Krystal Biotech

Sanofi Advances Respiratory Pipeline with Promising Amlitelimab Data in Heterogeneous Inflammatory Asthma

a month ago

• Sanofi's amlitelimab demonstrated clinically meaningful efficacy in heterogeneous inflammatory asthma, showing over 70% reduction in exacerbations in patients with specific biomarkers at week 60 of the TIDE-Asthma phase 2 study. • The company is expanding its respiratory portfolio with lunsekimig now targeting chronic rhinosinusitis and COPD alongside asthma, with phase 2 readouts anticipated in 2026. • Itepekimab, developed in partnership with Regeneron, is broadening its clinical program beyond COPD into chronic rhinosinusitis, with phase 3 readouts in COPD expected in H2 2025.

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.

Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions

2 months ago

• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors. • Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing. • Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.

FDA Grants Fast Track Designation to Sanofi's mRNA Chlamydia Vaccine Candidate

2 months ago

• The US FDA has granted fast track designation to Sanofi's mRNA vaccine candidate for chlamydia prevention, recognizing its potential to address a serious unmet public health need. • The vaccine aims to protect against primary genital tract infection and reinfection by Chlamydia trachomatis, with a Phase 1/2 clinical trial in adults aged 18-29 set to begin imminently. • With 129 million global chlamydia cases reported in 2020 and over 80% being asymptomatic, the vaccine could transform prevention efforts where current antibiotic treatments have failed to control rising infection rates.

Repurposing Losartan: Hypertension Drug Shows Promise for Treating Epidermolysis Bullosa in Children

2 months ago

• Losartan, a medication long used to treat high blood pressure in adults, has demonstrated promising results in healing lesions associated with epidermolysis bullosa (butterfly skin disease) in children. • Epidermolysis bullosa is a rare genetic condition affecting approximately 500 people in Spain, causing painful sores that can remain open for years due to the lack of collagen 7 protein. • This potential treatment option emerges alongside recent advances in gene therapy, with the European Medicines Agency recently approving Vyjuvek, which has shown to heal 70% of wounds within three months.

Dupilumab Shows Strong Efficacy in Chronic Spontaneous Urticaria Across All BMI Ranges

3 months ago

• Phase 3 LIBERTY-CSU CUPID trials demonstrate dupilumab's effectiveness in reducing itch and urticaria activity in patients unresponsive to H1-antihistamines. • Clinical benefits of dupilumab were consistent across all BMI ranges, including patients in obese categories, providing important efficacy data for diverse patient populations. • FDA review of dupilumab for chronic spontaneous urticaria is underway with a target action date of April 18, 2025, potentially offering a new treatment option for resistant cases.

FDA Grants Breakthrough Therapy Status to Radiprodil for GRIN-Related Seizure Treatment

3 months ago

• GRIN Therapeutics' radiprodil receives FDA Breakthrough Therapy designation for treating seizures in patients with GRIN-related neurodevelopmental disorder, showing 86% median reduction in seizure frequency during Phase 1b trials. • The investigational drug demonstrated significant efficacy in Phase 1b Honeycomb study, with 71% of patients achieving over 50% reduction in countable motor seizures and most patients showing behavioral improvements. • The company plans to initiate Phase 3 pivotal trials in mid-2025, backed by a $200M capital commitment from Blackstone Life Sciences, to evaluate radiprodil's impact on seizures and behavioral outcomes.

FDA Approves Vyjuvek: Breakthrough Topical Gene Therapy for Dystrophic Epidermolysis Bullosa

3 months ago

• The FDA has granted approval for Vyjuvek, making it the first-ever topical gene therapy authorized for treating dystrophic epidermolysis bullosa (DEB), a rare genetic condition causing severe skin fragility. • In pivotal clinical trials, Vyjuvek demonstrated remarkable efficacy with 65% of treated wounds achieving complete healing compared to 26% in the placebo group, offering new hope for DEB patients. • Krystal Biotech plans to launch Vyjuvek in Q3 at $24,250 per vial, with typical annual treatment costs estimated at $631,500, addressing a condition that currently burdens US families with monthly wound care costs of up to $10,000.

U.S. Gene Therapy Market Projected to Reach $22.23 Billion by 2034, Growing at 19.8% CAGR

3 months ago

• The U.S. gene therapy market is expected to grow from $4.37 billion in 2025 to $22.23 billion by 2034, driven by increasing investments, rising prevalence of chronic diseases, and integration of advanced technologies. • Gene silencing has emerged as the dominant segment in the market, while oncology applications currently lead but neurology treatments are anticipated to gain significant market share in the coming decade. • Artificial intelligence is revolutionizing gene therapy development by analyzing genomic data, identifying therapeutic targets, enabling personalized treatment strategies, and optimizing manufacturing processes to reduce costs.

Long-term EASE Trial Shows Promise: Birch Triterpenes Demonstrates Sustained Wound Reduction in Epidermolysis Bullosa Patients

3 months ago

• Phase 3 EASE trial reveals birch triterpenes (Filsuvez) demonstrates sustained reduction in wound burden for epidermolysis bullosa patients over 24 months of treatment. • The topical therapy showed a favorable long-term safety profile, with most adverse events being mild-to-moderate in severity among 77.1% of participants. • Study investigator Dr. Anna Bruckner reports encouraging results for patients with dystrophic and junctional epidermolysis bullosa, with no increased risk of wound infections or squamous cell carcinoma.

Abeona Therapeutics' Pz-cel BLA Resubmission Accepted by FDA for RDEB Treatment

4 months ago

• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB). • The FDA has set a PDUFA target action date of April 29, 2025, for its decision on pz-cel, an autologous cell-based gene therapy. • Abeona Therapeutics has made significant strides toward the potential commercialization of pz-cel in 2025, including discussions with payers and target treatment centers.

Krystal Biotech and CRISPR Therapeutics Announce Key Updates in Gene Therapy Programs

4 months ago

• Krystal Biotech reported a 473% increase in full-year revenue, driven by VYJUVEK sales, and anticipates European and Japanese regulatory decisions in 2025. • Krystal Biotech's KB407 for cystic fibrosis received full sanctioning for its Phase 1 CORAL-1 study, with interim molecular data expected mid-2025. • CRISPR Therapeutics reported positive momentum in the CASGEVY launch, with over 50 activated treatment centers and patients initiating cell collection globally. • CRISPR Therapeutics expects key clinical data updates in 2025 for CTX112 in oncology and autoimmune diseases, and for CTX310 and CTX320 in cardiovascular programs.

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.

Enhertu Approved for HER2-Low and HER2-Ultralow Metastatic Breast Cancer

5 months ago

• The FDA has approved Enhertu for HR-positive, HER2-low or HER2-ultralow metastatic breast cancer after endocrine therapy progression. • DESTINY-Breast06 trial data showed a 36% reduction in disease progression or death risk compared to chemotherapy. • Patients on Enhertu had a median progression-free survival of 13.2 months versus 8.1 months on chemotherapy. • This approval expands Enhertu's use to an earlier treatment setting and a broader patient population.

EMA Postpones Decision on Krystal Biotech's B-VEC for Dystrophic Epidermolysis Bullosa

5 months ago

• The European Medicines Agency (EMA) has delayed its decision on Krystal Biotech's B-VEC (beremagene geperpavec-svdt) for treating dystrophic epidermolysis bullosa (DEB). • The EMA's Committee for Medicinal Products for Human Use (CHMP) has requested additional written responses from Krystal Biotech to address remaining issues. • Krystal Biotech anticipates a CHMP opinion in the first quarter of 2025 and plans to launch B-VEC in Germany in the second quarter of 2025. • B-VEC, known as VYJUVEK® in the U.S., is a gene therapy designed to deliver the COL7A1 gene to treat DEB at the molecular level.

GSK's Nucala (Mepolizumab) Shows Promise in COPD Treatment, Regulatory Submissions Underway

5 months ago

• GSK's Nucala (mepolizumab) demonstrates a clinically meaningful reduction in moderate/severe exacerbations in COPD patients with an eosinophilic phenotype. • The MATINEE Phase III trial supports regulatory submissions for Nucala as an add-on maintenance treatment for COPD, potentially the first monthly biologic for this condition. • Regulatory reviews are underway in both the US (FDA) and China (CNMPA), with the US PDUFA date set for May 7, 2025. • Nucala, already approved for other IL-5 mediated conditions, could offer a targeted therapy for up to 40% of COPD patients with type 2 inflammation.

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.

Novartis and Apellis Advance C3G Therapies Towards FDA Submission

6 months ago

• Novartis' Fabhalta (iptacopan) demonstrated significant proteinuria reduction in the Phase III APPEAR-C3G trial, meeting its primary endpoint and supporting regulatory submissions. • Apellis' Empaveli (pegcetacoplan) showed a substantial reduction in proteinuria and C3c deposit clearance in the Phase III VALIANT trial, indicating early efficacy in C3G patients. • Nephrologists express a strong interest in new C3G therapies that can slow eGFR decline and reduce proteinuria, addressing the urgent need for innovative treatments. • Spherix data suggests treatment preferences may be influenced by administration route, with Fabhalta's oral administration potentially offering an advantage.

FDA Accepts Abeona Therapeutics' BLA Resubmission for Prademagene Zamikeracel in RDEB Treatment

6 months ago

• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB). • Pz-cel, an autologous cell-based gene therapy, aims to address the unmet needs of RDEB patients by providing collagen VII expression at wound sites. • The BLA is supported by data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up, showcasing clinical efficacy and safety. • The FDA has set a PDUFA target action date of April 29, 2025, with potential for Abeona to receive a Priority Review Voucher upon approval.

Intellia Therapeutics Initiates Phase 3 Trial of NTLA-2002 for Hereditary Angioedema

8 months ago

• Intellia Therapeutics has dosed the first patient in its Phase 3 HAELO trial evaluating NTLA-2002 for hereditary angioedema (HAE). • The HAELO trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE. • NTLA-2002, a single-dose CRISPR-based therapy, targets the _KLKB1_ gene to reduce plasma kallikrein activity and prevent HAE attacks. • Intellia anticipates completing enrollment in the second half of 2025 and plans for a potential U.S. launch in 2027, pending regulatory approval.