For biotech investors, the news of an approval by the US FDA is a genuinely exciting milestone.
The FDA is the ultimate gatekeeper for the US pharmaceutical market, which is by far the world’s largest.
Approval from the FDA not only validates the safety and efficacy of a new drug, but also opens the door to substantial commercial opportunities, as it allows the company to market and sell the drug in the highly lucrative American market.
This milestone often leads to increased investor confidence, potential revenue growth, and a boost in the company’s stock price.
In 2024, several ASX biotech companies have earned FDA approval, while others are eagerly awaiting imminent decisions.
Here, we highlight these companies and their progress.
ASX biotechs with FDA approvals in 2024
Botanix is the latest ASX-listed biotech to receive an FDA clearance.
Last week, the company received an FDA approval for Sofdra (sofpironium) gel, 12.45%, marking a significant milestone as the first new drug for treating primary axillary hyperhidrosis (excessive underarm sweating) in both adults and children aged 9 and older.
This approval is crucial because there are approximately 10 million people in the US affected by this condition, with limited effective treatment options available.
Botanix plans to launch a patient experience program in Q3 CY2024, with the first revenue expected in early Q4 CY2024 from Sofdra.
Hyperhidrosis is a condition where people sweat excessively, more than what’s needed to cool the body, and is the third most common dermatological condition in the US.
The FDA approved Sofdra based on results from two important Phase 3 studies called ‘CARDIGAN’. These studies involved 701 patients with primary axillary hyperhidrosis..
Botanix plans to start a program in Q3 this so that eligible patients can try Sofdra early. The wider release of Sofdra is expected in early Q4 CY2024, with sales beginning shortly after.
“As the first and only new chemical entity, Sofdra represents a new therapeutic approach for dermatologists to treat patients with this disabling medical condition,” noted Botanix Executive Chairman, Vince Ippolito in a recent statement.
Earlier this month, GSS, a company known for its molecular diagnostic solutions for infectious diseases, received clearance from the FDA for its EasyScreen Gastrointestinal Parasite Detection Kit and GS1 automated workflow.
This is a significant announcement in the world of clinical diagnostics, especially for expanded testing for gastrointestinal (GI) parasites.
The approval means that healthcare professionals now have a powerful new tool to accurately and quickly diagnose these kinds of infections.
Every year, more than 3.5 billion people worldwide get infected with GI parasites, causing more than 200,000 deaths and a lot of health and economic problems. In the US alone, there are about 65 million cases of GI infections each year, but only 15% of them are actually reported to doctors.
These parasites can come from contaminated water, food, or surfaces, and even from international travel. Sometimes people don’t even have symptoms, but they can still spread the infection.
Currently, diagnosing these infections in the US relies a lot on using microscopes, which takes a long time, is complicated, and needs highly trained staff.
Neil Gunn, interim CEO of Genetic Signatures, said:
“Our unique 3base technology offers significant differentiation and advantages for molecular testing of the most challenging samples and gastrointestinal parasites that impact over 60 million patients a year in the US.”
On the back of that announcement, GSS has appointed Allison Rossiter as its new permanent CEO. Rossiter has plenty of experience in the diagnostics industry, particularly with Roche Diagnostics.
GSS also announced a $30 million capital raise.
Wound care medical company, AVITA, received an FDA approval for its latest product, the RECELL GO System, in late May.
The RECELL system essentially harvests a small sample of a patient’s skin cells, typically taken from a healthy area of skin. These cells are then processed to create a suspension of Spray-On Skin Cells.
By using less donor skin, patients experience reduced pain and faster closure, with enhanced aesthetic results at the donor site, with fewer procedures needed for complete wound closure.
CEO Jim Corbett believes this FDA approval will revolutionise wound care, making treatment more accessible and effective.
“FDA approval of RECELL GO marks a paradigm shift in the treatment of partial-thickness and full-thickness wounds,” said Corbett in an announcement on May 30.
“By streamlining processes and enhancing operational efficiency with the use of RECELL GO, clinicians can now treat a greater number of patients and more broadly experience the proven benefits of RECELL technology.”
The RECELL GO system will launch in top burn treatment centres in the US initially, with plans for broader availability in the future.
In April, Adherium announced that its Hailie Smartinhaler is FDA-cleared for use with AstraZeneca’s Airsupra and Breztri inhalation devices.
Airsupra is the first FDA approved rescue medication for asthma patients, while Breztri is a triple combination for COPD patients. Both were developed by AstraZeneca.
This new compatibility allows patients and doctors to monitor medication usage and improve treatment through real-time data from the Smartinhaler. The personalised approach is expected to make treatments more effective and improve patient outcomes.
Dr Paul Mastoridis, the newly hired CEO of Adherium, told Stockhead the Hailie device attaches on to the inhaler and is linked to an app (paired via Bluetooth), giving valuable information such as how regular and how well patients are inhaling the medicine to make sure they’re taking it as prescribed.
He said Adherium is the only company out there with the technology that provides this kind of capability.
“We’ve been partners with AstraZeneca for a while, as we were involved in some of their clinical trials. But this was the first time AstraZeneca has a rescue medication that’s been approved by the FDA, and it’s the only one of its kind,” said Mastoridis.
“We now have over 90% of the inhalers out there that fit our Hailie device.”
“In total, we have 12 FDA approved devices. So we can now say to the physicians, whatever device or whatever drug you want to give to patients, we have a Hailie device.”
Read more about Adherium: Adherium’s focus on US market could pay off big time for its novel asthma device
In mid June, the US FDA extended a Rare Paediatric Disease Designation (RPDD) to Race Oncology’s novel RC220 bisantrene drug.
The RPDD extension was given to treat AML (acute myeloid leukemia) in children, which comes as interest grows in the drug from paediatric oncologists globally.
RPDD is granted for new treatments of serious or life-threatening diseases which affect fewer than 200,000 people in the US and which primarily affect individuals less than 18 years of age.
Race says ~70% of rare diseases are exclusively paediatric in onset, with 95% of rare diseases having no approved treatments. The company was previously granted RPDD by the US FDA for the RC110 formulation of bisantrene in 2018.
Read more here: Race Oncology stock gains on US FDA milestone
A week earlier, the US FDA had extended its orphan drug designation to RC220 for the same indication.
Being granted ODD offers various benefits to sponsors of new treatments for orphan diseases. These include seven-year US marketing exclusivity, a 25% federal tax credit for clinical research expenses incurred in the US, and a waiver of fees under the Prescription Drug User Fee Act (PDUFA). RAC says the waiver of fees amount to more than US$4m in FY24.
Additionally, an ODD makes recipients eligible to receive research grants, and allows access to further regulatory assistance and guidance from the FDA.
Read more about that here: US FDA extends orphan drug designation for Race Oncology’s lead asset
PYC also received an Orphan Drug Designation (ODD) from the US FDA in late May for its drug candidate, PYC-001, aimed at treating OPA1-associated vision loss.
PYC-001 is said to be a significant advancement in precision therapy. The drug was designed to restore the expression of the OPA1 gene to levels necessary for optimal retinal function.
Utilising the company’s proprietary drug-delivery technology, PYC has tackled a major hurdle for RNA drugs by ensuring effective delivery to the targeted cells affected by ADOA (Autosomal Dominant Optic Atrophy).
ADOA is a progressive and irreversible blinding eye disease. It affects approximately 1 in every 35,000 people representing a market size of ~$2 billion per annum.
ADOA is caused by a mutation in one copy of the OPA1 gene, which results in inadequate levels of OPA1 gene expression, impacting the normal function of retinal ganglion cells.
Argenica Therapeutics (ASX:AGN)
In March, Argenica’s shares jumped on the back of an FDA-related announcement.
The brain-tissue-focused biotech said the US FDA had granted its neuroprotective drug ARG-007 – a Rare Pediatric Disease Designation (RPDD) for the treatment of Hypoxic Ischaemic Encephalopathy (HIE) in newborn term infants.
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, said 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.”
In January, the FDA cleared Amplia’s Investigational New Drug (IND) Application for a clinical trial of narmafotinib in the USA.
Amplia is now set to conduct a clinical trial for the drug in pancreatic cancer patients in the US. This trial aims to test narmafotinib alongside chemotherapy drug, FOLFIRINOX, which is commonly used in the US for treating advanced pancreatic cancer.
The approval is a big deal for Amplia because it allows it to expand research into a potentially large market for pancreatic cancer.
The company has already been conducting trials with narmafotinib in Australia and South Korea, but this new trial in the US will focus specifically on how narmafotinib works with FOLFIRINOX.
Amplia is now in the process of planning for this important trial.
FOLFIRINOX is the preferred treatment for pancreatic patients in the USA and most of Europe, and therefore this combination trial is highly relevant.
Genomic life science company EZZ has achieved a key milestone with the US FDA approving nine of its products in the food category.
EZZ says the US market represents a significant opportunity for the company with online sales of health supplements totalling US$23.8 billion in 2023.
The company says the nine FDA approved products were selected by the company due to their success in other markets and will form the basis of the initial market launch.
The products include: EZZ Children’s Essential Minerals, EZZ Children’s Eye Health, EZZ Bone Growth Chews and EZZ Brain Focus just to name four.
EZZ chairman Glenn Cross mentioned the company is “thrilled to have received FDA approval” for a range of its products.
“This is a monumental achievement for EZZ and a validation of our commitment to excellence in product quality and innovation,” he said.
“Entering the US market is a pivotal part of our plans for future growth, and we are excited about the opportunities this approval presents.
“We look forward to bringing our high-quality products to consumers in the United States and continuing to drive value for our shareholders.”
Read more: EZZ US bound after FDA approval for nine products
ASX Stocks with potentially imminent FDA approvals
In early June, Artrya submitted a Q-Submission (Q-Sub) to the FDA for its flagship product, Salix Coronary Anatomy (SCA), as a key step in finalising its 510(k) application process.
The company expects to meet with the FDA in around July or August to discuss this submission. After the meeting, it plans to submit the 510(k) application.
A Q-Sub is a formal request for feedback from the FDA to guide product development and application preparation.
Artrya successfully held its first Q-Sub meeting with the FDA in June 2023, where the company established a pathway to regulatory clearance for Salix.
Artrya has requested this second meeting to validate its approach since the first meeting, which is a crucial final step in completing their FDA application.
Salix is a breakthrough technology that can detect vulnerable plaque biomarkers from a CCTA (Coronary CT Angiography) within minutes.
The platform is used in emergency situations, or where there is a CCTA machine – for example, imaging centres.
Patients who use the product range from those who turn up at one of these centres with chest pain, to those who were referred by doctors.
“Because the SCA gives such a granular, clear 3D picture of where the issues are outside of just the typical narrowing of the artery, it gives the opportunity for the clinician to say ‘you’re fine’ or ‘hang on, we need to sit down and do something different here’,” explained Artrya CEO, Mathew Regan,
“It’s a technology they haven’t had before.”
Read more about Artyrya here: FDA submission looms as Artrya’s AI platform aims to capture the huge cardio diseases market
Imricor Medical Systems (ASX:IMR)
Imricor has just started the VISABL-AFL clinical trial at the Cardiovascular Institute of South Paris for a potential US FDA approval.
This trial aims to evaluate the safety and effectiveness of atrial flutter ablation using the company’s Vision-MR Ablation Catheter System.
It is expected to enrol 91 patients across the US and Europe, with plans for interim analysis after 76 patients. Imricor aims to complete enrollment by the end of the year, paving the way for FDA approval in 2025.
“This is a huge milestone for all of us at Imricor,” noted Imricor’s Chair and CEO, Steve Wedan late last week.
“We are on track with VISABL-AFL to complete enrolment this year, supporting our goal of FDA approval for our platform of technology in the US in 2025.”
The Vision-MR Ablation Catheter is Imricor’s main product, designed to be guided by real-time MRI for more effective and safer treatment of type 1 atrial flutter compared to traditional x-ray guided catheters.
It’s already approved in the European Union and Saudi Arabia.
In early June, positive results were achieved from LTR Pharma’s recent primary and secondary clinical studies for its SPONTAN erectile dysfunction (ED) spray.
SPONTAN is the company’s novel treatment for ED, and is designed to be a world-first, fast acting, on-demand nasal spray solution.
The company noted that initial data showed that the nasal spray reached the same Cmax (maximum concentration) level as an oral administration, despite being administered at a lower dose, with volunteers showing observable effects of the drug in as little as nine minutes, with an average of 12 minutes across the study compared to 56 minutes (Tmax) in patients receiving oral treatment.
The studies were conducted in Sydney, in partnership with Southern Star Research and Scientia Clinical Research and assessed the relative bioavailability of Vardenafil – a widely used PDE5 inhibitor to treat ED – following administration of SPONTAN nasal spray, when compared to oral delivery of Vardenafil tablets alone.
The studies’ findings backed up initial results that have shown SPONTAN achieves rapid absorption and faster onset of action compared to oral PDE5 inhibitors (i.e., vardenafil, sildenafil, tadalafil).
These oral PDE5 tablets are currently the gold-standard treatments, but have a high discontinuation rate with consumers.
SPONTAN is designed to overcome the issues with oral PDE5 tablets and to be a fast acting-on demand treatment for ED, potentially transforming men’s lives.
In April, Paradigm submitted key documents to the FDA for review, aiming to advance its pivotal Phase 3 clinical trial of lead drug, injectable pentosan polysulfate sodium (iPPS/Zilosul) to treat osteoarthritis (OA).
Often referred to as degenerative joint disease or ‘wear and tear’ arthritis, OA stands out as the most common form of this condition.
PAR aims to minimise the disease’s impact by improving the functional mobility, preserving the joint structure, and reducing pain of affected individuals.
The company is developing injectable pentosan polysulfate sodium (iPPS/Zilosul) and Mucopolysaccharidosis, where inflammation significantly contributes to the disease process.
A Phase 2 trial demonstrated one course of iPPS can provide 12-month pain relief and improved function in knee OA sufferers. The same clinical trial also demonstrated improvement in cartilage volume and thickness at six months following treatment compared to placebo.
Read more about it here: Paradigm makes key submission to US FDA to advance osteoarthritis Phase 3 trial
At Stockhead we tell it like it is. While Paradigm Biopharma, LTR Pharma, EZZ Life Science and Race Oncology are Stockhead advertisers, they did not sponsor this article.
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