ADMA Biologics | MedPath

ADMA Biologics Secures FDA Clearance for Yield Enhancement Process, Poised for Margin Expansion in 2026

12 days ago

• ADMA Biologics has received FDA clearance for a yield enhancement process that is expected to significantly improve production margins for its immunoglobulin products starting in 2026. • The company plans to strategically allocate its strong cash flow toward debt reduction, commercial initiatives, R&D support, and potential stock repurchases while maintaining financial flexibility. • Health economic outcome data for ASCENIV is on track for publication later this year, aiming to demonstrate real-world benefits including reduced hospitalizations that justify its premium pricing to payers.

CG Oncology's Bladder Cancer Drug Shows Promising Durability in Phase 3 Trial

25 days ago

• CG Oncology's cretostimogene grenadenorepvec demonstrated a 75.5% complete response rate in high-risk BCG-unresponsive non-muscle invasive bladder cancer patients, with 34% maintaining complete response at 24 months. • The FDA has granted Fast Track and Breakthrough Therapy designations to cretostimogene, potentially expediting its regulatory pathway for a disease with limited treatment options. • The drug's 27.9-month median duration of response may provide a competitive edge over Johnson & Johnson's TAR-200, positioning CG Oncology as a significant player in the bladder cancer treatment landscape.

FDA Approves ADMA Biologics' Innovative Yield Enhancement Process for Immune Globulin Production

25 days ago

• ADMA Biologics has received FDA approval for a groundbreaking production process that increases immune globulin yields by approximately 20% from the same plasma volume, becoming the first U.S. company to achieve this regulatory milestone. • The yield enhancement technology will benefit both ASCENIV and BIVIGAM product lines, substantially increasing production capacity and supporting revenue growth beginning in late 2025 with further acceleration expected through 2026. • This innovation represents a significant advancement in plasma fractionation efficiency, potentially expanding access to immune globulin treatments for immunocompromised patients while improving the company's profit margins.

Ironwood Pharmaceuticals Faces Setback as FDA Requires Additional Phase 3 Trial for Apraglutide

a month ago

• Ironwood Pharmaceuticals' stock plummeted by 37% after the FDA required a confirmatory Phase 3 trial for apraglutide, a treatment for short bowel syndrome with intestinal failure. • Pharmacokinetic analysis revealed that exposure and dosing in the original STARS Phase 3 trial were lower than intended due to preparation and administration issues. • Despite promising efficacy data showing 27 patients achieving enteral autonomy in the long-term extension trial, the company has engaged Goldman Sachs to explore strategic alternatives.

Novartis Announces $23 Billion Investment to Expand US Manufacturing and R&D Footprint

a month ago

• Novartis plans to invest $23 billion over five years to establish seven new facilities and expand three existing ones across the United States, enabling 100% end-to-end US production of key medicines. • The expansion includes a $1.1 billion research hub in San Diego and new radioligand therapy manufacturing facilities in Florida and Texas, creating approximately 5,000 jobs directly and indirectly. • This strategic investment reverses Novartis' recent US pullback and comes amid trade policy uncertainties, positioning the company to maintain its projected sales growth of +5% CAGR through 2029.

Recursion Doses First Patient in Phase 1 Trial of REC-3565 for Relapsed B-cell Lymphomas

a month ago

• Recursion has initiated the EXCELERIZE Phase 1 clinical trial of REC-3565, a selective MALT1 inhibitor designed to treat relapsed or refractory B-cell lymphomas. • The AI-designed compound demonstrated strong preclinical results and features an allosteric design that potentially reduces liver toxicity risks associated with other MALT1 inhibitors. • Approximately 41,000 relapsed/refractory patients with CLL and B-cell lymphomas in the U.S. and EU5 are eligible for this potential treatment annually.

RSV Treatment Landscape Expands with New Vaccines and Clinical Trials in 2024-2025

3 months ago

• Moderna's mRESVIA vaccine received MHRA approval in February 2025 for adults aged 60 and older, joining GSK's Arexvy and Pfizer's Abrysvo in the expanding RSV vaccine market. • The FDA has mandated new safety warnings for RSV vaccines regarding Guillain-Barré Syndrome risk, highlighting ongoing safety monitoring efforts in early 2025. • Multiple pharmaceutical companies including Sanofi, GSK, and Pfizer are conducting advanced clinical trials in 2024, focusing on various patient populations and novel therapeutic approaches.

Protocol Amendment Expands Anixa's Ovarian Cancer CAR-T Trial: Second Dosing and New Cancer Types Added

3 months ago

• Anixa Biosciences and Moffitt Cancer Center receive approval for protocol amendment allowing second CAR-T doses in their ovarian cancer clinical trial, enhancing potential treatment benefits. • The amended protocol expands patient eligibility to include rare ovarian cancer types such as sex cord-stromal tumors and Sertoli Leydig cell tumors. • This development eliminates the need for individual IND approvals for second doses, streamlining the process for patients showing potential benefit from additional treatment.

Adaptive Biotechnologies Reports Strong MRD Business Growth in 2024 Financial Results

4 months ago

• Adaptive Biotechnologies reported 42% growth in its Minimal Residual Disease (MRD) business for 2024, with clonoSEQ test volume increasing 35% to 76,105 tests delivered throughout the year. • The company secured a 17% increase in Medicare reimbursement rates for clonoSEQ tests and expanded Medicare coverage to include Mantle Cell Lymphoma, enabling new promotional efforts. • Despite overall revenue growth of 5% to $179 million in 2024, Adaptive reduced its net loss to $159.6 million from $225.3 million in 2023, with a focus on achieving MRD profitability in 2025.

Phio Pharmaceuticals' PH-762 Shows Complete Tumor Clearance in Skin Cancer Trial

4 months ago

• Phio Pharmaceuticals reports two patients with cutaneous squamous cell carcinoma achieved complete tumor clearance in a Phase 1b trial of PH-762. • The ongoing study (NCT 06014086) evaluates the safety and tolerability of intratumoral PH-762 in various stages of skin cancers, including melanoma and Merkel cell carcinoma. • PH-762, utilizing Phio's INTASYL technology, targets PD-1 to enhance immune cell-mediated cancer cell killing, with no dose-limiting toxicities observed. • A partial response, with 90% tumor clearance, was observed in another patient with cutaneous squamous cell carcinoma, indicating promising early results for PH-762.

Arcturus Therapeutics Initiates Phase 1 Trial of mRNA Vaccine for H5N1 Influenza

4 months ago

• Arcturus Therapeutics has commenced a Phase 1 clinical trial for ARCT-2304 (LUNAR-H5N1), a self-amplifying mRNA vaccine targeting the H5N1 influenza virus. • The Phase 1 trial aims to evaluate the safety and immune responses of ARCT-2304 across different dose levels and vaccination schedules in approximately 200 healthy adults. • The trial is funded by the Biomedical Advanced Research and Development Authority (BARDA), highlighting the importance of pandemic preparedness efforts. • Interim Phase 1 data is anticipated in the second half of 2025, which will provide insights into the vaccine's potential for preventing H5N1 influenza.

Precision BioSciences' PBGENE-HBV Shows Promise in Phase 1 Hepatitis B Trial

5 months ago

• Precision BioSciences' PBGENE-HBV demonstrated a favorable safety profile in initial Phase 1 trial results, with no serious adverse events reported in the first cohort. • Early data indicates substantial reduction in Hepatitis B surface antigen (HBsAg) in two of three participants after a single dose administration. • The ELIMINATE-B trial is expanding globally, with active recruitment in Moldova, Hong Kong, and New Zealand, and plans for U.S. and U.K. expansion. • PBGENE-HBV leverages the ARCUS platform to target and eliminate covalently closed circular DNA (cccDNA), the root cause of chronic Hepatitis B.

FDA Accepts sBLA for Glofitamab Plus Chemotherapy in Relapsed/Refractory DLBCL

6 months ago

• The FDA has accepted Roche's sBLA for glofitamab combined with gemcitabine and oxaliplatin (GemOx) for relapsed/refractory DLBCL patients ineligible for autologous stem cell transplant. • The sBLA is based on the phase 3 STARGLO trial, which demonstrated a statistically significant and clinically meaningful improvement in overall survival compared to rituximab plus GemOx. • The FDA is expected to make a decision on the approval of glofitamab in combination with GemOx by July 20, 2025, offering a potential new treatment option. • The safety profile of glofitamab plus GemOx was consistent with the known safety profiles of the individual agents, with cytokine release syndrome being a common adverse event.

RSV Therapeutic Pipeline Shows Promise with Novel Therapies and Clinical Advancements

7 months ago

• Over 50 companies are actively developing more than 50 therapeutic options for Respiratory Syncytial Virus (RSV) treatment, indicating a robust pipeline. • GSK's Arexvy vaccine demonstrated encouraging Phase III results, preventing lower respiratory tract disease (LRTD) in older adults across three RSV seasons. • Merck's clesrovimab showed positive topline results in a Phase IIb/III trial as a preventive treatment for RSV in infants, highlighting potential for vulnerable populations.

AbbVie's Tavapadon Shows Positive Phase 3 Results for Parkinson's Disease

8 months ago

• AbbVie's tavapadon met its primary endpoint in the Phase 3 TEMPO-1 trial, significantly improving disease burden in early Parkinson's patients. • The trial also achieved a key secondary endpoint, demonstrating clinically meaningful improvements in motor aspects of daily living compared to placebo. • Tavapadon is a selective D1/D5 dopamine receptor partial agonist, potentially offering a novel approach to managing Parkinson's symptoms. • AbbVie plans to submit the TEMPO-1 data for regulatory approval, with results from the TEMPO-2 trial expected by the end of 2024.

Aldeyra Therapeutics Plans Reproxalap NDA Resubmission for Dry Eye Disease Following FDA Feedback

a year ago

• Aldeyra Therapeutics plans to resubmit its NDA for reproxalap to treat dry eye disease after addressing FDA concerns. • A new dry eye chamber study will be initiated in H1 2024 to assess ocular discomfort as the primary endpoint. • The company expects to resubmit the NDA in H2 2024, with a potential six-month review period by the FDA. • Aldeyra's cash reserves of $142.8 million are projected to fund operations beyond 2026, supporting ongoing clinical development.

Vertex Initiates Rolling NDA Submission for Suzetrigine in Acute Pain Management

a year ago

• Vertex Pharmaceuticals has begun a rolling NDA submission to the FDA for suzetrigine (VX-548) for moderate-to-severe acute pain, with completion expected in Q2 2024. • Phase III trials demonstrated suzetrigine's efficacy in reducing pain intensity across various surgical and non-surgical pain conditions, supporting the NDA submission. • Vertex is also advancing suzetrigine in neuropathic pain, with phase III trials planned for diabetic peripheral neuropathy (DPN) in the second half of 2024. • Suzetrigine holds FDA Fast Track and Breakthrough Therapy designations for acute pain and DPN, highlighting its potential to address unmet needs in pain management.

Stoke Therapeutics' STK-001 Shows Promising Results in Dravet Syndrome Trials

a year ago

• Stoke Therapeutics' STK-001 demonstrated substantial reductions in convulsive seizure frequency in children and adolescents with Dravet syndrome. • The treatment also showed improvements in cognition and behavior, suggesting potential disease modification in this patient population. • The FDA has allowed Stoke to proceed with a dosing regimen of 70mg followed by 45mg, paving the way for registrational studies. • Stoke Therapeutics is preparing to meet with regulatory agencies to discuss a registrational study based on the encouraging data.