Krystal Biotech, Inc.

Plus, news about Krystal Biotech and Curewell Capital: 🇨🇳 Two more Chinese biotechs file for IPOs in Hong Kong: LongBio Pharma and Shanghai Bao Pharmaceuticals both applied for an IPO on the Hong Kong Stock Exchange on Thursday. Bao has raised about $213 million to date and had about $63 million in cash and equivalents at the end of June, per the IPO filing. It’s working on treatments for cancer and bacterial infections, among other disease categories. LongBio had about $40 million in cash and equivalents at the end of June. The biotech is developing therapies for allergy and autoimmune conditions. — Kyle LaHucik 🦠 Lighthouse Pharmaceuticals gets $49.2M grant from NIH: The company plans to use the funding for a Phase 2 trial for patients with Alzheimer’s disease who have a confirmed Porphyromonas gingivalis infection. P. gingivalis is a bacterium associated with gum disease; the company said chronic infection with the bacterium can “contribute” to the progression of Alzheimer’s. — Jaimy Lee 🏛Krystal Biotech pauses advanced melanoma study: The company’s decision to pause enrollment in the Phase 1/2 study of intratumoral KB707 was reportedly due to uncertainty about the FDA’s approach to regulation, according to STAT . The regulator rejected a similar therapy from Replimune in July. Both treatments are engineered using a herpes virus. Krystal said Thursday that it is still studying an inhaled version of its therapy, called KB707, in lung cancer. — Lei Lei Wu 💵 Curewell Capital raises $535M, in part for US pharma services: The Los Angeles-based private equity company closed a fund of $535 million, which will be used to acquire US companies that do work in pharma and healthcare services, medical devices and health tech. The fund was raised less than five months after launch. — Anna Brown

PITTSBURGH, PA, USA I August 21, 2025 I Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced an update on development plans for KB707, the Company’s redosable immunotherapy designed to drive sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) in the tumor microenvironment and promote systemic immune-mediated tumor clearance following inhaled or intratumoral delivery to solid tumors. Based on promising early evidence of efficacy in the treatment of non-small cell lung cancer (NSCLC), the Company was granted an End of Phase 2 meeting with the United States Food and Drug Administration (FDA) in October to discuss potential development pathways to support the registration of inhaled KB707. “The acceleration of our work on inhaled KB707 is a reflection of both the clear and acute unmet need that exists for new treatments of NSCLC and the promising efficacy profile we have observed to date with inhaled KB707,” said Suma Krishnan, President of Research and Development of Krystal Biotech. “We look forward to meeting with the FDA and bringing another urgently needed therapeutic option to patients.” Evidence supporting the prioritization of inhaled KB707, including monotherapy activity and durable responses in heavily pre-treated patients with NSCLC, was disclosed by the Company at the 2025 American Society of Clinical Oncology Annual Meeting earlier this year. As of data cut-off of April 15, 2025, the objective response rate in this heavily pre-treated NSCLC patient cohort was 36%. Median duration of response and progression free survival were not reached. Inhaled KB707 continued to be safe and generally well tolerated and amenable to administration in an outpatient setting, with no Grade 4 or 5 adverse events observed. Enrollment continues in the Company’s KYANITE-1 study, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating inhaled KB707 in patients with locally advanced or metastatic solid tumors of the lung. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326. With the prioritization of inhaled KB707, the Company has paused enrollment in OPAL-1, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating intratumoral KB707 in patients with locally advanced or metastatic solid tumor malignancies. Patients on OPAL-1 continue to be followed and based on safety and efficacy results from OPAL-1, the Company may adjust development plans for intratumoral KB707. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT05970497. About Krystal Biotech, Inc. Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK ® , the Company’s first commercial product, is the first-ever redosable gene therapy, and the first genetic medicine approved in the United States, Europe, and Japan for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com , and follow @KrystalBiotech on LinkedIn and X (formerly Twitter). SOURCE: Krystal Biotech

iStock, Olena Koliesnik Krystal Biotech’s decision follows the FDA’s rejection last month of Replimune’s RP1, which works similarly to Krystal Biotech’s KB707. The biotech said this has introduced “heightened uncertainty” regarding a potential accelerated pathway for the candidate. Krystal Biotech has discontinued a Phase I/II study of its investigational intratumoral therapy KB707, which the Pennsylvania-based company was advancing for the treatment of solid tumors, in particular melanoma. This move comes after the FDA last month handed a surprise rejection to Replimune’s RP1, which works similarly to KB707. Both RP1 and KB707 are immunotherapies that use modified herpes simplex virus type 1 vectors to trigger an immune response against tumors. RP1 was also being developed for melanoma. In its news release on Thursday, Krystal announced that it had suspended enrollment into the Phase I/II OPAL-1 study , though it continues to monitor patients who had already been dosed for safety and efficacy. Based on the findings of the trial, Krystal “may adjust development plans” for intratumoral KB707. “Heightened uncertainty regarding potential accelerated approval pathways” for KB707 following the FDA’s rejection of RP1 contributed to the biotech’s decision, Stéphane Paquette, Krystal’s vice president for corporate development, told STAT News . The FDA’s rejection of RP1 was controversial. Earlier this month, both Endpoints News and STAT reported that Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office on Oncology Diseases, overruled agency reviewers and pushed for the candidate’s rejection. Shortly after the rejection, scientists who helped design and run Replimune’s Phase III IGNYTE trial urged the FDA to re-review the application. In an open letter signed by more than 20 melanoma and cancer experts, researchers addressed concerns that the agency raised in its complete response letter. For example, the experts argued that one issue cited by FDA reviewers, the heterogeneity of IGNYTE’s patient population, in fact provided a more accurate representation of the real-world scenario than a more uniform population would have. Melanoma treatment guidelines currently in place “show multiple treatment pathways in both the adjuvant and advanced disease setting funneling to the point of IGNYTE eligibility,” they wrote. Now, with intratumoral KB707 all but deprioritized, Krystal is turning its focus to an inhaled version of the drug, which it is developing for non-small cell lung cancer (NSCLC). The biotech announced on Thursday that it had been granted an End of Phase II meeting with the FDA following “promising early evidence of efficacy” of the candidate. A readout in June showed that inhaled KB707 elicited an objective response rate of 36% in heavily pretreated NSCLC patients, while median duration of response and progression-free survival were not reached. There were no grade 4 or 5 adverse events. The regulatory meeting is scheduled for October and will help Krystal determine potential pathways for bringing inhaled KB707 forward.