Clinical-stage gene therapy company Krystal Biotech Inc. (KRYS:NASDAQ), which utilizes its proprietary Skin TARgeted Delivery (STAR-D) platform to develop innovative redosable gene therapies for rare skin diseases, today announced "positive topline results from the pivotal GEM-3 trial of investigational beremagene geperpavec (B-VEC), now known as VYJUVEKTM, for the treatment of dystrophic Epidermolysis Bullosa (dystrophic EB)."
The company advised that in the GEM-3 study, topical VYJUVEKTM met the primary endpoint of complete wound healing and demonstrated statistical significance compared to placebo after six-months. The firm added that VYJUVEKTM is now the only genetically corrective dystrophic EB treatment that has successfully completed a double blinded Phase 3 trial.
Krystal Biotech indicated that 31 patients were enrolled in the GEM-3 trial and that 67% of all wounds treated with VYJUVEKTM met the primary endpoint of "investigator assessed complete wound healing" after a period of six months versus 22% of the wounds treated with placebo. Similarly, 71% of all wounds treated with VYJUVEKTM also successfully achieved the secondary endpoint identified as complete wound healing after three months compared to just 20% of wounds treated with placebo.
Krystal Biotech's Founder and COO Suma Krishnan commented, "Dystrophic Epidermolysis Bullosa is referred to as 'the worst disease you've never heard of' because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEKTM which showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds…we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need."
Bullous Disease Clinic Director and Associate Professor of Dermatology at Stanford University Dr. Peter Marinkovich, M.D., stated, "Today's positive B-VEC results represent the culmination of years of study on the molecular basis and genetic correction of this disease. Finally, dystrophic EB patients may have an easily administered genetically targeted therapy which has been shown to promote durable wound healing in this clinical trial. This is a long overdue milestone for patients living with this disease, and one that has potential to drastically change the treatment paradigm."
The company advised that based upon the results in the pivotal Phase 3 GEM-3 trial, it is planning to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in H1/22. The firm indicated that it sees this filing as the first step in its global commercialization efforts.
Krystal Biotech added that it intends to submit a Marketing Authorization Application (MAA) in Europe quickly after its U.S. BLA filing and is exploring similar paths in Japan and elsewhere. The company noted that it will keep producing VYJUVEKTM at its ANCORIS cGMP manufacturing facility and listed that it is now building a second and even larger facility called ASTRA, which is expected to be ready in 2022 that will aid in supporting its potential global product launch of VYJUVEKTM.
The company's Chairman and CEO Krish Krishnan remarked, "We founded Krystal less than six years ago with the goal of developing a non-invasive, genetically corrective therapy for dystrophic EB…These pivotal data provide important validation of our redosable gene delivery technology, emboldening us to expand our pipeline to address other genetic skin diseases, continue to explore the potential in genetic lung diseases, and invest in growing the platform capability to address new organ systems as well."
The report explained that the Phase 3 GEM-3 trial enrolled 31 patients ages 1 thru 44 at 3 sites for the purpose of evaluating the efficacy and safety of VYJUVEKTM for treating dystrophic EB. Each patient's wounds and condition were assessed by the study's investigator and patients were randomized to receive a once-weekly topical application of VYJUVEKTM. The same patient in many cases received treatment and a placebo on a primary wound pair.
The primary pre-established endpoint in the study was "wound healing determined by the Investigator in VYJUVEKTM treated wounds versus placebo treated at the six-month timepoints." A second primary endpoint measured "mean change in pain severity using either a VAS or FLACC-R Scale at weeks 22, 24 and 26."
While the double-blind study generally was set up to measure one wound pair in each participant with one treated with VYJUVEKTM and the other with placebo, subjects enrolled in the trial also received VYJUVEKTM treatments for other additional or secondary wounds which benefited both patients and treating physicians.
The company pointed out that "Dystrophic EB (DEB) is a rare and severe monogenic disease that affects the skin and mucosal tissues." The firm added that "DEB patients suffer from open wounds, which leads to skin infections, fibrosis which can cause fusion of fingers and toes, and ultimately an increased risk of developing squamous cell carcinoma, which in severe cases can be fatal."
The company described VYJUVEKTM as "an investigational non-invasive, topical gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds." The firm listed that VYJUVEKTM has been granted both FDA and EMA orphan drug status for use in treating DEB.
Krystal Biotech is an advanced, pivotal-stage gene therapy company headquartered in Pittsburgh, Pa. The firm utilizes its redosable gene therapy platform and internal manufacturing methods and expertise to create treatments for rare and serious diseases. The company is largely focused on developing novel topical and intradermal "off-the-shelf" therapies for orphan and rare dermatological conditions but is now exploring applications to treat many other more common skin conditions.
Krystal Biotech began the day with a market cap of around $885.3 million with approximately 22.24 million shares outstanding and a short interest of about 9.3%. KRYS shares opened nearly 130% higher today at $91.20 (+$51.39, +129.09%) over Friday's $39.81 closing price and reached a new 52-week high price this morning of $102.99. The stock has traded today between $83.76 and $102.99 per share and is currently trading at $89.34 (+$49.53, +124.42%).
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