Krystal Biotech (KRYS) announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of Vyjuvek (beremagene geperpavec-svdt, or B-VEC) for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB). The intended DEB patient population includes those who have mutations in the COL7A1 gene starting from birth.DEB is a rare and severe monogenic disease that affects the skin and mucosal tissues. I ...

EC approval decision anticipated in second quarter of 2025PITTSBURGH, Feb. 28, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today welcomed the European Medicines Agency's (EMA’s) announcement that its Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for the European Commission (EC) to approve VYJUVEK® (beremagene geperpavec-svdt, or B-VEC) for the treatment of wounds in patients with dystrop ...

Company Overview - Krystal Biotech, Inc. is a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to address diseases with high unmet medical needs [3] - The company's first commercial product, VYJUVEK®, is the first-ever redosable gene therapy and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa [3] - Krystal Biotech is advancing a robust pipeline of investigational genetic medicines across various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3] Upcoming Events - The company will participate in the TD Cowen 45th Annual Health Care Conference on March 4, 2025, in Boston [1] - Krish S. Krishnan, the Chairman and CEO, will engage in a fireside chat at 1:50 pm ET and will host investor meetings throughout the day [1] - A webcast of the presentation will be available starting at 1:50 pm ET on March 4, 2025, and will be posted on the company's website [2]

Core Viewpoint - Investors are encouraged to consider Krystal Biotech, Inc. (KRYS) due to significant improvements in earnings estimates and positive short-term price momentum [1] Estimate Revisions - The upward trend in earnings estimate revisions indicates growing analyst optimism regarding Krystal Biotech's earnings prospects, which is expected to positively influence its stock price [2] - The Zacks Rank system, which ranges from 1 (Strong Buy) to 5 (Strong Sell), has shown that stocks with a Zacks Rank 1 have generated an average annual return of +25% since 2008, highlighting the potential for Krystal Biotech [3] Current-Quarter Estimates - For the current quarter, Krystal Biotech is projected to earn $1.52 per share, reflecting a remarkable change of +4966.67% from the previous year's reported figure [4] - The Zacks Consensus Estimate for the company has increased by 21.2% over the last 30 days, with one estimate rising and no negative revisions [4] Current-Year Estimates - For the full year, the expected earnings per share for Krystal Biotech is $6.64, indicating a year-over-year increase of +121.33% [5] - In the past month, four estimates have been raised while one has been lowered, resulting in a 10.69% increase in the consensus estimate for the current year [5] Zacks Rank - Krystal Biotech currently holds a Zacks Rank 2 (Buy), reflecting favorable estimate revisions and the potential for strong performance compared to the S&P 500 [6] Investment Outlook - The stock has gained 21.4% over the past four weeks, driven by solid estimate revisions, suggesting that its earnings growth prospects may lead to further stock price increases [7]

Core Insights - Zacks Premium offers tools for investors to enhance their stock market strategies and confidence [1] - The Zacks Style Scores are designed to help investors select stocks with the highest potential to outperform the market in the short term [2] Zacks Style Scores Overview - The Style Scores categorize stocks into four types: Value Score, Growth Score, Momentum Score, and VGM Score, each focusing on different investment strategies [3][4][5][6] - Value Score identifies undervalued stocks using financial ratios [3] - Growth Score assesses a company's future growth potential based on earnings and sales [4] - Momentum Score tracks price trends to capitalize on upward or downward movements [5] - VGM Score combines all three styles to provide a comprehensive evaluation of stocks [6] Zacks Rank and Performance - The Zacks Rank is a proprietary model that uses earnings estimate revisions to identify strong investment opportunities [7] - Stocks rated 1 (Strong Buy) have historically achieved an average annual return of +25.41%, significantly outperforming the S&P 500 [8] - Investors are encouraged to focus on stocks with a Zacks Rank of 1 or 2 and Style Scores of A or B for optimal returns [9] Stock Highlight: Krystal Biotech, Inc. (KRYS) - Krystal Biotech is a biotechnology company focused on genetic medicines for unmet medical needs [11] - KRYS holds a Zacks Rank of 2 (Buy) and a VGM Score of B, with a Momentum Style Score of A [11] - The stock has increased by 21.4% in the past four weeks, indicating strong momentum [11] - Recent upward revisions in earnings estimates for fiscal 2025 have raised the consensus estimate to $6.64 per share, reflecting a positive outlook [12]

Core Viewpoint - Krystal Biotech, Inc. (KRYS) has received an upgrade to a Zacks Rank 2 (Buy), indicating a positive outlook on its earnings estimates, which is a significant factor influencing stock prices [1][4]. Earnings Estimates and Ratings - The Zacks rating system is primarily based on a company's changing earnings picture, with the Zacks Consensus Estimate tracking EPS estimates from sell-side analysts [2]. - The recent upgrade reflects an upward trend in earnings estimates, which is correlated with near-term stock price movements [3][5]. Impact of Institutional Investors - Changes in earnings estimates significantly influence institutional investors' valuation models, leading to buying or selling actions that affect stock prices [5]. - The rising earnings estimates for Krystal Biotech suggest an improvement in the company's underlying business, likely resulting in increased stock prices as investors respond positively [6]. Earnings Estimate Revisions - Empirical research supports a strong correlation between earnings estimate revisions and near-term stock movements, making tracking these revisions beneficial for investment decisions [7]. - The Zacks Rank system effectively utilizes earnings estimate revisions to classify stocks, with a proven track record of performance [8]. Specific Earnings Projections - Krystal Biotech is projected to earn $5.62 per share for the fiscal year ending December 2025, reflecting a year-over-year increase of 87.3% [9]. - Over the past three months, the Zacks Consensus Estimate for Krystal Biotech has increased by 3.1%, indicating growing analyst confidence [9]. Zacks Rating System Overview - The Zacks rating system maintains a balanced distribution of 'buy' and 'sell' ratings across its universe of over 4000 stocks, with only the top 20% receiving favorable ratings [10][11]. - The upgrade to Zacks Rank 2 positions Krystal Biotech among the top 20% of stocks in terms of earnings estimate revisions, suggesting potential for market-beating returns in the near term [11].

Group 1 - Brendan completed a Ph.D. at Stanford University in organic synthesis in 2009 [1] - He worked for Merck from 2009 to 2013 and has experience in biotech startups such as Theravance and Aspira [1] - Brendan is a co-founder of 1200 Pharma, which spun out of Caltech and received significant investment in the 8 figures [1] Group 2 - Brendan remains an avid investor focused on market trends, particularly in biotechnology stocks [1]

Core Viewpoint - Krystal Biotech reported strong earnings per share for Q4 2024, driven by the growing traction of its gene therapy product, Vyjuvek, despite missing revenue expectations [1][2]. Financial Performance - Q4 2024 earnings per share were $1.52, exceeding the Zacks Consensus Estimate of $1.29, compared to $0.30 in the same quarter last year [1] - Total revenues for Q4 2024 were $91.1 million, falling short of the Zacks Consensus Estimate of $97 million, with revenues solely from Vyjuvek sales, up from $42.1 million in the year-ago quarter [2] - For the full year 2024, total revenues reached $290.5 million, slightly below the Zacks Consensus Estimate of $292.1 million, compared to $50.7 million in 2023 [8] - Full-year earnings per share were $3, surpassing the Zacks Consensus Estimate of $2.9, compared to $0.39 in the previous year [8] Product Development and Market Access - Vyjuvek, approved by the FDA in 2023, is the first revocable gene therapy for treating dystrophic epidermolysis bullosa (DEB) [3] - As of February 2025, Krystal Biotech secured over 510 reimbursement approvals for Vyjuvek in the U.S., achieving positive access determinations for 97% of lives covered under commercial and Medicaid plans [4] - The gross margin for the reported quarter was 95%, with patient adherence to weekly treatment at 85% as of the end of 2024 [4] Research and Development - Research and development expenses for Q4 2024 were $13.5 million, a 19% increase year over year, while selling, general, and administrative expenses totaled $31.3 million, up 26% from the previous year [7] - The company is advancing a robust pipeline of investigational genetic medicines across various fields, including respiratory, oncology, dermatology, and aesthetics [10][11][12][13][14][15][16] Market Reaction - Despite mixed earnings results, Krystal Biotech's shares rose by 12.4% following the announcement, attributed to record revenue growth from Vyjuvek sales [5] - Over the past three months, shares of Krystal Biotech have decreased by 3.4%, while the industry has seen a growth of 0.8% [5]

Financial Data and Key Metrics Changes - For the year 2024, net income per share was $3.12, up from $0.40 per share in 2023, marking the sixth consecutive quarter of positive EPS and fourth straight quarter of sequential earnings growth [10] - Net revenue for VYJUVEK in Q4 was $91.1 million, bringing total revenue for 2024 to $290.5 million, with gross margins consistent with prior quarters [12][46] - The company ended Q4 with $344.9 million in cash on hand and $749.6 million in total cash plus short-term and long-term investments, reflecting a quarterly growth of about $55 million [49] Business Line Data and Key Metrics Changes - VYJUVEK's U.S. launch is progressing well, tracking in the top tier of rare disease launches, with strong early signs of efficacy [7] - Jeune Aesthetics reported strong Phase 1 data for KB301, with plans to start a Phase 2 study later in 2025, achieving $341 million in net revenue within 18 months of launch [11] Market Data and Key Metrics Changes - The U.S. market for VYJUVEK has seen over 500 reimbursement approvals, with a strong access landscape continuing into 2025 [14] - The company expects to launch VYJUVEK in the EU and Japan, with over 1,000 identified deaf patients in Germany and France [30][72] Company Strategy and Development Direction - The company aims to successfully launch VYJUVEK in the EU and Japan, translate early efficacy signs into strong results across cystic fibrosis and alpha-1 antitrypsin programs, and prepare for the anticipated launch of B-VEC for treating lesions in the eye of DEB patients [8][9] - The company is committed to treating DEB globally and is progressing its second product, KB803, for ocular complications associated with DEB [33] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving a global peak sales estimate of over $1 billion for VYJUVEK, emphasizing the strong patient benefits and compliance rates observed [51][56] - The company is aware of potential competition in the DEB market but believes the value proposition of VYJUVEK will maintain its market share [90][91] Other Important Information - Research and development expenses for Q4 were $13.5 million, driven by increased clinical trial-related costs and manufacturing costs for pipeline candidates [47] - Selling, general, and administrative expenses for Q4 were $31.3 million, reflecting increased personnel-related costs and VYJUVEK sales and marketing costs [47] Q&A Session Summary Question: Was there any year-end stocking in Q4 for VYJUVEK? - Management confirmed there was no substantial difference in Q4 compared to previous quarters regarding stocking or inventory [61][62] Question: How is enrollment going for the CF study? - Enrollment in Cohort 3 has begun, and it is progressing well with several active sites [64][66] Question: What are the expectations for Europe in the second half of the year? - Approximately 600 patients are identified in Germany and 400 in France, with pricing strategies discussed for both markets [72][73] Question: Can you quantify the impact of the annual cap on VYJUVEK in 2024? - About 8% of commercial patients have been on the cap consistently since the beginning [102] Question: What is the update on the regulatory process? - The delay in the CHMP decision was due to the need for a favorable label, which has now been achieved [88]

Product Launch and Revenue - VYJUVEK, the first FDA approved treatment for dystrophic epidermolysis bullosa (DEB), was launched in the U.S. in August 2023, with reported net product revenue of $341.2 million since launch[30]. - The company estimates over 3,000 DEB patients in the U.S., with 1,200 identified at launch through analytics and pre-launch activities[30]. - As of February 2025, the company secured positive coverage decisions from plans covering over 97% of commercial and Medicaid lives for VYJUVEK[31]. - The FDA approved VYJUVEK on May 19, 2023, and issued a Rare Pediatric Disease Priority Review Voucher (PRV), which was sold for $100 million in Q3 2023[34]. Clinical Trials and Studies - The company initiated Phase 1 testing of B-VEC in May 2018, with positive interim results announced in October 2018 and further positive results from the complete Phase 1/2 study published in Nature Medicine in March 2022[39]. - The GEM-3 trial for topical B-VEC enrolled 31 participants and showed positive results, with full results published in December 2022[40]. - The OLE study enrolled 47 participants, with no new safety concerns noted, and concluded in Q3 2023[41]. - The Japan OLE study was initiated and completed enrollment, with all four patients achieving complete wound closure at six months[42]. - KB407, a gene therapy for cystic fibrosis, has been dosed in the Phase 1 CORAL-1 study, with interim safety data showing mild to moderate adverse events[54]. - KB408 for AATD received FDA ODD designation, with successful gene delivery and AAT expression observed in the SERPENTINE-1 study[59][60]. - KB707 is an immunotherapy targeting solid tumors, utilizing a vector platform for sustained cytokine expression in the tumor microenvironment[64]. - In the KYANITE-1 study, inhaled KB707 demonstrated an objective response rate (ORR) of 27% and a disease control rate (DCR) of 73% in patients with advanced non-small cell lung cancer (NSCLC)[69]. - The initial clinical update for inhaled KB707 included safety data for 37 patients, with no Grade 4 or 5 adverse events observed[69]. - The OPAL-1 study for intratumoral KB707 began dosing patients in October 2023, with ongoing evaluations for safety and efficacy[71]. - Initial Phase 2 data for KB105 showed improvement on the Investigator's Global Assessment (IGA) scale, with maximum effects observed in high-dose treatment areas[76]. - The PEARL-1 trial for KB301, targeting aesthetic skin conditions, demonstrated positive proof-of-concept efficacy and safety data in March 2022[86]. - Enrollment in the KYANITE-1 and OPAL-1 studies is ongoing, with additional dose expansion cohorts being evaluated[70][71]. - KB301 showed positive interim safety and efficacy results in two new cohorts, with 94% of subjects in the décolleté region reporting increased satisfaction with wrinkle appearance[88]. - The company expects to complete the development of a décolleté-specific evaluation scale and dose the first subject in a Phase 2 study in the second half of 2025[88]. - KB304 is currently in a Phase 1 study with 21 subjects expected to be enrolled, randomized 2:1 to KB304 or placebo, with top-line results anticipated in the second half of 2025[92]. Manufacturing and Facilities - The company has established in-house CGMP manufacturing facilities to support the production of its gene therapy products[26]. - The company built in-house CGMP facilities, with the first facility, ANCORIS, producing VYJUVEK for commercial sales and receiving FDA and EMA certifications[95][96]. - The second commercial scale CGMP facility, ASTRA, was completed and qualified in 2023, adding significant capacity to support the growing pipeline[96]. - The proprietary manufacturing process for VYJUVEK ensures reproducible production of multiple commercial and clinical batches in a six-week cycle time frame[102]. Intellectual Property and Patent Strategy - The company actively seeks patent protection for its product candidates and proprietary technologies, evaluating its patent portfolio continuously[113][114]. - The technology platform VYJUVEK is primarily protected by composition of matter and methods of use patents, with key patents expiring on December 28, 2036[117]. - The company holds multiple patents for its product candidates, including U.S. patent 9,877,990 for compositions comprising HSV vectors, which is set to expire on December 28, 2036[119]. - Patent term extensions may be available under the Hatch-Waxman Act, allowing for up to 5 years of additional protection beyond the standard 20-year term[116]. - The company has a diverse patent portfolio across multiple regions, including patents in Japan, Australia, and Europe, all expiring in 2036[118]. - The company holds multiple patents related to pharmaceutical compositions for improving skin condition, with expiration dates set for April 26, 2039[127]. Regulatory and Compliance - The FDA regulates the company's biologic products, including gene therapy, under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act[130]. - The process for obtaining FDA marketing authorization involves extensive preclinical testing, IND application submission, and clinical trials[134]. - Clinical trials are conducted in three phases, with Phase 3 trials being pivotal for FDA approval, involving a larger patient population[139]. - The FDA requires annual progress reports detailing clinical trial results to ensure ongoing compliance and safety monitoring[140]. - The company must adhere to additional standards for gene therapy trials, including long-term monitoring of subjects for delayed adverse effects[141]. - User fees are associated with BLA submissions under the Prescription Drug User Fee Act, which are adjusted annually[142]. - The FDA imposes rigorous post-approval requirements, including compliance with CGMP regulations and reporting of adverse effects[159]. - Non-compliance with regulatory requirements can lead to marketing restrictions or product withdrawal, along with potential civil or criminal sanctions[160]. - The FDA's Rare Pediatric Disease Priority Review Voucher program allows sponsors to apply for priority review of subsequent marketing applications for different products[158]. Market and Competitive Landscape - The commercial success of VYJUVEK is critical for the company's near-term prospects and future growth[179]. - The company faces significant competition from other firms developing alternative therapies, which may have greater resources and faster regulatory approval timelines[187]. - The Biologics Price Competition and Innovation Act (BPCIA) allows for an abbreviated approval pathway for biosimilars, potentially impacting the exclusivity of VYJUVEK[184]. - The company has established several wholly-owned subsidiaries in Europe and Japan to support the commercialization of VYJUVEK and its product pipeline[176]. Financial and Operational Risks - The healthcare reform initiatives in the U.S. may affect drug pricing and reimbursement, impacting the company's revenue potential[170]. - The company may need to conduct expensive pharmacoeconomic studies to demonstrate the cost-effectiveness of its products to secure third-party reimbursement[168]. - The company faces potential litigation regarding patent rights, which could limit demand and pricing for VYJUVEK and future product candidates[189]. - Product liability lawsuits could lead to substantial liabilities, impacting the commercialization of VYJUVEK and other product candidates[190]. - Negative publicity regarding product safety could adversely affect financial condition and operational results[191]. - The company may need to increase product liability insurance coverage, which is becoming increasingly expensive[192]. - Public perception and regulatory scrutiny of gene therapy could hinder commercialization and regulatory approval of VYJUVEK[193]. - The company has experienced significant growth in personnel and infrastructure, which may pose management challenges[198]. - Retaining key employees and scientific advisors is critical for achieving research and commercialization objectives[199]. - Legislative changes, such as the IRA, could impact the company's ability to profitably sell VYJUVEK and other product candidates[207]. - The company is evaluating the uncertain effects of the IRA on its business operations and financial condition[208]. - Heightened governmental scrutiny over drug pricing could lead to regulatory changes affecting the company's market strategies[209]. Compliance with Healthcare Laws - The company is subject to various federal and state healthcare fraud and abuse laws, which could result in substantial penalties if not complied with[211]. - The federal Anti-Kickback Statute prohibits remuneration for the purchase or recommendation of items reimbursable under federal healthcare programs, impacting sales and marketing practices[212]. - Non-compliance with healthcare laws may lead to civil and criminal penalties, damages, fines, and exclusion from government healthcare programs[213]. - The company faces significant costs to ensure compliance with healthcare laws, which may affect financial operations and reputation[215]. Environmental and Safety Regulations - Environmental, health, and safety laws impose liabilities that could materially affect the company's business and financial condition[217]. - The company does not carry specific biological or hazardous waste insurance, increasing potential liability from contamination or injury[218]. - Future healthcare reforms may impose more rigorous coverage criteria and downward pressure on product pricing, impacting revenue generation[210]. - The company may incur substantial costs to comply with increasingly stringent environmental regulations, which could impair research and development efforts[218]. - Legal challenges related to compliance could divert management's attention and incur significant legal expenses[216]. - The company’s operations involve hazardous materials, and any resulting contamination could lead to liabilities exceeding available resources[217].