Krystal Biotech

Cystic fibrosis causes mucus buildup in lungs and digestive system, affecting 40,000 in the U.S. CFTR modulators like Vertex's Trikafta treat symptoms by correcting the CFTR protein. Five biotech companies—Enterprise Therapeutics, Krystal Biotech, Recode Therapeutics, Sionna Therapeutics, and Vertex Pharmaceuticals—are developing transformative treatments, including nebulized formulations, gene therapies, and small molecules targeting CFTR mutations. The global cystic fibrosis therapeutics market is growing, but high drug prices remain a concern.

Krystal Biotech reported a profitable Q3 2024 with earnings per share of $0.95, up from $0.54 in Q2 2024, driven by VYJUVEK's $83.8 million in Q3 revenue and over $250 million since launch. The company is preparing for a CHMP opinion in Europe and a 2025 launch in Japan, with a strong financial position of $374 million in cash and investments. Krystal Biotech aims for 60% penetration of the identified patient pool within two years of VYJUVEK's launch and anticipates a $300 million milestone payment in early 2025. Despite increased R&D and SG&A expenses, the company secured over 460 reimbursement approvals for VYJUVEK and reported five consecutive quarters of positive EPS.

Krystal Biotech's Q3 2023 10-Q report highlights $83.8M in product revenue, $27.2M net income, and 92% gross margin. VYJUVEK, FDA-approved for DEB, achieved 97% positive access determinations. The company plans to launch VYJUVEK in Germany and Japan in 2025, with ongoing R&D in oncology, ophthalmology, and aesthetics. Challenges include dependence on VYJUVEK, biosimilar competition, and regulatory risks.

The global cell and gene therapy CRO market is projected to grow from $1.65 billion in 2024 to $3.51 billion by 2035, with a CAGR of 7.1%. Over 105 CROs are driving innovation, with 80% offering comprehensive support from R&D to clinical trials. Over 3,500 clinical trials are evaluating cell and gene therapies across 16 therapeutic areas, with North America leading in trial registrations and patient enrollment.

The global cell and gene therapy CRO market is projected to grow from $1.65 billion in 2024 to $3.51 billion by 2035, at a CAGR of 7.1%. This growth is driven by increasing demand for cell and gene therapies, with over 90 therapies approved globally and more than 3,500 clinical trials underway. The market features over 105 CROs offering services across preclinical, clinical, and commercial scales, with partnerships and collaborations fueling growth. The complexity and cost of developing these therapies are key drivers for outsourcing to CROs.

Krystal Biotech to report Q3 2024 financial results on Nov 4, 2024, with a conference call and webcast at 8:30 am ET. A 30-day replay will be available on the company's website.

Krystal Biotech to report Q3 2024 financial results on Nov 4, 2024, with a conference call and webcast at 8:30 am ET. A replay will be available for 30 days on the company's website.

Abeona Therapeutics resubmitted a BLA for pz-cel, a gene therapy for RDEB, addressing FDA's CMC concerns. Supported by phase 3 VIITAL study data, pz-cel showed significant wound healing and pain reduction. If approved, it would be the second RDEB gene therapy, following B-VEC's 2023 approval.

Krystal Biotech to report Q3 2024 financial results on Nov 4, 2024, with a conference call at 8:30 am ET. A webcast will be available, with a 30-day replay on the company's website.

AbbVie submitted a BLA to the FDA for accelerated approval of Teliso-V, targeting c-Met overexpressing NSCLC, based on phase II LUMINOSITY study data showing ORRs of 35% and 23% in c-Met high and intermediate patients, respectively. If approved, Teliso-V would be the first therapy for this indication. AbbVie's shares have risen 25.7% YTD.