Latest news with #RettSyndrome
Yahoo
4 days ago
- Business
- Yahoo
ProQR Announces Second Quarter 2025 Operating and Financial Results
Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseases Advancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett Syndrome Hosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 Phase 1 trial design and 2025 data expectations, plus updates across differentiated liver and CNS pipeline € 119.8 million cash and cash equivalents as of end Q2 – providing runway into mid-2027, not including additional potential milestones from Lilly partnership LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial and operating results for the second quarter ended June 30, 2025, and provided a business update. 'We continued to execute across our pipeline in the second quarter. Notably, we submitted the Clinical Trial Application for AX-0810, our lead RNA editing program targeting NTCP for cholestatic diseases, and expect to share initial data from the trial later this year,' said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR. 'As AX-0810 advances and we progress additional pipeline programs, including our first CNS program AX-2402 targeting MECP2 for Rett syndrome, we remain committed to delivering value by addressing high unmet need patient populations with innovative RNA editing therapies. We look forward to providing additional updates on our Axiomer pipeline and platform progress at our Analyst and Investor Event this fall.' Recent Progress In July, the Company presented at the RNA Editing Summit in Boston, highlighting applications of its Axiomer RNA editing platform technology in CNS. In June, ProQR announced it submitted at Clinical Trial Application (CTA) to the European Medicines Agency (EMA) for a first-in-human Phase 1 study of AX-0810, targeting NTCP in healthy volunteers. This milestone marks the first clinical entry for ProQR's Axiomer RNA editing platform. Pending regulatory clearance, the study is expected to commence at a single site in the Netherlands with initial data anticipated in Q4 2025. The trial will assess safety, tolerability, pharmacokinetics, and target engagement. In May, ProQR showcased its scientific leadership in RNA editing through multiple presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting and the Oligonucleotide and Peptide Therapeutics Conferenece (TIDES USA), highlighting advances in its Axiomer RNA editing platform, including novel applications. In April, ProQR announced the appointments of Dennis Hom as Chief Financial Officer and Cristina Lopez Lopez, MD, PhD as Chief Medical Officer. These key leadership appointments support the advancement of the Company's Axiomer platform technology and pipeline of RNA editing programs as it enters the clinical stage. Anticipated Upcoming Events ProQR will host a virtual Analyst and Investor Event in the fall to highlight a detailed overview of the AX-0810 Phase 1 trial design, set expectations for initial data in 2025, and provide broader pipeline updates, including for the first CNS program, AX-2402 for Rett syndrome. Additional information, including date and registration details for this event will be shared in a future announcement. Pipeline programs Program Target Indication Upcoming milestone AX-0810 NTCP Cholestatic diseases Q4 2025 initial Phase 1 data in healthy volunteers AX-2402 MECP2 Rett Syndrome (R270X) 2025 clinical candidate selection AX-2911 PNPLA3 MASH 2025 clinical candidate selection AX-1412 B4GALT1 Cardiovascular disease 2025 update on optimization for GalNAc delivery Continue to execute on partnership with Eli Lilly and Company (Lilly), with potential data updates, milestone income from the existing partnership, and an option to exercise for an additional five targets for expansion to a total of 15 targets, which would result in a $50 million opt-in payment to ProQR. Financial Highlights At June 30, 2025, ProQR held cash and cash equivalents of approximately € 119.8 million, compared to € 149.4 million at December 31, 2024. Net cash used in operating activities during the six-month period ended June 30, 2025 was € 27.2 million, compared to € 21.4 million used for the same period last year. During the first half of 2025, the Company achieved certain milestones in the collaboration agreement with Eli Lilly amounting to $2.0 million (~€ 1.8 million). Research and development (R&D) costs were € 23.7 million for the six month period ended June 30, 2025 compared to € 16.3 million for the same period last year. General and administrative costs were € 8.1 million for the six month period ended June 30, 2025 compared to € 6.5 million for the same period in 2024. Net loss for the six-month period ended June 30, 2025 was € 22.3 million, or € 0.21 per diluted share, compared to € 10.4 million, or € 0.13 per diluted share, for the same period last year. For further financial information for the period ended June 30, 2025, please refer to the Q2 financial report 6-K filing. About Axiomer™ ProQR is pioneering a next-generation RNA base editing technology called Axiomer™, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer™ 'Editing Oligonucleotides', or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer™ EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease. About ProQR ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell's own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Learn more about ProQR at Forward Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'continue,' "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, technology, strategy, preclinical and clinical model data, our initial pipeline targets and the upcoming strategic priorities and milestones related thereto, the continued advancement of our lead development pipeline programs, including ongoing and planned clinical trials, expectations regarding regulatory feedback and the potential registrational pathway for AX-0810 in NTCP for cholestatic diseases, the anticipated timing of initial Phase 1 clinical data for our lead program, AX-0810, in Q4 2025, and clinical updates across multiple programs in 2025, our Axiomer™ platform, including the continued development and advancement of our Axiomer platform, the therapeutic potential of our Axiomer RNA editing oligonucleotides and product candidates, the timing, progress and results of our preclinical studies and other development activities, including the release of data related thereto, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended benefits thereof, including timing for data updates, potential milestones, exercise of an option to expand targets and the receipt of an opt-in payment, our ability to selectively form new partnerships and enter into future collaborations, and our financial position and cash-runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those expressed or implied by these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our most recent annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market, economic sanctions and international tariffs; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical events and conflicts, high inflation, rising interest rates, tariffs and potential for significant changes in U.S. policies and regulatory environment. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law. ProQR Therapeutics N.V. Investor and media contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@ contact:Peter KelleherLifeSci AdvisorsT: +1 617 430 7579 pkelleher@ Financial Tables PROQR THERAPEUTICS Condensed Consolidated Statement of Financial PositionJune 30, December 31, 20252024 €1,000€1,000 Assets Property, plant and equipment13,25814,113 Investments in financial assets—— Non-current assets13,258 14,113 Cash and cash equivalents119,765149,408 Prepayments and other receivables3,9313,747 Other taxes583690 Current assets124,279 153,845 Total assets137,537 167,958 Equity and liabilities Equity Equity attributable to owners of the Company66,98388,560 Total equity66,983 88,560 Liabilities Borrowings—— Lease liabilities10,48111,067 Deferred income26,98529,429 Non-current liabilities37,466 40,496 Borrowings4,7274,582 Lease liabilities1,6541,567 Derivative financial instruments290468 Trade payables1,28316 Social securities and other taxes2811,478 Deferred income17,45021,942 Other current liabilities7,4038,849 Current liabilities33,088 38,902 Total liabilities70,554 79,398 Total equity and liabilities137,537 167,958 PROQR THERAPEUTICS Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income (€ in thousands, except share and per share data)Three month periodSix month period ended June 30, ended June 30, 20252024 20252024 €1,000€1,000€1,000€1,000 Revenue3,817 6,305 8,336 10,755 Other income158 156 380 366 Research and development costs(11,408)(7,048)(23,731)(16,331) General and administrative costs(4,816)(3,013)(8,050)(6,465) Total operating costs(16,224)(10,061)(31,781)(22,796) Operating result(12,249)(3,600)(23,065)(11,675) Finance income and expense1925136471,001 Results related to financial liabilities measured at fair value through profit or loss(104)195178127 Result before corporate income taxes(12,161)(2,892)(22,240)(10,547) Income taxes(18)200(18)197 Result for the period(12,179)(2,692)(22,258)(10,350) Other comprehensive income (foreign exchange differences on foreign operation)(682)85(1,053)276 Total comprehensive income (12,861)(2,607)(23,311)(10,074)Result attributable to Owners of the Company(12,179)(2,692)(22,258)(10,350) Non-controlling interests———— (12,179)(2,692)(22,258)(10,350) Total comprehensive income attributable to Owners of the Company(12,861)(2,607)(23,311)(10,074) Non-controlling interests———— (12,861)(2,607)(23,311)(10,074) Share information Weighted average number of shares outstanding1105,343,89781,665,565105,320,49581,618,038Earnings per share attributable to owners of the Company (Euro per share) Basic loss per share1(0.12)(0.03)(0.21)(0.13) Diluted loss per share1(0.12)(0.03)(0.21)(0.13) For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal. PROQR THERAPEUTICS Condensed Consolidated Statement of Changes in EquityAttributable to owners of the Company Numberof shares ShareCapital SharePremium Equity settledEmployeeBenefitReserve TranslationReserve AccumulatedDeficit TotalEquity €1,000€1,000€1,000€1,000€1,000€1,000 Balance at January 1, 2024 84,248,384 3,370 412,894 25,159 817 (400,850)41,390 Result for the period —————(10,350)(10,350) Other comprehensive income ————276—276 Recognition of share-based payments ———1,364——1,364 Treasury shares transferred(326,455)—————— Share options lapsed———(359)—359— Share options exercised / RSUs vested326,455—174(288)—288174 Balance at June 30, 2024 84,248,384 3,370 413,068 25,876 1,093 (410,553)32,854 Balance at January 1, 2025 107,710,916 4,308 483,812 26,248 1,350 (427,158)88,560 Result for the period —————(22,258)(22,258) Other comprehensive income ————(1,053)—(1,053) Recognition of share-based payments ———1,667——1,667 Treasury shares transferred(131,525)—————— Share options lapsed———(1,462)—1,462— Share options exercised / RSUs vested131,525—67(181)—18167 — Balance at June 30, 2025 107,710,916 4,308 483,879 26,272 297 (447,773)66,983 PROQR THERAPEUTICS Condensed Consolidated Statement of Cash FlowsThree month period Six month period ended June 30, ended June 30, 2025202420252024 €1,000€1,000€1,000€1,000 Cash flows from operating activities Net result(12,179)(2,692)(22,258)(10,350) Adjustments for: — Other income(158)—(380)— — Depreciation6757111,3531,402 — Share-based compensation9096281,6671,364 — Financial income and expenses(139)(513)(647)(1,001) — Results related to financial liabilities measured at fair value through profit or loss104(195)(178)(127) — Income tax expenses18(200)18(197)Changes in working capital(1,178)(4,614)(7,900)(13,838) Cash used in operations(11,948)(6,875)(28,325)(22,747) Corporate income tax (paid)/received(18)199(18)196 Interest received6176101,4051,542 Interest paid(52)(190)(261)(379) Cash flow from investing activities Increase in financial asset - current———(17,000) Purchases of property, plant and equipment(101)(267)(325)(999) Cash flow from financing activities Proceeds from exercise of share options—1267174 Repayment of lease liability(293)(294)(860)(875) Net decrease in cash and cash equivalents(11,795)(6,805)(28,317)(40,088) Currency effect cash and cash equivalents(854)62(1,326)133 Cash and cash equivalents, at beginning of the period132,41485,713149,408118,925 Cash and cash equivalents at the end of the period119,765 78,970 119,765 78,970 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
News.com.au
4 days ago
- Business
- News.com.au
Health Check: Neuren says the US Rett syndrome market is a case of glass half full
Neuren's US partner Acadia reports a 14% sales boost Look out for a slew of advanced clinical trial results in early 2026 IDT Australia shares plunge up to 39% after CEO departs 'with immediate effect' Despite firming US sales of its Rett syndrome treatment Daybue, Neuren Pharmaceuticals (ASX:NEU) says two-thirds of eligible patients are yet to try the drug. Neuren's US partner Acadia Pharmaceuticals overnight reported quarterly net sales of US$96.1 million, up 14% year on year. The Nasdaq-listed Acadia says a record number of patients received a shipment, growing for the third consecutive quarter. Acadia holds the global rights to Daybue and pays royalties to Neuren, which initially developed the drug. Affecting boys, Rett syndrome is a rare neurodevelopmental disorder resulting in slow development after the ages of six to 18 months. Acadia has guided to net sales of US$380-405 million, which at the current exchange rate will generate $62-67 million of royalties for Neuren in calendar 2025. Neuren earned $14.7 million in the June quarter, up 16% year on year. By their nature, royalties are pretty much pure profit. More patients – and they're more persistent Acadia says a record 987 patients received Daybue in the quarter, up from 954 in the March quarter. Despite concerns about side effects, 50% of patients persisted after 12 months' treatment. Neuren adds that 70% of active patients have now been on therapy for 12 months or longer. This was up from 65% previously. The company says two-thirds of the 5500 to 5800 diagnosed US patients are yet to try Daybue. The FDA approved the therapy in March 2023 and Acadia expects European assent in the March 2026 quarter. Neuren shares this morning spurted as much as 8%. They've more than doubled since their mid-April trough of $8.60. Investors spoil IDT's golden jubilee Veteran contract drug maker IDT Australia's (ASX:IDT) golden jubilee has been spoiled by a savage share rout, resulting from the news that CEO Paul McDonald would depart 'with immediate effect'. The company also issued a lacklustre trading update, flagging a boost in revenue for the year to June 30, 2025, but a widening loss. A former Pfizer exec, McDonald had been in the job for almost three years. Chairman Mark Simari becomes executive chair while the company searches for a new CEO. IDT says its full-year revenue should come in at $19.9 million, a 40% increase. The company cites an increase in disbursement revenue. This derived from raw material costs and equipment charged to customers at a 'modest' margin. IDT says mainly applying to new contracts, disbursements are a 'positive lead indicator'. IDT also expects a net loss of $7.5 million, compared with a $5.4 million deficit last year. This reflects $1.2 million of bad debts from two customers defaulting on payments. Founded in 1975, IDT has had a convoluted history, including missing out on a government funded Covid vaccine plant at the last minute. These days IDT pursues gene technology, antibody drug conjugates, medical marijuana and psychedelic treatments for mental disorders. The company is in the third year of its five-year strategy, spurred by a board 'refresh' in September 2022. IDT's $30 million worth is backed by the value of the company's Boronia premises in eastern Melbourne. This hard-to-replicate is in the books at a conservative $21 million. We're on track, say drug developers Investors can look forward to a slew of company-making trial results in 2026, according to presenters at this year's Bioshares summit in Hobart. Alzheimer's disease drug developer Actinogen Medical (ASX:ACW) says it's on track to report interim results from its advanced phase IIb/III trial, by January next year. The company is enrolling 220 patients for its Xanamia trial for mild to moderate sufferers, across 15 Australian and 20 US sites. Actinogen's candidate Xanamem targets excess levels of cortisol in the brain – a novel mechanism of action. The company expects to unveil data from the first 100 patients, after 24 weeks' treatment. Final results are expected in late 2026. Stem cell developer Cynata Therapeutics (ASX:CYP) expects to release results of its phase III osteoarthritis (OA) trial between February and April next year. Cynata's mesenchymal stromal cells modulate the immune system and enable tissue repair. The OA program is the most advanced of Cynata's four trials that also cover graft-versus-host disease, diabetic foot ulcers and kidney transplants. Vitiligo is a circa US$500 million market, says Clinuvel The developer of an approved treatment for a rare sun intolerance disorder abbreviated as EPP, Clinuvel Pharmaceuticals (ASX:CUV) is advancing its proposed treatment for the more common vitiligo. The company has fully enrolled its 210 patient phase III trial, with a readout due in the June half. Affecting about 1% of the population, vitiligo is skin discolouration that results from the destruction of pigment-producing cells called melanocytes. The late Michael Jackson was a celebrity sufferer. If the drug gets US Food and Drug Administration (FDA) approval, the company estimates US$490-570 million of revenue in years one and two. Put in context, Clinuvel reported revenue of $35.6 million in the 2024 December half, up 10%. These sales derived from the company's EPP treatment, Scenesse. Doing the numbers on Imricor Broker Canaccord reckons Imricor Medical Systems (ASX:IMR) could be worth more than twice its current valuation – but faces constrained hospital budgets for the time being. In a 60-page appraisal of the cardiac ablation catheter developer, the firm estimates 120 US hospitals could be using the device within five years. Assuming 35% penetration of the ablation procedures, this equates to a $2.50 per share valuation compared with around $1.40 now. 'The big question ... is how quickly can these installs happen,' the firm says. Uniquely, Imricor's catheters are guided in real-time with magnetic resonance imaging – MRI – rather than via x-ray. The procedure is called interventional cardiac magnetic resonance cardiac ablation (ICMR CA). The company initially is focused on the US$12b arrhythmia market. Canaccord believes Imricor is still 12-18 months away from a 'material revenue ramp'. But after that it's off to the races. Imricor has an initial US target market as 120 large academic hospitals, 20 of them high volume. The firm estimates that facilities will install 190 ICMR US labs globally five years post launch. Of these, 100 will be in the US with total revenue of US$250 million. Imricor currently is undertaking a trial aimed at FDA approval for atrial flutter procedures, with a decision by the end of 2026.
Yahoo
6 days ago
- Business
- Yahoo
Taysha Gene Therapies to Release Second Quarter 2025 Financial Results and Host Conference Call and Webcast on August 12
DALLAS, Aug. 05, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will report its financial results for the second quarter ended June 30, 2025, and host a corporate update conference call and webcast on Tuesday, August 12, 2025, at 8:30 AM Eastern Time. Conference Call DetailsToll Free: 877-407-0792International: 201-689-8263Conference ID: 13754869Webcast: About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company's management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit Company Contact:Hayleigh Collins Senior Director, Corporate Communications and Investor RelationsTaysha Gene Therapies, Media Contact:Carolyn HawleyInizio in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Associated Press
7 days ago
- Health
- Associated Press
Biomed Industries Presents Four Breakthrough Studies on Alzheimer's, Rett Syndrome, and Obesity Therapies at AAIC 2025
Biomed Industries, Inc. Presents Four Pivotal Studies at AAIC 2025 Highlighting Breakthrough Therapies for Alzheimer's, Rett Syndrome, and Obesity 'NA-831 is the only drug to date that has halted Alzheimer's disease progression. Combining it with existing drugs like Donanemab could optimize therapeutic efficacy and reduce serious side effects.'— Dr. Lloyd L. Tran, CEO of Biomed SAN JOSE, CA, UNITED STATES, August 4, 2025 / / -- Biomed Industries, Inc., a leading biopharmaceutical innovator developing transformative therapies for neurological and metabolic diseases, today announced the presentation of four major scientific papers at the Alzheimer's Association International Conference (AAIC), held July 27–31, 2025, in Toronto, Canada. The presentations featured Biomed's next-generation oral therapies for Alzheimer's disease, Rett syndrome, and obesity, with a focus on novel combination strategies designed to enhance safety, efficacy, and accessibility. Featured AAIC 2025 Presentations: 1. The End of the Amyloid Era? Evidence for a Paradigm Shift in the Quest to Treat Alzheimer's Disease 2. A Phase 3 Clinical Protocol of NA-831 Combined with Donanemab in Early Alzheimer's Disease: A Placebo-Controlled, Double-Blind Study 3. Associations Between Alzheimer's Disease and Rett Syndrome: Clinical Trials of NA-831 and NA-921 4. Neuro-Metabolic Link Between Alzheimer's Disease and Obesity: Clinical Evaluation of NA-831 and NA-931 1. PRESENTATION: The End of the Amyloid Era? A Paradigm Shift in Alzheimer's Research For over three decades, the 'amyloid hypothesis' has dominated Alzheimer's disease (AD) research, asserting that amyloid-β accumulation is a primary driver of neurodegeneration. Biomed's comprehensive analysis of Phase 3 trial data from seven anti-amyloid drugs — including aducanumab, lecanemab, donanemab, gantenerumab, bapineuzumab, crenezumab, and solanezumab — challenges this paradigm. Across all trials, both treatment and placebo groups exhibited similar cognitive decline, as measured by CDR-SB and ADAS-Cog. The average differences between treatment and placebo arms were minor and not clinically significant (CDR-SB: -0.25; ADAS-Cog: -0.79). Slope comparisons further revealed near-identical rates of decline between groups. 'Our analysis of Phase 3 clinical trial data for seven anti-amyloid drugs, including FDA-approved Aducanumab, Lecanemab, and Donanemab, indicates that none could halt disease progression in a clinically meaningful way — and all carried serious safety concerns,' said Dr. Zung Tran, VP of Biostatistics and AI at Biomed. 2. PRESENTATION: Phase 3 Clinical Protocol of NA-831 Combined with Donanemab NA-831, Biomed's lead oral candidate, is a first-in-class therapy that promotes neuroprotection, neurogenesis, and memory enhancement. Phase 2 trials demonstrated its disease-modifying potential with a favorable safety profile compared to traditional anti-amyloid drugs. In this upcoming Phase 3 study, NA-831 will be evaluated in combination with Donanemab, a recently FDA-approved monoclonal antibody, to explore synergistic effects. The goal is to lower Donanemab dosing, thereby reducing risks such as cerebral edema and microbleeds, while enhancing cognitive outcomes. 'NA-831 is the only drug to date that has halted disease progression in Phase 2 trials,' said Dr. Lloyd Tran, CEO of Biomed Industries. 'Combining it with existing drugs like Donanemab could optimize therapeutic efficacy and reduce serious side effects.' 3. PRESENTATION: Alzheimer's and Rett Syndrome: Shared Mechanisms and Dual Therapeutic Potential Biomed also presented findings on NA-921, a structural analog of NA-831 developed for Rett syndrome, a rare X-linked neurodevelopmental disorder. NA-921 modulates MeCP2 expression, targeting the disorder's core epigenetic dysfunction. A double-blind, placebo-controlled Phase 2/3 trial ( ID: NCT06849973) demonstrated promising results: Clinical Global Impression–Improvement (CGI-I) at week 12: NA-921: 3.60 | Placebo: 3.83 | P = 0.0020 | Effect size = 0.42 NA-921 was well tolerated, with a significantly improved safety profile compared to trofinetide: - Diarrhea: Trofinetide 82%, NA-921 24%, Placebo 19% - Vomiting: Trofinetide 29%, NA-921 9%, Placebo 11% - Fever: Trofinetide 9%, NA-921 5%, Placebo 4% These findings underscore a possible biological link between Alzheimer's disease and Rett syndrome, opening new cross-indication opportunities for NA-831 and NA-921. 4. PRESENTATION: Neuro-Metabolic Connections: NA-831 and NA-931 in Alzheimer's and Obesity Biomed's data also revealed compelling evidence of a neuro-metabolic bridge between Alzheimer's disease, diabetes, and obesity. In addition to NA-831's CNS benefits, Biomed is advancing NA-931, an oral quadruple receptor agonist (IGF-1, GLP-1, GIP, and glucagon) designed to treat obesity. In a 13-week Multiple Ascending Dose (MAD) study: - Mean body weight reduction: Up to 13.8% at 150 mg daily, 12.4% greater than placebo - ≥12% weight loss achieved by 72% of NA-931-treated patients vs. 2% in placebo group NA-931 demonstrated a strong safety profile, with mild and transient GI-related adverse events, and no observed muscle loss. 'Our pipeline shows how targeting interconnected pathways across the CNS and metabolic systems can unlock significant clinical potential,' said Michael Willis, VP of Business Development. 'With six active programs across Alzheimer's, ALS, Rett syndrome, stroke, obesity, and MASH, we are building a diversified platform to accelerate innovation and value creation.' ABOUT BIOMED INDUSTRIES, INC. Biomed Industries, Inc. is a pioneering biopharmaceutical company committed to developing novel therapeutics that address unmet medical needs. Its innovative research platform has produced treatments for conditions including Alzheimer's disease, ALS, Traumatic Brain Injury, Major Depressive Disorder, Diabetes, Obesity, MASH, Stroke, and rare diseases such as Huntington Disease and Rett Syndrome. (Website: ) Michael Willis Biomed Industries, Inc. email us here Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content 'as is' without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
News.com.au
03-08-2025
- Health
- News.com.au
Hope grows as cannabis contender enters the Rett syndrome fight
Rett syndrome is rare but relentless Neuren struck biotech gold with Daybue Neurotech brings cannabis to the fight Rett syndrome is the kind of diagnosis that hits hard and lingers long. It affects around one in 10,000 girls, and typically appears after what seems like a normal start to life. Then comes the regression - loss of speech, hand skills, mobility - followed by the onset of seizures, bone fragility, gut issues, scoliosis and, often, a haunting silence that replaces early babble. It's caused by mutations in the MECP2 gene, which plays a crucial role in brain development. While it's classed as rare, the ripple effects through families are enormous. Sleep disturbances are common. Breathing irregularities, like breath-holding spells, can leave caregivers powerless. And while most girls survive into adulthood, it's with round-the-clock support and complex medical needs. AussieRett and InterRett studies Professor Helen Leonard, principal research fellow at the Kids Research Institute, has spent decades studying the condition. Leonard's long-running AussieRett and InterRett studies have helped establish how Rett symptoms evolve over time, and her team has also created global care guidelines for things like nutrition, scoliosis and bone health. 'Rett syndrome is an unusual condition in that it mainly affects girls who, following a period of apparent normal development, gradually show signs of regression," she told Stockhead. "Between the ages of 6 and 18 months they lose skills, particularly in relation to hand function and communication. 'As well as loss of hand function, these individuals develop unusual patterns of hand movements, such as hand-wringing or clapping known as stereotypies." Neuren's moonshot moment Until recently, there was no approved treatment for Rett, only a patchwork of management strategies and hope. That changed when ASX-listed Neuren Pharmaceuticals (ASX:NEU) struck gold with its drug trofinetide, now marketed as DAYBUE in the US. Approved by the FDA in March 2023, DAYBUE became the first and only drug for Rett syndrome, unlocking a commercial windfall for Neuren. Since 2019, Neuren's stock has surged over 1,200%, and the company now commands a $2 billion market cap. Thanks to a savvy licensing deal with Acadia Pharmaceuticals, Neuren pockets royalties and milestone payments with no royalty outgoings - every dollar drops to the bottom line. 'The FDA approval of trofinetide for Rett syndrome is very exciting, and represents the first ever treatment for the disorder,' said Leonard. For investors, it was a reminder that rare paediatric disorders, long overlooked, are now a serious biotech frontier. And that's where Neurotech (ASX:NTI) enters the frame, with a somewhat different approach. Cannabis steps into the ring Neurotech's lead therapy, NTI164, is a full-spectrum cannabis extract containing a cocktail of cannabinoids like CBDA, CBC and CBN. But it only contains 0.08% THC, meaning it's non-intoxicating and suitable for children. The company recently published results from its Phase I/II study in the Journal of Paediatrics and Child Health. This report shows the therapy was well tolerated and offered signs of clinical improvement across neurological, behavioural and functional domains. The drug's unique formulation is designed to reduce neuroinflammation, support synaptic function and modulate glial cells - factors believed to play a key role in Rett's progression. NTI164 is gaining traction internationally. It's already secured Orphan Drug Designation (ODD) in the US and European Union. This unlocks a range of incentives, including market exclusivity, reduced regulatory fees and access to research funding. It's the kind of support that can help fast-track rare-disease drugs through the system. Meanwhile, data from the same study was presented by lead investigator Professor Carolyn Ellaway at the World Rett Syndrome Congress. That put NTI164 front and centre in a growing global conversation about next-gen Rett treatments. Caution, hope and next steps Research into cannabis for Rett is still early, and Leonard urges caution when interpreting results from small, open-label trials. 'I think that we need a larger national double-blind placebo-controlled study before making any judgement,' she said. 'I would hope that this would use an alternative outcome measure to the RSBQ.' Her research has shown that Rett's behavioural symptoms - like those measured by the RSBQ - tend to decrease with age; unlike its clinical severity, which often worsens. That disconnect, she believes, can muddy trial results, and partially explains why she urges a broader toolkit for measuring impact in future studies. Still, she acknowledged that apart from the Rett Syndrome Symptom Severity (RTT-SIS) scale, some of the other measures used in the NTI164 study 'were showing positive changes'. She's not easily swayed by early signals but she recognises momentum when it's building. And regulators seem to agree. A closer look under NTI's hood From a clinical standpoint, NTI164 is ticking key boxes. Its pharmacokinetic (PK) data shows rapid absorption, minimal THC exposure and consistent dosing with no cannabinoid build-up - making it suitable for chronic paediatric use. NTI164 has also shown promising results in other paediatric neurological conditions like autism and PANDAS/PANS, potentially supporting its use in Rett by building a broader safety and efficacy profile. Perhaps most notably, its primary cannabinoid, CBDA, doesn't just convert into CBD like many assume. It appears to act directly on the brain, interacting with receptors linked to mood and inflammation. That matters because Rett isn't just neurological, it's deeply inflammatory. 'The clear validation of systemic stability, safety and targeted therapeutic action highlights NTI164's potential as a disease-modifying therapy,' said Neurotech CEO, Dr Anthony Filippis. Whether that turns into a commercial home run is still to be seen, but NTI164 has already demonstrated solid safety and early signs of symptom relief. In Rett, that bar is high. So are expectations. And that's what makes Neurotech's path an interesting one to watch. At Stockhead we tell it like it is. While Neurotech is a Stockhead advertiser, it did not sponsor this article.
