On October 15, 2026 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) reported a positive clinical update supporting initial proof of concept for KB113, an intratumoral 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, in the treatment of basal cell carcinomas (BCCs) in patients with Gorlin syndrome:

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Broad antitumor responses were detected in all three Gorlin syndrome patients treated with KB113, with over 80% of assessed BCC lesions reducing in size;
Responses were observed in both injected and non-injected lesions, including substantial regression in lesions that were not directly injected with KB113, consistent with an abscopal effect; and
Complete clearance of an injected lesion was also confirmed histologically for each of the three patients treated with KB113.

Gorlin syndrome is a genetic disease characterized by a greatly increased risk of developing BCCs. Patients with Gorlin syndrome can develop BCCs as early as infancy and may have hundreds of BCCs over their lifetimes requiring frequent and potentially disfiguring surgical procedures. The psychological and physical toll of Gorlin syndrome builds over time, as patients are subjected to repeated cycles of surveillance, surgery, and recovery to manage emergent BCCs, with each surgery adding to the cumulative risk of scarring and permanent tissue loss. Prevalence data for Gorlin syndrome is limited but available data suggest the number of patients with Gorlin syndrome in the United States could exceed 10,000.

"For individuals living with Gorlin syndrome, the lifelong emergence of multiple BCCs creates an immense cumulative treatment burden—often resulting in extensive scarring and significant impacts on quality of life," said Shireen V. Guide, MD, Mission Dermatology Center. "The observed antitumor responses with KB113 across both injected and non-injected lesions represent encouraging preliminary findings that strongly support its continued clinical development."

Gorlin Syndrome Pilot Study Clinical Results

KB113 is the low dose of intratumoral KB707 that showed promising early efficacy signals in patients with BCC in the Company’s OPAL-1 Phase 1/2 study, an open label, multicenter, dose escalation and expansion Phase 1/2 study in patients with injection-accessible solid tumors. The Gorlin Syndrome Pilot Study was a multicenter, single-arm, open label study designed to evaluate the safety and preliminary efficacy of KB113 for the treatment of BCCs in patients with Gorlin syndrome. Three adult patients with Gorlin syndrome were enrolled in the study. All three patients received six doses of up to 2 mL of 108 PFU/mL KB113 over a period of 14 weeks, with the first two doses spaced two weeks apart followed by dosing once every three weeks. Landmark lesions, defined as facial BCCs ≥5 mm in diameter or non-facial BCCs ≥9 mm in diameter, were identified by the investigator and visually assessed for clinical regression throughout the 14-week treatment period and again 30 days after the final dose using a pre-specified, investigator-assessed 5-point clearance scale. The scale categorizes each lesion by magnitude of regression. Only landmark lesions were eligible for injection with KB113. The investigator selected which landmark lesions to inject, irrespective of anatomical location, and determined the injection volume for each lesion based on its clinical presentation. Additional details on the OPAL-1 study can be found at www.clinicaltrials.gov under NCT identifier: NCT05970497.

Regression of landmark lesions was observed as early as two weeks after initiation of treatment, with responses broadening and deepening over time. At the final assessment conducted 30 days after last dose, over 80% of all assessed landmark lesions had regressed. Over half of all landmark lesions demonstrated greater than 50% clinical regression, and a subset demonstrated at least 90% clinical regression, as assessed using the investigator-assessed 5-point clearance scale. Substantial regression was observed in lesions that had never been directly injected, providing preliminary evidence of an abscopal effect.

Details on response rates at the final assessment are summarized below:

Combined Results for All Three Gorlin Syndrome Patients

Clinical Regression Injected Non-Injected Overall
Any clinical regression in lesion size* 29/36 (80.6%) 40/47 (85.1%) 69/83 (83.1%)
> 50% clinical regression** 15/36 (41.7%) 27/47 (57.4%) 42/83 (50.6%)
≥ 90% clinical regression 4/36 (11.1%) 4/47 (8.5%) 8/83 (9.6%)
* Inclusive of lesions with > 50% clinical regression
** Inclusive of lesions with ≥ 90% clinical regression

To further assess clinical regression, one injected lesion from each patient that had achieved at least 90% clinical regression was biopsied. All three biopsied lesions demonstrated histologically confirmed complete response, with no evidence of residual tumor tissue.

Consistent with prior experience in the low dose escalation cohort of OPAL-1, KB113 was well tolerated by all three patients enrolled in the Gorlin Syndrome Pilot Study. All reported adverse events were mild or moderate in severity, transient, and resolved during the study.

"The breadth and depth of responses reported today on landmark lesions located in various parts of the patient’s body showcase the systemic immune-activating potential of HSV-1 encoded IL-2 and IL-12 and have exciting implications for Krystal and the many patients we aim to serve," said Suma Krishnan, President of Research and Development at Krystal Biotech. "Leveraging our extensive HSV-1 dataset and clinical experience, we believe there is an opportunity to quickly advance our newly announced program for Gorlin syndrome and further our commitment to patients suffering from skin diseases."

The Company has initiated discussions with the United States Food and Drug Administration (FDA) regarding the advanced clinical development of KB113 for the treatment of Gorlin syndrome. The Company will provide an update on its clinical development plans following alignment with the FDA.

A poster summarizing the Gorlin Syndrome Pilot Study results will be presented by study investigator Dr. Guide at the 14th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference, October 15-17, 2026 in Alexandria, Virginia. The poster is available to view online on the Investor section of the Company’s website.

(Press release, Krystal Biotech, OCT 15, 2026, View Source [SID1234673496])

On October 15, 2026 Labcorp (NYSE: LH), a global leader of innovative and comprehensive laboratory services, and OPKO Health, Inc. (Nasdaq: OPK), a multinational biopharmaceutical company, reported that they have entered into an agreement for Labcorp to acquire select assets of BioReference Health, LLC, a wholly owned subsidiary of OPKO Health.

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Through this transaction, Labcorp will acquire BioReference Health’s laboratory testing businesses focused on clinical and women’s health testing services, urology, corrections and certain operational assets, including over 80 additional patient service centers. As part of the agreement, Labcorp will also acquire BioReference Health’s proprietary 4Kscore Test, an FDA-approved blood test that helps predict the likelihood of finding aggressive prostate cancer before a biopsy decision, making this important test more broadly available to patients across the country.

When complete, the transaction is expected to provide patients, physicians and customers with greater access to Labcorp’s comprehensive, high-quality laboratory services, including a broad specialty testing menu, convenient patient service center network and robust data analytics and digital tools.

"This acquisition strengthens our ability to deliver affordable, high-quality, specialized diagnostic services to more patients and providers around the country," said Bryan Vaughn, Executive Vice President and President of Diagnostics at Labcorp. "By expanding our clinical, women’s health, urology and corrections testing capabilities and broadening access to innovative diagnostics such as the FDA-approved 4Kscore Test, we can help providers make more informed decisions, improve patient care and increase access to the comprehensive laboratory services people need to live healthier lives."

"This transaction will allow OPKO to streamline operations and focus our resources on our biopharmaceutical business," said Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO. "Labcorp’s national scale and capabilities will broaden patient and provider access to BioReference’s 4Kscore Test to many more patients across the country. We believe this transaction is an important catalyst to further unlock additional value for our shareholders."

The purchase price for the transaction is $722 million in cash at closing, subject to an upward or downward adjustment (if any) based on closing business revenues, and up to an additional $150 million in an earnout based on revenue from the 4Kscore Test. Labcorp expects the business revenue to be approximately $250 million in the first year after close, based on Labcorp’s current post-closing forecasts.

This transaction is subject to regulatory approvals and customary closing conditions. Until the transaction closes, there will be no changes to operations or services, and both companies will continue to operate independently.

Lazard is serving as Labcorp’s financial advisor, Hogan Lovells Cadwalader US LLP is serving as lead transaction counsel and Parker Poe is serving as real estate counsel.

Piper Sandler & Co. is serving as OPKO’s exclusive financial advisor, and Greenberg Traurig P.A. is serving as legal counsel.

(Press release, Opko Health, OCT 15, 2026, View Source [SID1234673495])

On October 15, 2026 Revolution Medicines, Inc. (Nasdaq: RVMD), a global, commercial-stage oncology company dedicated to discovering, developing and delivering innovative medicines for patients with RAS-addicted cancers, reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of RASONQUE (daraxonrasib) once-daily oral tablets as a monotherapy indicated for the treatment of adult patients with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy. If approved by the European Commission (EC), RASONQUE would provide the first broad RAS-targeted treatment option in the European Union for adults with metastatic pancreatic adenocarcinoma.

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"Today’s positive CHMP opinion marks a major step toward making RASONQUE available to eligible adults with metastatic pancreatic cancer in the European Union," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "RASONQUE is an oral medicine designed to address RAS, the key driver of pancreatic cancer, and unprecedented results from the Phase 3 RASolute 302 trial demonstrated its potential to benefit patients with this devastating disease. Its expedited review under the EMA’s Cancer Medicines Pathfinder project highlights how innovative regulatory pathways may help bring promising medicines to patients sooner. We look forward to the EC’s consideration of this CHMP positive opinion as we continue to work with regulatory authorities around the world to pursue additional approvals and broaden access to RASONQUE."

RASolute 302: Phase 3 Clinical Trial Results Supporting Positive CHMP Opinion

The positive CHMP opinion is based on results from RASolute 302, a global, randomized Phase 3 trial evaluating RASONQUE versus investigator’s choice of cytotoxic chemotherapy in patients with previously treated metastatic PDAC. The trial met all primary and key secondary endpoints in both the RAS G12 mutant population and the overall intent-to-treat (ITT) population, which included patients with or without an identified tumor RAS mutation.

The primary analysis from RASolute 302 was presented at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting with simultaneous publication in The New England Journal of Medicine. In the ITT population, RASONQUE reduced the risk of death by 60% compared with chemotherapy, with a hazard ratio (HR) of 0.40 (95% confidence interval [CI]: 0.30–0.53; p<0.0001). The median overall survival (OS) was 13.2 months (95% CI: 10.0–NE) with RASONQUE compared to 6.7 months (95% CI: 5.8–8.0) for chemotherapy. RASONQUE also showed significant improvements in progression-free survival (PFS) with an HR of 0.49 (95% CI: 0.38–0.64; p<0.0001). The median PFS was 7.2 months (95% CI: 5.7–7.5) with RASONQUE compared to 3.6 months (95% CI: 2.9–4.2) with chemotherapy. Results were generally consistent in the RAS G12 population. A subsequent ad hoc OS analysis showed the median OS was 12.4 months with RASONQUE compared to 6.7 months for chemotherapy [HR: 0.49 (95% CI 0.39, 0.62)], which was generally consistent with the prespecified primary analysis.

The most common adverse reactions in patients treated with RASONQUE were diarrhea (63.1%), dermatitis acneiform (62.4%), stomatitis (55.5%), nausea (50.8%), vomiting (41.6%) and fatigue (33.2%).

Once granted by the EC, the centralized marketing authorization will be valid in all EU member states, as well as in the European Economic Area (EEA) countries Iceland, Norway and Liechtenstein; marketing authorization by the EC is not applicable in the United Kingdom or Switzerland, which have separate processes for considering new drug applications.

This CHMP Positive Opinion follows the approval of RASONQUE by U.S. Food and Drug Administration (FDA) on August 26, 2026.

About Pancreatic Adenocarcinoma

Pancreatic adenocarcinoma, or PDAC, is the most common form of pancreatic cancer and among the most lethal malignancies, frequently with a late diagnosis, aggressive biology, and limited responsiveness to available treatments. RAS, a key growth control switch in human cells, is the main driver of pancreatic cancer, which is characterized by excessive RAS signaling. Approximately 147,000 people are diagnosed with pancreatic cancer in Europe each year, and the five-year survival rate remains below 10%.1,2 Because early-stage pancreatic cancer often causes few or no symptoms, approximately 80% of patients are diagnosed after the disease has spread, when treatment options are more limited.3

About RASONQUETM (daraxonrasib)

RASONQUE (daraxonrasib) is an oral, RAS(ON) multi-selective, noncovalent, tri-complex inhibitor (TCI), approved by the U.S. FDA for the treatment of adult patients with metastatic PDAC who have received at least one prior systemic therapy or who are not candidates for multiagent systemic therapy.4 RASONQUE is being investigated through a global Phase 3 registrational program in patients with PDAC and metastatic RAS mutant non-small cell lung cancer (NSCLC).

Outside the U.S., RASONQUE is an investigational agent that has not been approved by any regulatory authority. RASONQUE received orphan medicinal product designation for pancreatic cancer and high-priority status under EMA’s Cancer Medicines Pathfinder project based on its potential to address a significant unmet need.

U.S. INDICATION

RASONQUE is indicated for the treatment of adult patients with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for multiagent systemic therapy.

U.S. IMPORTANT SAFETY INFORMATION

RASONQUE is associated with the following Warnings and Precautions: Dermatologic and Soft Tissue Toxicity, Stomatitis and Oral Disorders, Diarrhea, Gastrointestinal Perforation, Interstitial Lung Disease (ILD)/Pneumonitis, and Embryo-Fetal Toxicity.

U.S. WARNINGS AND PRECAUTIONS

Dermatologic and Soft Tissue Toxicity

RASONQUE can cause dermatologic toxicity, which may be severe. Clinical manifestations included, but were not limited to, rash, pruritus, paronychia, dry skin, and skin fissures. In clinical trials of patients with pancreatic adenocarcinoma, dermatologic toxicity occurred in 86% of patients treated with RASONQUE, of which 10% were Grade 3.

Monitor patients who develop dermatologic or soft tissue toxicities while receiving RASONQUE. Initiate prophylactic measures (e.g., topical corticosteroids, emollient creams, sunscreen, oral antibiotics) prior to the first dose of RASONQUE to reduce the risk of moderate to severe dermatologic reactions. Advise patients to limit sun exposure while taking RASONQUE. Initiate supportive measures (e.g., oral corticosteroids) as clinically indicated, and consider dermatologic consultation. Withhold, reduce the dose, or permanently discontinue RASONQUE based on severity.

Stomatitis and Oral Disorders

RASONQUE can cause stomatitis, including mouth ulcers and oral mucositis. In clinical trials of patients with pancreatic adenocarcinoma, stomatitis occurred in 57% of patients, of which 9% were Grade 3.

Monitor patients for signs and symptoms of stomatitis while receiving RASONQUE. Initiate a steroid-containing mouthwash for treatment of stomatitis and administer other topical treatments (e.g., chlorhexidine mouthwash, 2% lidocaine viscous) as clinically indicated. Withhold, reduce the dose, or permanently discontinue RASONQUE based on severity.

Diarrhea

RASONQUE can cause diarrhea. In clinical trials of patients with pancreatic adenocarcinoma, diarrhea occurred in 63% of patients, of which 6% were Grade 3.

If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Withhold, reduce the dose, or permanently discontinue RASONQUE based on severity.

Gastrointestinal Perforation

RASONQUE can cause gastrointestinal perforation. In clinical trials of patients with pancreatic adenocarcinoma, gastrointestinal perforation occurred in 0.9% of patients treated with RASONQUE, of which 0.5% were Grade 3, one event was Grade 4, and one event was fatal.

Monitor patients for gastrointestinal perforation. Withhold RASONQUE if gastrointestinal perforation is suspected. Reduce the dose or permanently discontinue RASONQUE if no other potential causes of gastrointestinal perforation are identified.

Interstitial Lung Disease (ILD)/Pneumonitis

RASONQUE can cause interstitial lung disease or pneumonitis. In clinical trials of patients with pancreatic adenocarcinoma, ILD/pneumonitis occurred in 2.4% of patients treated with RASONQUE, of which 0.9% were Grade 3, and one event was fatal.

Monitor patients for new or worsening pulmonary symptoms. Withhold RASONQUE if ILD/pneumonitis is suspected. Reduce the dose or permanently discontinue RASONQUE if no other potential causes of ILD/pneumonitis are identified.

Embryo-Fetal Toxicity

Based on findings in animals, RASONQUE can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with RASONQUE and for 1 week after the last dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with RASONQUE and for 1 week after the last dose.

ADVERSE REACTIONS

Serious adverse reactions occurred in 30% of patients treated with RASONQUE. Serious adverse reactions occurring in ≥ 2% of patients treated with RASONQUE were diarrhea (3.7%), pyrexia (3.3%), sepsis (2.9%), fatigue (2.1%), and hemorrhage (2.1%).

Adverse reactions leading to permanent discontinuation of RASONQUE occurred in 2.9% of patients, including two patients who discontinued due to rash (0.8%).

The most common (≥ 20%) adverse reactions in patients treated with RASONQUE were rash, diarrhea, stomatitis, nausea, fatigue, vomiting, abdominal pain, edema, decreased appetite, and hemorrhage.

DRUG INTERACTIONS

Strong CYP3A Inhibitors with P-gp Inhibition: Avoid concomitant use.
Strong CYP3A Inhibitors without P-gp Inhibition: Reduce RASONQUE dosage.
Moderate CYP3A Inhibitors with or without P-gp Inhibition: Reduce RASONQUE dosage.
P-gp Inhibitors: Reduce RASONQUE dosage.
Cyclosporine A: Avoid concomitant use.
Strong CYP3A Inducers: Avoid concomitant use. Increase RASONQUE dosage if concomitant use cannot be avoided.
Moderate CYP3A Inducers: Increase RASONQUE dosage.
P-gp Substrates: Take at least 4 hours apart from RASONQUE.



PROPHYLACTIC MEASURES

When initiating RASONQUE and throughout treatment, prophylactic and concomitant medications are recommended to reduce the risk of dermatologic reactions:

administer a topical corticosteroid (applied to the face and chest) and emollient creams
advise patients to limit sun exposure and use broad-spectrum sunscreen (SPF 30 or higher)
consider prophylactic oral antibiotics (e.g., doxycycline or minocycline)

(Press release, Revolution Medicines, OCT 15, 2026, View Source [SID1234673494])

On October 15, 2026 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on developing innovative antibody-based therapeutics for the treatment of cancer, reported that the first patient has been dosed in the Phase 1 study of MGC030, a first-in-class antibody-drug conjugate (ADC) directed against an undisclosed target, in patients with advanced solid tumors.

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"MGC030 demonstrated encouraging antitumor activity in multiple preclinical models, and we look forward to advancing this product candidate through the Phase 1 study. We expect that this trial will deepen our understanding of how the molecule may help address the unmet needs of patients with selected advanced solid tumors," said Ezio Bonvini, M.D., Senior Vice President and Chief Scientific Officer of MacroGenics. "The dosing of a first patient with MGC030 builds upon the strong early-stage clinical momentum we have sustained across our innovative ADC pipeline, as this molecule is now our third ADC in active clinical development."

The Phase 1, open-label, multi-center, dose-escalation, dose-optimization and cohort-expansion study of MGC030 is designed to characterize the safety, tolerability, pharmacokinetics (PK), immunogenicity and preliminary antitumor activity of MGC030, as well as explore biomarkers that may be associated with these endpoints. Participants with relapsed or refractory solid tumors that are unresectable, locally advanced, or metastatic, including urothelial, colorectal, gastric or gastroesophageal junction, esophageal, and pancreatic cancers, will be enrolled. The dose escalation portion of the study will enroll up to 54 participants with the potential to further expand enrollment at selected dose levels.

About MGC030

MGC030 is a clinical-stage ADC that targets an undisclosed antigen expressed across several solid tumors. There are currently no approved therapeutics directed against this target. MGC030 incorporates Synaffix’s proprietary ADC technology and the SYNtecan E topoisomerase I inhibitor-based linker-payload, which consists of a cleavable, exatecan-based cytotoxic payload conjugated at a drug-to-antibody ratio (DAR) of 4.

MGC030 has demonstrated potent antitumor activity in multiple in vivo models and an encouraging safety and tolerability profile in cyno tox studies. The ongoing Phase 1 study of MGC030 is an open-label clinical trial evaluating the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary antitumor activity of MGC030 in patients with advanced solid tumors. Additional information about the study is available at www.clinicaltrials.gov using the identifier NCT07822399.

(Press release, MacroGenics, OCT 15, 2026, View Source [SID1234673493])

On October 15, 2026 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immuno-oncology technologies addressing difficult to treat cancers, reported that its collaboration partner, PeriNess Ltd. ("PeriNess"), has informed the Company that the first patient has been dosed in an investigator-initiated clinical study evaluating systemic recombinant human DNase in combination with anti-CD19 CAR T-cell therapy in patients with large B-cell lymphoma ("LBCL") at the Tel Aviv Sourasky University Medical Center ("Ichilov Center") in Israel.

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The milestone marks the first clinical evaluation of Xenetic’s systemic DNase technology as an adjunct to CAR T-cell therapy. The study, sponsored by the Ichilov Center, was approved by the Israeli Ministry of Health and the applicable Institutional Review Board in June 2026 and is designed to evaluate whether degradation of cell-free chromatin and neutrophil extracellular traps (NETs) can improve CAR T-cell expansion, persistence and clinical response.

The study is enrolling 12 patients with LBCL who have stable or progressive disease prior to lymphodepletion therapy, a population with a historically poor prognosis and one-year progression-free survival of approximately 20% to 30%. Patients will receive a commercial anti-CD19 CAR T-cell product, tisagenlecleucel, axicabtagene ciloleucel or lisocabtagene maraleucel, in combination with DNase I administered as a 50 mL intravenous infusion at 1.0 mg/kg on Days 0, 3, 6, 10 and 15 following CAR T-cell infusion.

Clinical efficacy will be evaluated by objective response rate (ORR) at one and three months following CAR T-cell infusion, duration of response (DOR), disease control rate (DCR) and overall survival (OS) at 12 months. The study also includes a translational component designed to assess biomarker response, including circulating cell-free DNA and NET markers, as well as anti-CD19 CAR T-cell expansion and persistence.

Dr. Ron Ram, Professor of Medicine and Head of the Bone Marrow Transplantation Unit at the Ichilov Center, is the principal investigator of the study. "Advancing this approach into patients provides an important opportunity to evaluate this biological hypothesis in a clinical setting," said Dr. Ram. "Accumulation of cell-free chromatin and NETs within the tumor microenvironment may contribute to CAR T-cell dysfunction through impaired expansion, exhaustion and immunosuppression. By targeting these mechanisms with DNase I, we aim to determine whether we can create a more favorable environment for CAR T cells to expand, persist and deliver improved clinical responses, particularly in patients entering treatment with stable or progressive disease."

"The initiation of this study represents a significant step forward for Xenetic as our systemic DNase technology enters clinical evaluation alongside what we believe to be one of the most important advances in cancer immunotherapy," said James Parslow, Interim Chief Executive Officer and Chief Financial Officer of Xenetic. "Our preclinical work has demonstrated the potential for DNase to enhance CAR T-cell expansion, persistence and functionality, improve tumor control, delay relapse and prolong survival across multiple leukemia and lymphoma models. We are pleased to be working with PeriNess and the experienced team at the Ichilov Center and look forward to the continued advancement of the study."

(Press release, Xenetic Biosciences, OCT 15, 2026, View Source [SID1234673491])