Breakthroughs in biotech bring us closer to better health

Biotechnology involves the exploration and modification of living organisms to develop innovative products and methodologies enhancing human health and quality of life.

This discipline promises transformative advancements across healthcare, agriculture environmental management and more.

As we move further into 2024, it’s worth taking a look at innovation in biotechnology in 2023 and some of the significant breakthroughs made in the sector.

Breakthroughs in Alzheimer’s research

FDA Approves LEQEMBI

2023 was marked by significant advancements in Alzheimer's disease treatment, notably with the FDA's approval of LEQEMBI (lecanemab-irmb). Developed by Eisai Co., Ltd. and Biogen Inc (NASDAQ:BIIB, ETR:IDP)., LEQEMBI has shown considerable efficacy in slowing disease progression and cognitive decline in patients with early-stage AD. This marks the first instance of a treatment that positively alters the course of the disease.

Vaccine developments

The progress in Alzheimer's vaccines has also been a highlight, with Vaxxinity's UB-311 emerging as a promising candidate. Targeting toxic amyloid beta forms in the brain, UB-311 has shown safety and efficacy in Phase 2 trials, pointing towards a novel treatment pathway. This synthetic, peptide-based vaccine forms part of Vaxxinity's innovative platform, aiming for cost-effective and enduring solutions.

Innovations in obesity treatment

Expanding drug market

The battle against obesity has seen notable innovations in 2023, with the emergence of GLP-1 analogues and gene therapy. These treatments offer a deeper understanding of obesity's metabolic and hormonal aspects, promising more effective interventions.

Bioprinting and gene therapy

Advances in bioprinted tissues and gene therapy highlight the biotech sector's efforts in creating accurate models for drug testing and targeting obesity's genetic factors, showcasing the potential for lasting solutions.

CRISPR-based therapy

Casgevy's milestone

2023 witnessed the groundbreaking approval of Casgevy, the first CRISPR-based therapy, by the UK's MHRA and the FDA. This treatment offers a new horizon for inherited blood disorders, though its high-cost poses challenges for global accessibility.

RNA technologies

Vaccine and rare disease advances

The success of mRNA vaccines has expanded into treatments for various diseases, while RNA technology's role in addressing rare diseases opens new therapeutic avenues, promising for future medical innovations.

The role of AI in biotech

Protein engineering and drug repurposing

AI has significantly influenced protein engineering and drug repurposing, enabling precision in developing new therapeutics and identifying new applications for existing drugs, revolutionising the approach to rare disease treatment and beyond.

These developments across Alzheimer's research, obesity treatments, genetic therapies, RNA technologies, and AI integration mark 2023 as a year of hope, promise, and groundbreaking advancements in biotechnology.

Aussie small caps doing big biotech things

Anteris Technologies

Anteris Technologies Ltd (ASX:AVR, OTC:AMEUF) continues investing in R&D to commercialise its DurAVRTM THV technology. Net cash inflow for the quarter including FX movements was $23.6 million. The company raised $41.3 million during the quarter with a  $33.3 million placement of 1.664 million new shares at $20 each in November and $8 million in proceeds from the exercise of 550,000 options. The Company had a cash balance of $30.8 million at December 31, 2023.

There were several highlights for the Anteris during the quarter. These included:

• Enrolment for the 15-patient US FDA approved Early Feasibility Study (EFS) of DurAVR™ THV to treat severe aortic stenosis completed October 17.
• Preliminary results 30 days post-procedure from 14 out of the 15 enrolled patients (one awaiting scheduling) showing outstanding haemodynamic function, excellent safety data and no incidence of stroke, myocardial infarction, life-threatening bleeds or all-cause mortality.
• Eight patients with severe aortic stenosis were successfully treated with DurAVR™ THV at the Tbilisi Heart and Vascular Clinic, Tbilisi, Georgia (Tbilisi).

Of its results, Anteris chief medical office Dr Chris Meduri said, "These haemodynamics are unparalleled.

“This biomimetic valve not only allows us to have this acute improvement in flow from a classic haemodynamic performance but, also, we’ve seen (on cardiac MRI) normalisation of laminar flow out of the aorta. It’s likely to have significant implications as we think about the long-term durability of the valve, stress on the leaflets but, also, aortopathies, potentially inflammation and other things as well.”

Emyria

Emyria Ltd (ASX:EMD) is delivering and developing new treatments for major mental health challenges and select neurological conditions. 

Emyria’s Managing Director, Dr. Michael Winlo, reflected: “Emyria made significant progress this quarter. We successfully initiated a pioneering MDMA-assisted therapy trial for PTSD and made substantial progress in securing Authorised Prescriber approval for our lead psychiatrist. The strategic addition of Greg Hutchinson to our Board directly enhanced our service delivery and research capabilities which directly aligns with our mission to establish scalable, clinically effective, and commercially viable care models while advancing novel mental health and neurological treatment development programs in collaboration with globally recognised research partners.”

In Q2 FY24, net cash used in operating activities rose to $1.9 million, primarily due to one-off personnel termination payments and increased research and patent costs for our MDMA analogue program (up from $1.1 million in Q1 FY24).

It has several key achievements.

Emyria which has distinguished itself as the sole ASX-listed entity focusing on psychedelic-assisted therapy evaluations, has initiated the dosing stage in its trailblazing MDMA-assisted therapy trial. This marks a global first for private community-based clinics, where Emyria is exploring an innovative MDMA-assisted therapy approach for Post-Traumatic Stress Disorder (PTSD), a condition impacting over one million Australians. To date, two participants have successfully completed the dosing phases, with a third currently undergoing enrolment.

The company's leading psychiatrist has garnered ethical approval for their Authorised Prescriber application, a milestone achieved in January 2024, enabling Emyria to administer the therapy to meticulously screened patients within a rigorous regulatory framework established by the Therapeutic Goods Administration (TGA).

Furthermore, Emyria has secured approval from Health Canada to import its inaugural batch of the drug, ensuring a supply for up to 70 trial participants or patients. This batch has been safely received, marking another significant step forward in the trial's progress.

During the quarter, Emyria also broadened its developmental partnership with the University of Western Australia, focusing on the creation of new and exclusive MDMA analogues. These compounds hold the promise for the future treatment of various mental health and neurological disorders. Concurrently, Emyria's proprietary cannabinoid-based medications have made significant progress through an entirely funded preclinical evaluation program, overseen by the National Institutes of Health in the United States.

Genetic Technologies

Genetic Technologies Limited (ASX:GTG, NASDAQ:GENE, OTC:GNTLF) is a global leader in guideline-driven genomics-based testing in health, wellness and serious diseases. It had cash receipts totalling A$3.6 million for the quarter ending December 2023.

“In the past quarter, we achieved further significant milestones in the business starting with a record number of geneType test processed,” CEO Simon Morriss said.

“Additionally, the team have successully recruited many new healthcare professionals, setting up a strong base for increased adoption of our tests in our key markets, Australia and the U.S. Other achievements include the launch of the geneType HBOC test in the U.S., continued partnering discussions for the the rapidly expanding healthcare sector in Indonesia and we are excited about the upcoming launch of our branded tests in the UK through the National Pharmacies Association (NPA).

“Locally our Precision Medicine Clinic pilot at Gold Coast Private Hospital (GCPH), a member of Healthscope, is getting under way utilising GTG’s geneType Multi Risk test combined with Pharmacogenomics (PGx) tests with the ultimate objective of improving outcomes for GCPH’s patients. Additionally, being named the sole industry partner for a Medical Research Future Fund (MRFF) Genomics Health Futures Mission Grant further underscores our commitment to groundbreaking research.”

Highlights included:

1. Appointment of two experienced commercial advisors to accelerate our Direct to Consumer (‘DTC’) plans for Genetype in the U.S., capitalising on the burgeoning consumer personalised health and wellness sector.
2. Record number of geneType tests processed, with six times more commercial samples received compared to the prior corresponding period.
3. Launched Hereditary Breast & Ovarian Cancer Risk Assessment Test (HBOC) via an exclusive Santa Monica event for a select group of clinicians.
4. Commenced Precision Medicine Pilot with the Gold Coast Private Hospital, a member of Healthscope.
5. Expanding Global Footprint South East Asia. Discussions continue with potential partners to access Indonesia's healthcare market, with the geneType Multi-Risk test
6. Expanding Global Footprint UK. In April 2024 the Company’s branded tests are to be offered to subscribers of the UK National Pharmacies Association (NPA) via a targetted campaign.
7. Commenced Activities associated with CASSOWARY Trial. MRFF Grant named GTG as sole industry partner for the $2.4 million clinical trial. Results will inform how cancer risk is assessed and has the potential to reshape care in General Practice.

Island Pharmaceuticals

Australian antiviral drug development company, Island Pharmaceuticals Ltd (ASX:ILA) had a strong quarter. 

CEO Dr David Foster said, “We made significant progress this quarter, with the first two cohorts of our ISLA-101 Single Ascending Dose study completed, and the first dose provided to our third cohort. Data read-outs are on track for early 2024, which will help optimise our planned Phase 2a PEACH clinical trial.

“Our loan agreement with Radium Capital provided an important form of non-dilutive funding to support the progress of ISLA-101 development, while our ISLA-101 intellectual property portfolio was further boosted with the granting of a key Australian patent.”

Highlights included: 

• Following human research ethics approval, Island completed dosing cohorts 1 and 2 in its ISLA-101 Single Ascending Dose study.
• Post quarter, third cohort dosed under fasted conditions following confirmation from the Data Safety Review Committee that ISLA-101 was deemed safe and tolerable for the 16 subjects dosed across cohorts 1 and 2.
• ISLA-101 granted key patent from IP Australia covering method of treating or preventing flavivirus infections by administering ISLA-10.
• Island received tax refund of $386,345 for FY2023 and secured facility with Radium Capital for prepayment of forecast R&D Tax Incentive (RDTI).
• Island closed the quarter with a cash position of $1 million.

Immuron 

Immuron Ltd (NASDAQ:IMRN, ASX:IMC) was highly active during the December quarter.

The globally integrated biopharmaceutical company  initiated the second cohort of a clinical trial aimed at assessing the effectiveness of Travelan® in preventing infectious diarrhoea caused by enterotoxigenic Escherichia coli (ETEC).

The Phase 2 clinical trial, which follows the completion of a first cohort in August 2023, now includes 34 participants who have been randomised to receive either Travelan® or a placebo.

Further to this the US Naval Medical Research Command (NMRC) initiated the clinical evaluation of a new oral therapeutic targeting Campylobacter and Enterotoxigenic Escherichia coli (ETEC) developed in collaboration with Immuron.

The Phase 2 clinical trial is designed to evaluate the safety and protective efficacy of the new product compared to a placebo in a controlled human infection model (CHIM). The primary efficacy outcome is prevention and/or reduction of moderate to severe diarrhea. ClinicalTrials.gov Identifier: NCT06122870.

The company also received a bull case valuation of A$0.35 and a base case valuation of A$0.25 from Pitt Street Research in a report.

The analysts highlight that the key catalysts for realising this potential shareholder value are the advancement of the company’s clinical-stage assets through the clinic with the end goal of bringing these assets to market.

Orthocell

Orthocell Ltd (ASX:OCC, OTC:ORHHF) has reported a noteworthy increase in its half-yearly revenue, reaching $3.2 million, a 51% rise from the previous half-year's $2.1 million as of December 31, 2022. This revenue growth is primarily attributed to a 68% surge in product sales up to 31 December 2023. Following the launches of Striate+™ in the US and Remplir™ in Australia in Q2 FY23, the company's half-yearly revenue has consistently grown by an average of 23%, compounded over the last two fiscal halves.

The company has strengthened its long-term relationship with the University of Western Australia (UWA) by converting all royalty entitlements into equity. Under this arrangement, Orthocell issued 1.70 million fully paid ordinary shares to UWA at a deemed price of $0.35 per share, increasing UWA's holding to 2.35 million shares or 1.18% of Orthocell's equity on an undiluted basis.

The appointment of new chair John Van Der Wielen has ushered in a new era of governance and board rejuvenation. The addition of two prominent Independent Directors, Professor Fiona Wood AM in October 2023 and the Hon Kim Beazley AC in January 2024, has resulted in a majority-independent board. This strategic board composition, along with a strong management team, positions Orthocell favorably for further growth in product sales through partnerships with BioHorizons and Device Technologies.

Orthocell has initiated a nerve repair study vital for US regulatory approval, with completion expected in the third quarter of CY2024. This study aims to gather mechanism of action data, supporting marketing, regulatory approval, and reimbursement strategies.

Additionally, a pivotal tendon repair study has demonstrated that OrthoATI™ is as effective as surgery in treating lateral epicondylitis, showing significant pain resolution in patients one month post-treatment compared to six months in the surgery group. Orthocell plans to engage a US corporate adviser to find a strategic partner for OrthoATI™, aiming for advancement without substantial investment shortly.

With $19.6 million in cash reserves at the quarter's end, Orthocell is well-equipped to enhance commercial success with Striate+™ now approved in the US, EU/UK, and Australia, and Remplir™ approved in Australia. The company is also in a strong position to pursue US regulatory approval for Remplir, supporting its ongoing growth and product distribution initiatives.

“We are very pleased with the performance of our distribution partners and the continued increase in product revenue, OCC MD Paul Anderson said. “With a highly respected and experienced Board now in place, we are in a very strong position to continue to gain commercial traction and drive Remplir, our breakthrough nerve repair device, into global markets.”

Recce Pharmaceuticals

Recce Pharmaceuticals Ltd (ASX:RCE, OTC:RECEF) is developing a New Class of Synthetic Anti-infectives. 

During its December quarter, the Safety Committee approved an increased infusion rate of 15 minutes for a Phase I/II clinical trial targeting urinary tract infections (UTI) and urosepsis. This trial, focusing on rapid infusion methods, has begun dosing its first subjects from the recruited cohort.

Concurrently, research outcomes from the Murdoch Children’s Research Institute have shown positive results in combating Escherichia coli in UTIs through both intravenous infusion and a new direct-to-bladder delivery approach. Additionally, the institute reported significant reductions in Neisseria gonorrhoeae bacteria levels in another animal study.

In the financial and intellectual property arenas, Recce has been granted A$54.9 million by AusIndustry for its research and development in anti-infective and anti-viral programs. This funding is divided into A$43.7 million for anti-infective research and A$11.2 million for anti-viral initiatives. Furthermore, the company has secured a Canadian patent for its RECCE® Anti-Infectives, and its trademark has been accepted in Brazil.

These developments were part of the updates shared at Recce Pharmaceuticals' 2023 Annual General Meeting. The company also made a presentation at the Ord Minnett Healthcare Forum, detailing its progress and plans in the development of anti-infective treatments.

These steps forward in clinical trials, research outcomes, and corporate achievements indicate ongoing advancements in the treatment of UTIs and urosepsis, as well as broader efforts in the fight against infectious diseases.