weekly over 24 weeks. It was designed to specifically meet the requirements for market approval of Presendin for the treatment of IIH in the EU, UK and Australia.

Paradigm Biopharmaceuticals (ASX:PAR)

PAR specialises in drug repurposing (new uses for approved drugs) to cure illnesses related to degenerative osteoarthritis, bone marrow edema lesions, respiratory, alphavirus, and chronic obstructive pulmonary disease.

PAR’s core offering is based on the efficacy of an anti-inflammatory drug called pentosan polysulfate sodium  (PPS). Used in humans for more than 60 years, PPS is a semi-synthetic drug made from beechwood hemicellulose that is used to treat conditions such as blood clots and bladder pain.

As Stockhead’s Tim Boreham noted PAR is now lifting the hopes of superannuated footballers with PPS to treat knees and joints.  While PPS is yet to be approved to treat knee and joint osteoarthritis, it hasn’t stopped dozens of former AFL players using the drug under a special access scheme.

The company in September completed a circa $50 million capital raising (remember it costs a lot of money to run a trial) and dosed its first patients in April in the global Phase III osteoarthritis trial that will determine whether the drug can be approved.

The Phase III trial will measure the change in pain and function after injections of PPS, compared with injections of placebo in participants with knee osteoarthritis pain.

Medlab Clinical (ASX:MDC) 

Phase III trials for Medlab’s lead asset cannabis based-pain medication NanaBis for treating cancer-related bone pain are due to start shortly. NanaBis is currently available for Australian patients under the Special Access Scheme (SAS) and launching in the UK under the Named Patient Program (NPP).

Early work was done on botanicals characterised (pure and standardised) to 98%, however in its pre-IND meeting with the FDA it was strongly suggested to look for 100% (also known as neat) synthetics as a more preferred option.

Medlab now has an FDA recognised drug master for the two synthetic cannabinoids, and is completing its chemical manufacturing and control (CMC) package for IND approvals relating to the Phase III trial.

Currently the Phase III has Australian Ethics approval and TGA clinical trial number, whilst in the UK the trial has a favourable ethics outcome. The Phase III will target cancer bone pain as a monotherapy, subject to any final remarks from the FDA.

The trial, posted on US NIH site clinicaltrials.gov is a quad randomised non-inferiority trial with an intervention period of six weeks.

Medlab has several site agreements already in place for the study whilst currently onboarding others and conducting investigator product training.

At Stockhead we tell it like it is. While Medlab Clinical and Dimerix are Stockhead advertisers, they did not sponsor this article.