Krystal is a biotechnology company focused on developing genetic medicines for diseases with high unmet needs. In 2023, the group launched its first FDA-approved product, VYJUVEK®, to treat the rare condition DEB. Its innovative platform uses engineered HSV-1 to deliver therapeutic genes to targeted cells, aiming for non-invasive treatments. With a growing pipeline of programs, Krystal is dedicated to advancing gene therapies for serious and rare diseases. Let’s take a closer look.
Krystal focus on four business segments: Dermatology, Respiratory, Oncology, and Ophthalmology.
- Dermatology: Focuses on skin health, offering treatments for conditions like acne, eczema, and skin cancer, while also addressing cosmetic concerns.
- Respiratory: This segment deals with managing and treating lung and airway diseases, from asthma and COPD to more severe respiratory infections.
- Oncology: Dedicated to developing breakthrough therapies and diagnostics to treat various types of cancer, aiming to improve survival rates and patient quality of life.
- Ophthalmology: Focuses on eye health, providing treatments for vision issues, cataracts, and diseases like glaucoma, to help preserve and restore sight.
KB105, Krystal’s gene therapy for TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI), is designed to fill the critical gap in treating this rare and severe skin disorder. ARCI, caused by mutations that compromise the skin barrier, leads to chronic and painful complications, with no current therapies addressing the genetic cause. KB105 delivers the TGM1 gene directly to affected areas of the skin, aiming for local correction and clinical improvement. Early trials have shown promising results, particularly at higher doses, with the therapy being well-tolerated and showing improvements in skin condition. Further trials are planned for 2024 to continue assessing its potential.
Krystal’s broader gene therapy platform, leveraging the HSV-1 vector, addresses key challenges like immune response and limited gene delivery capacity seen in other therapies. By modifying the virus, it has enabled safer, repeat dosing and expanded the ability to treat complex conditions. The platform’s success is exemplified by VYJUVEK, Krystal’s groundbreaking treatment for dystrophic epidermolysis bullosa (DEB). Approved by the FDA in 2023, VYJUVEK delivers functional copies of the COL7A1 gene directly to the skin, offering significant healing for DEB patients, a group that previously only had symptomatic treatments available. Already serving around 1,200 U.S. patients, with plans to expand into Europe and Japan by 2025, the company is set to bring this transformative therapy to a global market.
Krystal aims to develop redosable, inhaled gene therapies to address monogenic disorders of the lung. Company’s KB407 is designed to target cystic fibrosis (CF), the most common inherited disorder in the U.S., affecting thousands of individuals with mutations in the CFTR gene. CF causes severe lung disease due to mucus buildup, recurrent infections, and chronic inflammation, leading to progressive lung damage and reduced life expectancy. KB407 leverages group’s HSV-1 platform to deliver functional copies of the CFTR gene directly to the lungs via inhalation. Unlike traditional CFTR modulators, which only benefit certain mutation types, KB407’s gene therapy approach has the potential to address a broader range of CF patients, including those with mutations that current treatments don’t cover.
Krystal is advancing its oncology pipeline with KB707, a gene therapy targeting locally advanced and metastatic solid tumors, which are responsible for a significant portion of the nearly 10 million cancer deaths globally in 2020, including over 300,000 deaths in the U.S. in 2023. KB707 delivers IL-2 and IL-12 directly to the tumor microenvironment, promoting a localized immune response to clear tumors more effectively. With FDA Fast Track Designation for relapsed or refractory melanoma and solid tumors with lung metastases, KB707 is in early-phase trials aimed at addressing the unmet needs of patients who have exhausted standard treatments.
It’s making strides in ophthalmology with Ophthalmic B-VEC, a redosable eye drop designed to treat ocular complications in DEB, a rare genetic disorder that leads to severe blistering and scarring in mucosal tissues, including the eyes. DEB patients often face corneal erosions and vision loss due to defective COL7 anchoring fibrils. Ophthalmic B-VEC delivers the COL7A1 transgene directly to eye cells, allowing them to produce functional COL7 protein and restore eye tissue integrity. With over 750 U.S. and 2,000 global patients affected, this therapy addresses a critical gap where no FDA-approved treatments exist. Krystal’s HSV-1 platform allows for larger gene delivery and repeat dosing, positioning it ahead in treating front-of-eye conditions.
Krystal Website: Slit lamp pictures of the right eye. A: Baseline ankyloblepharon. The visual acuity was hand motion (HM) B: Ocular surface of the right eye 6 months after the surgery after B-VEC applications.
The gene therapy field is highly competitive, with numerous companies developing treatments across a range of conditions. In DEB, Krystal competes with corrective approaches from Abeona Therapeutics and Castle Creek Biosciences, while others like Chiesi Farmaceutici focus on palliative treatments. In cystic fibrosis, major players like Vertex and ReCode Therapeutics are advancing nucleic-acid-based therapies. For alpha-1 antitrypsin deficiency, companies such as CSL and Takeda dominate with augmentation therapies, while newer approaches, like gene silencing and editing, are in development. In oncology, established names like Merck and Pfizer are pushing forward therapies for solid tumors.
Since the commercial launch of VYJUVEK in the U.S. in Q2 2023, Krystal has reported $50.7 million in net product revenue. Analysts are optimistic, projecting sales to reach nearly $300 million in 2024 and $670 million by 2026. This year could mark a turning point with the company expected to achieve positive EBITDA of $62 million, rising to $281 million by 2026, alongside a projected net income of $316 million. These outcomes depend on Krystal’s ability to advance VYJUVEK and secure FDA approvals for its other products.
With a market cap of nearly $5.2 billion, the company’s stock price has surged 1800% since 2018, reflecting strong investor confidence in its commercialization potential. However, the high valuation—seen in a P/E ratio of 318x for 2023 and 70x for 2024—indicates that any setbacks in product approvals or commercialization could lead to a significant drop in stock price.
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