Propagermanium

Amicus Therapeutics Reports 15% Revenue Growth in Q1 2025, Expands Portfolio with DMX-200 Licensing Deal

21 days ago

• Amicus Therapeutics reported Q1 2025 total revenue of $125.2 million, representing 15% year-over-year growth at constant exchange rates, driven by strong patient demand for both Galafold and Pombiliti + Opfolda. • The company has expanded its rare disease portfolio by in-licensing DMX-200, a Phase 3 treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no approved therapies and significant market potential. • Despite adjusting 2025 revenue growth guidance to 15-22%, Amicus remains on track to achieve GAAP profitability in the second half of 2025 and anticipates surpassing $1 billion in total sales by 2028.

FDA Confirms Proteinuria as Primary Endpoint for Dimerix's DMX-200 Phase 3 FSGS Trial

25 days ago

• The FDA has endorsed proteinuria as an acceptable primary endpoint for full marketing approval of Dimerix's DMX-200 in treating Focal Segmental Glomerular Sclerosis, potentially expediting the path to market. • Dimerix's DMX-200 has previously demonstrated positive effects on proteinuria in both pre-clinical and clinical studies, strengthening the significance of this regulatory development. • The FDA remains open to discussing endpoints that could support a potential Accelerated Approval application, offering hope for patients with this progressive kidney disease who have limited treatment options.

Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas

3 months ago

• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics. • February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million. • Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.

DXB Secures $107M Licensing Deal in Japan, Awaits Phase 3 Results

4 months ago

DXB has signed a significant $107M licensing deal in Japan for its FSGS clinical trial, marking its third such agreement before Phase 3 results are published. The company is on the brink of potentially securing accelerated FDA approval, with key milestones and results expected by August 2025.

Dimerix Licenses Phase 3 FSGS Drug DMX-200 to FUSO Pharma for ¥10.5 Billion

5 months ago

• Dimerix has granted FUSO Pharmaceutical Industries exclusive rights to develop and commercialize DMX-200 in Japan for focal segmental glomerulosclerosis (FSGS). • The agreement includes an upfront payment of ¥300 million to Dimerix, with potential milestone payments reaching up to ¥9.8 billion, plus royalties. • FUSO will manage development costs and regulatory submissions in Japan, while Dimerix retains rights outside of the licensed territories. • The Phase 3 ACTION3 study, conducted by Dimerix globally, will align with FUSO's efforts through a Joint Steering Committee to support global commercialization.

Telix's TLX250-CDx (Zircaix) BLA for Kidney Cancer Imaging Gains FDA Priority Review

5 months ago

• Telix Pharmaceuticals has submitted a Biologics License Application (BLA) to the FDA for TLX250-CDx (Zircaix) for kidney cancer imaging. • TLX250-CDx is an investigational PET drug product for the non-invasive diagnosis and characterization of clear cell renal cell carcinoma (ccRCC). • The FDA granted Priority Review and provided a PDUFA date of August 27, 2025, paving the way for a potential U.S. commercial launch in 2025. • Phase III ZIRCON trial results demonstrated 86% sensitivity, 87% specificity, and 93% positive predictive value for ccRCC across three independent radiology readers.

ASX Biotechs Gear Up for Critical Phase III Trial Results in 2025

5 months ago

• Several ASX-listed biotech companies are anticipating crucial Phase III clinical trial results in 2025, which could significantly impact their market value and future prospects. • Dimerix expects results from its ACTION3 Phase III trial of DMX-200 for focal segmental glomerulosclerosis (FSGS) by mid-2025, a rare kidney condition with no approved treatments. • Opthea anticipates Phase III trial results for sozinibercept (OPT-302) in combination with aflibercept or ranibizumab for wet age-related macular degeneration (wet-AMD) in 2025. • Recce Pharmaceuticals is set to commence a Phase III trial in Indonesia for RECCE 327 as a topical gel to treat diabetic foot infections (DFI) in early 2025.

Dimerix's Phase 3 Trial of DMX-200 for FSGS Completes Part 2 Recruitment, Interim Analysis Expected in August 2025

6 months ago

• Dimerix has completed the randomization of the first 144 patients in its ACTION3 Phase 3 trial of DMX-200 for Focal Segmental Glomerulosclerosis (FSGS). • A blinded interim analysis of the initial patient cohort is scheduled for August 2025 to assess statistical powering and confirm study continuation. • DMX-200 has Orphan Drug Designation in the US and Europe, potentially allowing for accelerated approval based on interim analysis and regulatory discussions. • The ACTION3 trial is a global, multi-center study evaluating DMX-200's efficacy and safety in FSGS patients already receiving an angiotensin II receptor blocker (ARB).

Dimerix's ACTION3 Phase 3 Trial for FSGS Receives Positive IDMC Review

6 months ago

• The Independent Data Monitoring Committee (IDMC) completed its fifth review of Dimerix's ACTION3 Phase 3 clinical trial data, with no safety concerns identified. • The IDMC recommended the continuation of the ACTION3 trial, which is evaluating DMX-200 for the treatment of Focal Segmental Glomerulosclerosis (FSGS). • DMX-200 offers hope for FSGS patients with limited treatment options, potentially avoiding the side effects of steroids and immunosuppressants. • The ACTION3 trial's design includes IDMC oversight and interim safety reviews to ensure patient safety and study integrity.

PARASOL Workshop Suggests Broader Proteinuria Endpoints for FSGS Drug Approval

7 months ago

• The PARASOL working group presented data at the American Society of Nephrology meeting, confirming the correlation between eGFR improvement and reduced risk of end-stage kidney disease. • Higher proteinuria thresholds (0.7 g/g to 1.5 g/g) have shown significant benefit in reducing the risk of renal failure progression in FSGS patients. • The FDA may now have sufficient data to grant approval based on proteinuria endpoints, potentially accelerating drug approval for FSGS treatments. • Dimerix intends to request a meeting with the FDA to discuss appropriate proteinuria endpoints for DMX-200 in the ACTION3 Phase 3 clinical trial.

Dimerix Partners with UK National Registry to Accelerate ACTION3 Trial for FSGS

8 months ago

• Dimerix collaborates with the UK's National Registry of Rare Kidney Diseases (RaDaR) to enhance patient recruitment for the ACTION3 Phase 3 trial. • RaDaR, the largest kidney registry globally, will identify and contact suitable FSGS patients in the UK for potential trial participation. • Dimerix has also received approval for its Investigational New Drug Application from the Thai Food and Drug Administration, paving the way for trial initiation in Thailand.

Dimerix's First Patient Completes Phase 3 Trial and Enters DMX-200 Open Label Extension Study for Kidney Disease

8 months ago

• Dimerix has enrolled the first patient into the Open Label Extension (OLE) study for its kidney disease drug candidate DMX-200 after completing the ACTION3 Phase 3 clinical trial. • The OLE study allows patients who have successfully completed the ACTION3 Phase 3 clinical trial to start or continue a two-year treatment of DMX-200 in an unblinded setting. • The OLE study will collect additional long-term data, such as safety and tolerability, to support future potential regulatory filings for DMX-200. • Interim analysis showed DMX-200 outperformed placebo in reducing proteinuria, with the Independent Data Monitoring Committee affirming no safety concerns.

Dimerix's DMX-200 Shows Promise in Phase 3 Trial for Rare Kidney Disease FSGS

a year ago

• Dimerix Limited's DMX-200 demonstrates successful interim results in a Phase 3 trial for Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease. • The trial included 72 patients and showed potential in treating FSGS, which currently has no approved drugs globally, marking a significant advancement. • Dimerix plans to expand the trial to include more countries, such as China, and broaden the patient base to include children over 12 years old. • The treatment, co-invented by researchers at the Harry Perkins Institute, could prevent kidney damage and the need for dialysis or transplantation in FSGS patients.

Drug Development Updates