Health Check: Neurizon free to advance motor neurone disease trial after FDA lifts ‘clinical hold’

• Having satisfied the FDA about safety concerns, Neurizon will motor on to the Healey trial platform
• LTR Pharma invests $1 million in omega-3 side venture
• Thanks to options, 4D Medical cashes up

Neurizon Therapeutics (ASX:NUZ) is free to advance its motor neurone disease (MND) drug candidate, after the US Food and Drug Administration (FDA) lifted a ‘clinical hold’ on the program.

The agency applied the brakes on January 17, pending “further clarification and additional  information”.

The FDA later said it wanted further animal exposure data to assess safety aspects. But it didn’t raise concerns about the company’s previous human trials.

In effect, the FDA has accepted Neurizon’s Investigational New Drug (IND) application, which is permission to mount a US trial.

The IND means Neurizon can progress to phase II/III trial stage with NUZ-001 (monepantel). This is the company’s repurposed animal drug to treat amyotrophic lateral sclerosis (ALS).

Neurizon CEO Dr Michael Thurn says the clearance “marks a significant  milestone in our mission to bring NUZ-001 closer to patients living with  ALS.”

Healey drives next stage

ALS is the most common form of MND and effective treatments have proved elusive.

The trial will be way of Healey, a US platform that tests several potential ALS therapies at the same time.

Neurizon expects the trial to start in the current quarter.

Neurizon isn’t short of supportive animal data and detailed manufacturing and quality information, given it licenses monepantel from veterinary health giant Elanco.

Farmers have long used monepantel as a sheep dip.

In mid-August Neurizon released the results of a ten-patient open-label extension study, showing NUZ-001 to be “safe and well tolerated” after two and a half years’ usage.

The trial also showed “preliminary efficacy”.

This included slowing functional and respiratory decline and a stable or downward trend in key disease biomarkers.

These results “support the potential of NUZ-001 as a disease-modifying therapy for ALS”, the company said at the time.

“Key milestone”

Petra Capital biotech analyst Tanu Jain dubs the FDA decision as a “key milestone and de-risking catalyst” for the company.

The FDA’s IND approval “essentially confirms” that all the pre-clinical, clinical and manufacturing data has passed scrutiny, “underscoring its robustness.”

She now expects investor focus to shift to the “attractive commercial opportunity in ALS and the relatively short timeline to market for NUZ-001.”

Neurizon shares this morning gained up to 14%

It’s oil good as LTR invests in side venture

Spray-based erectile dysfunction (ED) drug developer LTR Pharma (ASX:LTP) has invested $1 million to boost its stake in an omega-3 joint venture from 33% to 43%.

In mid-September LTR announced the JV, called LevOmega Pty Ltd, which aims to develop ‘nature identical’ omega-3 oils.

These oils have widely known health benefits, including fostering heart health and developing infants’ brains.

Omega-3 oils derive from fish, but overfishing and ocean pollution is constraining supply.

This underscores the need for “scaleable, sustainable alternatives”.

Lev Omega plans to supply its ingredients to the pharmaceutical, nutraceutical and food manufacturing sectors.

LTR didn’t pay anything upfront for the JV. The other JV parties are Green Blue Health Pty Ltd and product innovator Levur Pty Ltd.

LTR’s main game continues to be developing its ED treatments Spontan and Roxus.

Opthea boosts its kitty for Plan B

Opthea (ASX:OPT) has pocketed a $10.8 million research and development tax incentive.

This boosts the company’s war chest for an alternative activity, following the failure of its two-phase III eye disease programs.

After reaching a US$20 million settlement with investors owed up to US$680 million, the company in mid-August said it had US$20 million of residual cash.

The federal government’s R&D incentive program enables a company to receive a 43.5% rebate for eligible expenditure.

“We are taking clear and deliberate steps to advance the opportunities within our assets, with a focus on creating value for our shareholders,” executive chair Jeremy Levin says.

“We will share an update on our strategic plan later this year.”

Opthea shares have remained suspended since early March, when the company announced the horror trial results.

Sorry honey, I forgot about the paperwork

We pity the tiny minority of 4D Medical (ASX:4DX) shareholders who failed to exercise their deeply in-the-money options by the October 1 deadline.

The lung-imaging company on Friday said it had raised $22.6 million from the oppies. These were exercisable at 55 cents, with 99.7% converted into shares.

The company issued the options on a one-for-one basis in a February capital raising, at 36 cents a share (for retail holders).

Converting holders also get one-for-one ‘piggyback’  options, convertible at 75 cents by September 2027.

4D shares tore from 24 cents in late July, to this morning’s peak of $2.49.

In late August the FDA approved the company’s CT:VQ, to measure lung ventilation and perfusion from standard chest X-rays.

4D also has a swag of separate options with a $1.365 strike price, exercisable by December this year.

If fully exercised, these would raise a further $30.2 million.

‘Payday’ for Mesoblast

The Center for Medicare & Medicaid Services has granted a so-called J-Code for Mesoblast’s (ASX:MSB) Ryoncil, the company’s stem cell treatment for paediatric graft-versus-host disease.

The code provides a standardised, permanent billing pathway and thus makes Ryoncil easier to bill (and pay for).

Last December the FDA approved Ryoncil, the agency’s first consent for a mesenchymal stromal cell product.

Mesoblast develops off-the-shelf cellular medicines for severe and life-threatening inflammatory conditions.

The company is developing a variant,  Rexlemestrocel-L, for heart failure and chronic low back pain.

ASX biotechs have enjoyed a US reimbursement purple patch.

Last week, the CMS last week granted Avita Medical (ASX:AVH) a so-called New Technology Add-On Payment.

The US$4875 payment applies to procedures using Avita’s Recell, for non-burn full-thickness acute wounds resulting from trauma or surgery.

ImpediMed (ASX:IPD) has won coverage from the fifth-biggest US health insurer, which decreed the company’s Sozo lymphoedema detection device as “medically necessary”.

Shares in Echo IQ (ASX:EIQ) today entered trading halt. This is pending an announcement about US coverage for the company’s Echo Solv aortic stenosis detection tool.

At Stockhead, we tell it as it is. While Neurizon and LTR Pharma are Stockhead advertisers, the companies did not sponsor this article.