On November 2, 2026 Krystal Biotech, Inc. (the "Company" or "Krystal") (NASDAQ: KRYS) reported financial results for the third quarter ended September 30, 2026 and provided a business update.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Continued growth in VYJUVEK revenue is supporting profitability and funding the advancement of our pipeline," said Krish S. Krishnan, Chairman and Chief Executive Officer of Krystal Biotech. "Our financial strength allows us to invest fully in these programs and our global commercial expansion while returning capital to shareholders. The $400 million share repurchase program, under which we expect to repurchase shares opportunistically over the next two years, reflects our confidence in Krystal’s long-term growth prospects and our commitment to disciplined capital allocation."

VYJUVEK (beremagene geperpavec-svdt, or B-VEC)
for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

The Company recorded $127.3 million in global VYJUVEK net product revenue for the third quarter of 2026, an increase of 30% compared to the prior year third quarter. Gross margin for the quarter was 94%.

Patient demand in the United States continues to grow with over 760 reimbursement approvals secured for VYJUVEK nationwide. Steady expansion of the U.S. prescriber base is supporting continued adoption across the country.

The Company is also expanding its reach across its initial launch markets of Germany, France, and Japan, converting high physician interest and engagement into patient starts and broadening the prescriber base to bring VYJUVEK treatment closer to patients.

Pricing negotiations are now complete in both Germany and Italy, and the Company expects to launch VYJUVEK in Italy in early 2027. Pricing negotiations are ongoing in multiple European countries and the United Kingdom, with decisions expected in Spain later this year, and in France no earlier than 2027. The Company has also submitted a marketing authorization application for VYJUVEK in Australia and expects to submit an application for marketing authorization in Switzerland before year end.

Dermatology

KB111 for the treatment of Hailey-Hailey disease (HHD)

Enrollment is complete and dosing is ongoing in HALITE-1, the Company’s open-label, single-arm study evaluating the safety of KB111, administered once weekly for 12 weeks, in patients with HHD. HHD is a genetic disease characterized by painful rash and blistering in skin folds. In addition to pain, patients with HHD reporting debilitating symptoms of itch, burning, infections, and body odor, as well as severe, negative impacts on quality of life and psychological distress. The prevalence of HHD is not well characterized and is most commonly estimated at roughly 1 per 50,000, although underreporting is possible.

A total of five patients have been enrolled in HALITE-1. The Company expects to report interim study results before the end of the year. Details of the study can be found at www.clinicaltrials.gov under NCT identifier: NCT07717346.

The Company has also completed development of its HHD-specific severity scale for the clinical evaluation of KB111. The Company expects to meet with the FDA in the first quarter of 2027, following completion of scale validation, to discuss study results, the scale, and study designs to enable a registrational study start in 2027.

KB113 for the treatment of Gorlin syndrome (GS)

In October, the Company announced a positive clinical update supporting initial proof of concept for KB113 in the treatment of basal cell carcinoma (BCC) in patients with GS. GS 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. Prevalence data for GS is limited but available data suggest the number of patients with GS in the United States could exceed 10,000.

In the Company’s three patient pilot, multicenter, single-arm, open-label study, more than 80% of assessed BCC lesions showed investigator-assessed clinical regression. Regression was observed in both injected and non-injected lesions, consistent with an abscopal effect. Histologic assessment also confirmed complete clearance of an injected BCC lesion in each patient. KB113 was well tolerated, with all reported adverse events mild or moderate in severity, transient, and resolving during the study.

Building on these positive initial results, the Company has initiated discussions with the FDA regarding the advanced clinical development of KB113 for the treatment of BCCs in patients with GS. The Company will provide an update on its clinical development plans following alignment with the FDA.

Ophthalmology

KB803 for the treatment and prevention of corneal abrasions in DEB patients

The Company has enrolled three additional patients in its registrational IOLITE study, including one child aged one year. The additional enrollment replaces two patients who withdrew for reasons unrelated to safety and provides supporting evidence for the potential treatment of corneal abrasions in DEB patients from birth. The Company now expects the last patient visit in February 2027 with top-line results reported shortly thereafter. The primary efficacy endpoint is the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT07016750.

KB801 for the treatment of neurotrophic keratitis (NK)

Enrollment is ongoing in EMERALD-1, the Company’s registrational, 1:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for the treatment of NK. The Company has activated 22 sites across the United States and is close to activating sites in France, Spain and the United Kingdom. Patient enrollment has progressed slower than initially anticipated, reflecting the study’s narrowly defined eligibility criteria and the challenges of enrolling patients in a placebo-controlled trial in an indication with an available approved therapy. The Company now expects to complete enrollment of approximately 60 patients in EMERALD-1 and report top-line results in 2027. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT06999733.

Respiratory

KB407 for the treatment of cystic fibrosis (CF)

Three patients are currently being dosed in the Company’s ongoing open-label, single-arm study evaluating the safety of repeat-dose KB407 administration over 24 weeks in patients with CF who are ineligible for, do not tolerate, or do not benefit from modulator therapy. The Company expects to enroll and dose three additional patients before year end, with interim study results now anticipated in the first half of 2027. Earlier this year, the Company announced positive interim clinical findings demonstrating successful delivery and expression of wild-type CFTR protein in the lungs of patients treated with KB407. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT05504837.

In the third quarter, the Company was selected for the FDA’s Complex Innovative Trial Design (CID) Meeting Program, advancing ongoing discussions regarding the Company’s innovative registrational study design. The CID Meeting Program is intended to facilitate and advance the use of complex and novel clinical trial designs and provides the opportunity for increased interaction with the FDA. The Company will share the design and associated statistical analysis of the registrational study following alignment with the FDA and is presently on track to initiate the registrational study in 2027.

KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD) lung disease

The Company continues to enroll in repeat-dose Cohort 2B of SERPENTINE-1, the Company’s open-label dose escalation study evaluating KB408 in adult patients with AATD with a Pi*ZZ or a Pi*ZNull genotype. Cohort 2B is designed to evaluate the safety and tolerability of repeat KB408 dosing at the same dose level that was previously shown to safely deliver SERPINA1 to the lungs of AATD patients after a single dose. Details of the study can be found at www.clinicaltrials.gov under NCT identifier: NCT06049082. The Company expects to report interim study results in 2027.

Oncology

Inhaled KB707 for the treatment of non-small cell lung cancer (NSCLC)

KYANITE-1 is an ongoing Phase 1/2 open-label, multi-center, dose escalation and expansion study evaluating inhaled KB707, as monotherapy or in combination with standard of care therapies, in patients with locally advanced or metastatic solid tumors involving the lungs.

Interim clinical results from the KYANITE-1 dose expansion cohort evaluating inhaled KB707 in combination with pembrolizumab were presented at the American Society for Clinical Oncology 2026 Annual Meeting in May and demonstrated antitumor activity in patients with advanced NSCLC, with an objective response rate (ORR) of 31% and a disease control rate (DCR) of 75%. Multiple patients from this cohort remain on treatment and in follow-up with sustained disease control.

The Company is currently enrolling patients with advanced NSCLC in a global KYANITE-1 dose expansion cohort evaluating inhaled KB707 in combination with chemotherapy. An initial dose-optimization phase evaluated the safety and tolerability of the combination regimen in the first 10 patients and informed the selection of the chemotherapy dose for subsequent enrollment. Sites have also been activated in France and Spain. The Company expects to enroll approximately 70 patients to be dosed at the selected combination regimen by mid-2027.

Details of the KYANITE-1 study can be found at www.clinicaltrials.gov under NCT identifier: NCT06228326.

Aesthetics

KB304 for the treatment of wrinkles of the décolleté

Jeune Aesthetics, Inc., a wholly owned subsidiary of the Company, expects to initiate a Phase 2 study of its lead program KB304 in 2027.

Share Repurchase Program

The Company also announced today that its Board of Directors has authorized a share repurchase program under which Krystal may repurchase up to $400 million of its outstanding common stock. Repurchases may be made from time to time through open-market purchases, privately negotiated transactions, or by other means permitted under applicable securities laws, including through purchases compliant with Rule 10b-18 under the Securities Exchange Act of 1934, as amended (the "Exchange Act") and trading plans adopted under Rule 10b5-1 under the Exchange Act. The timing and amount of any repurchases will depend on a number of factors, including the market price of Krystal’s common stock, market and business conditions, the Company’s liquidity and capital requirements, clinical development and commercialization plans, alternative uses of capital, and legal and regulatory requirements. The program has no expiration date, does not obligate Krystal to repurchase any amount of common stock, and the program may be modified, suspended or discontinued at any time at the Company’s discretion.

Financial Results for the Quarter Ended September 30, 2026:

Cash, cash equivalents and investments totaled $1.2 billion as of September 30, 2026
Product revenue, net totaled $127.3 million and $97.8 million for the three months ended September 30, 2026 and September 30, 2025, respectively.
Cost of goods sold totaled $7.6 million and $4.3 million for the three months ended September 30, 2026 and September 30, 2025, respectively.
Research and development expenses for the three months ended September 30, 2026 were $14.9 million, inclusive of $2.6 million of stock-based compensation, compared to $14.6 million, inclusive of stock-based compensation of $2.6 million, for the three months ended September 30, 2025.
Selling, general, and administrative expenses for the three months ended September 30, 2026 were $33.7 million, inclusive of stock-based compensation of $11.3 million, compared to $37.6 million, inclusive of stock-based compensation of $10.6 million, for the three months ended September 30, 2025.
Net income for the three months ended September 30, 2026 was $63.1 million, or $2.13 per common share (basic) and $2.05 per common share (diluted). Net income for the three months ended September 30, 2025 was $79.4 million, or $2.74 per common share (basic) and $2.66 per common share (diluted).
For additional information on the Company’s financial results for the three months ended September 30, 2026, please refer to the Form 10-Q filed with the SEC.
Financial Results for the Nine Months Ended September 30, 2026:

Product revenue, net totaled $362.9 million and $282.0 million for the nine months ended September 30, 2026 and September 30, 2025, respectively.
Cost of goods sold totaled $20.3 million and $16.5 million for the nine months ended September 30, 2026 and September 30, 2025, respectively.
Research and development expenses for the nine months ended September 30, 2026 were $44.7 million, inclusive of $7.3 million of stock-based compensation, compared to $43.3 million, inclusive of stock-based compensation of $7.7 million, for the nine months ended September 30, 2025.
Selling, general, and administrative expenses for the nine months ended September 30, 2026 were $114.6 million, inclusive of stock-based compensation of $34.4 million, compared to $105.3 million, inclusive of stock-based compensation of $33.1 million, for the nine months ended September 30, 2025.
Net income for the nine months ended September 30, 2026 was $173.8 million, or $5.90 per common share (basic) and $5.66 per common share (diluted). Net income for the nine months ended September 30, 2025 was $153.4 million, or $5.31 per common share (basic) and $5.14 per common share (diluted).
For additional information on the Company’s financial results for the nine months ended September 30, 2026, please refer to the Form 10-Q filed with the SEC.
Financial Guidance

($ in millions) FY 2026 Guidance
Non-GAAP Research and Development ("R&D") and Selling, General and Administrative ("SG&A") expense(1) $155.0 – $165.0

(1) Refer to Non-GAAP Financial Measures section below for additional information. Non-GAAP combined R&D and SG&A expense guidance does not include stock-based compensation as we are currently unable to confidently estimate Full Year 2026 stock-based compensation expense. As such, we have not provided a reconciliation from forecasted non-GAAP to forecasted GAAP combined R&D and SG&A Expense in the above. This could materially affect the calculation of forward-looking GAAP combined R&D and SG&A Expense as it is inherently uncertain.

Conference Call

The Company will host a conference call and webcast on November 2, 2026, at 8:30 am ET.

Investors and the general public can access the live webcast at: View Source

For those unable to listen to the live conference call, a replay will be available for 30 days on the Investors section of the Company’s website at www.krystalbio.com.

About VYJUVEK

VYJUVEK is a non-invasive, topical, redosable genetic medicine designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. VYJUVEK was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. VYJUVEK is approved in the United States, Europe, and Japan.

U.S. INDICATION

VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

IMPORTANT SAFETY INFORMATION

Adverse Reactions

The most common adverse drug reactions (incidence >5%) were itching, chills, redness, rash, cough, and runny nose. These are not all the possible side effects with VYJUVEK. Call your healthcare provider for medical advice about side effects.

To report SUSPECTED ADVERSE REACTIONS, contact Krystal Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088 or View Source

Contraindications

None.

Warnings and Precautions

VYJUVEK gel may be applied by a healthcare provider, a caregiver, or the patient.

After treatment, patients and caregivers should be careful not to touch treated wounds and dressings until the next dressing change.

Wash hands and wear protective gloves when changing wound dressings. Disinfect bandages from the first dressing change with a virucidal agent, and dispose of the disinfected bandages in a separate sealed plastic bag in household waste. Dispose of the subsequent used dressings in a sealed plastic bag in household waste.

Patients should avoid touching or scratching wound sites or wound dressings.

In the event of an accidental exposure flush with clean water for at least 15 minutes.

For more information, see full U.S. Prescribing Information.

(Press release, Krystal Biotech, NOV 2, 2026, View Source [SID1234674039])

On November 2, 2026 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology innovation, reported the peer-reviewed publication of results from the investigator-sponsored, multicenter Phase 2 UNICORN trial evaluating short-course preoperative, or neoadjuvant, botensilimab (BOT), Agenus’ multifunctional, Fc-enhanced anti-CTLA-4 antibody, alone or in combination with balstilimab (BAL), Agenus’ anti-PD-1 antibody, in patients with resectable, locally advanced colon cancer. The manuscript, titled "Preoperative botensilimab with or without balstilimab for patients with resectable, locally advanced pMMR or dMMR colon cancer: the UNICORN trial by GONO," was published in Nature Medicine and is available at DOI 10.1038/s41591-026-04710-1.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The UNICORN trial enrolled 56 patients in four non-randomized cohorts of 14, evaluating BOT alone or BOT+BAL in mismatch repair proficient/microsatellite stable (pMMR/MSS) and mismatch repair deficient/microsatellite instability-high (dMMR/MSI-H) colon cancer. The study assessed pathologic response, event-free survival, circulating tumor DNA (ctDNA) and biological changes in tissue samples before and after treatment.

UNICORN clinical findings

Among the 14 patients with pMMR/MSS colon cancer treated with BOT+BAL, 36% achieved a major pathologic response (MPR), defined as 10% or less viable tumor remaining at surgery, and 29% achieved a pathologic complete response (pCR), meaning no viable tumor remained in the primary tumor or lymph nodes. Overall, 71% of patients had a pathologic response, defined as at least 50% tumor reduction.

For historical context, first-generation CTLA-4/PD-1 combination immunotherapy has demonstrated an MPR rate of approximately 20% while neoadjuvant chemotherapy has demonstrated an MPR rate of 8% in previously reported studies of pMMR colon canceri,ii.

In the separate pMMR/MSS BOT monotherapy cohort, 43% experienced partial tumor regression, but no patients achieved an MPR or pCR. The findings suggest BOT can initiate anti-tumor activity in a subset of pMMR tumors, while the deeper major and complete responses emerged with the addition of BAL and PD-1 blockade.

In dMMR/MSI-H disease, all 14 patients (100%) in the BOT+BAL treatment cohort achieved an MPR, including 13 (93%) with a pCR, compared with 36% and 29%, respectively, with BOT alone.

The responses with BOT+BAL were durable. At the February 9, 2026, data cutoff, no patients treated with BOT+BAL had their cancer return, with median follow-up of 19.2 months in pMMR/MSS disease and 16.7 months in dMMR/MSI-H disease. Twelve-month event-free survival was 100% in both combination cohorts.

Planned surgical timing was preserved in 98% of patients, and all 56 patients underwent surgical resection. One patient experienced a 10-day surgical delay associated with Grade 3 hyperthyroidism. Grade 3 or 4 immune-related adverse events occurred in 2% of patients.

Translational Findings

ctDNA clearance tracked with response: All five evaluable pMMR/MSS patients who achieved an MPR cleared ctDNA before surgery. No patients who achieved ctDNA clearance had their cancer return at the data cutoff.
Biological findings aligned with BOT’s Fc-enhanced design: Paired tumor samples collected before treatment and at surgery identified immune changes associated with response that were consistent with BOT’s Fc-enhanced mechanism and its ability to remodel the tumor immune environment, including reductions in immunosuppressive regulatory T-cell signatures and increased immune-cell infiltration.
The UNICORN publication follows the recent peer-reviewed publication of the independent Phase 2 NEST study evaluating neoadjuvant BOT+BAL. With NEST and UNICORN now both peer reviewed, the two studies provide complementary and clinical evidence across single-center and multicenter settings, with consistent deep pathologic responses, ctDNA clearance, preserved surgical feasibility and maturing longer term follow-up. While the studies differed in design, the consistency of findings across independently conducted trials strengthens the clinical foundation for evaluating BOT+BAL earlier in the course of colon cancer.

This is particularly relevant in MSS colon cancer, which represents approximately 85% of early-stage colorectal cancers and has historically derived limited benefit from conventional immunotherapyiii,iv. In the curative-intent setting, treatment before surgery creates an opportunity to activate the immune system while the primary tumor and surrounding immune environment remain intact, with the goal of eliminating residual disease and preventing recurrence so that more patients may ultimately be cured.

"UNICORN delivers compelling, independent evidence that BOT+BAL can produce deep responses in MSS colon cancer, a disease historically resistant to conventional immunotherapy," said Steven O’Day, M.D., Chief Medical Officer of Agenus. "With no recurrences observed to date, these findings reinforce the promise of treating earlier. Together with NEST, they provide a strong foundation for our planned Phase 3 ROBBIN trial and our ambition to change how MSS colon cancer is treated in the curative-intent setting."

Agenus is advancing ROBBIN, the planned global randomized Phase 3 trial evaluating neoadjuvant BOT+BAL followed by standard of care versus standard of care alone in previously untreated patients with high-risk Stage II or Stage III MSS/pMMR colon cancer. Event-free survival is the planned primary endpoint.

"UNICORN gave us a rare opportunity to look beyond whether a tumor responded and examine what was changing biologically during a very short period before surgery," said Filippo Ghelardi, M.D., first author and Medical Oncology Fellow of Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. "The alignment we observed between deep pathologic responses, clearance of circulating tumor DNA and changes within the tumor immune environment provides multiple signals of treatment activity. For MSS colon cancer, where immunotherapy has historically had limited impact, these findings give us important new insight into what may be possible when treatment is introduced earlier in the course of disease."

"For many years, MSS colon cancer has largely been considered resistant to immunotherapy. The UNICORN study challenges that assumption in the curative-intent setting," said Filippo Pietrantonio, M.D., senior author, study principal investigator and Head of the Gastrointestinal Oncology Unit, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. "The purpose of moving treatment earlier is not simply to shrink the tumor before surgery. It is to prevent the disease from returning and ultimately increase the number of patients who may be cured. Seeing deep responses after only a short course of treatment, with patients able to proceed to planned surgery and no recurrences observed to date in the combination cohorts, provides a strong rationale to test that objective in a larger randomized trial."

About the UNICORN Study
UNICORN (NCT05845450) is an ongoing multicenter, non-randomized, open-label Phase 2 window-of-opportunity platform trial sponsored by Fondazione GONO. The study evaluates short-course preoperative therapies in molecularly selected patients with resectable colorectal cancer.

The analysis reported in Nature Medicine evaluated Cohorts 4 through 7, comprising 56 patients with pMMR/MSS or dMMR/MSI-H colon cancer treated with BOT alone or BOT+BAL prior to curative-intent surgery. Patients receiving BOT monotherapy received botensilimab 1 mg/kg on Day 1. Patients receiving BOT+BAL received botensilimab 1 mg/kg on Day 1 and balstilimab 3 mg/kg on Days 1 and 15. Surgery was planned for Day 35 ± 5 days. The primary endpoint was centrally assessed major pathologic response, defined as 10% or less residual viable tumor. Secondary and exploratory analyses included safety, quality of life, longitudinal ctDNA, spatial transcriptomics, peripheral immune-cell dynamics, genomic correlates and microbiome analyses. The study was supported by GONO and other research funding sources. Agenus supplied BOT and BAL.

(Press release, Agenus, NOV 2, 2026, View Source [SID1234674038])

On November 2, 2026 Recursion (Nasdaq: RXRX), a leading clinical-stage company pioneering AI-enabled drug discovery and development to decode biology and radically improve lives, reported financial results for the third quarter ended September 30, 2026, together with business updates highlighting continued clinical, pipeline and platform execution.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Recursion will host a webinar with earnings updates on REC-4881 data, REC-1245 data, as well as REC-4881 KOL and patient panels, presented live from CGA-IGC on Tuesday, November 3, 2026 at 8:00 am ET / 6:00 am MT / 1:00 pm GMT from Recursion’s X, LinkedIn, and YouTube accounts giving analysts, investors, and the public the opportunity to ask questions of the Company by submitting questions here: View Source

"To pioneer a new blueprint for making medicines with AI, we believe you have to tie together three core capabilities in one engine: uncover novel biology, design differentiated molecules against challenging targets, and generate clinical evidence that matters for patients," said Najat Khan, Ph.D., President and Chief Executive Officer of Recursion. "The new TUPELO data are important clinical evidence, with REC-4881 showing deep polyp burden reductions across the upper and lower GI tract, including larger, higher-risk polyps, and reductions that persist after treatment stops. REC-1245 is an encouraging early example of that engine coming together within a single program, from biological insight surfaced by our platform to molecule design and now early clinical signals in heavily pretreated patients."

"Beyond our own pipeline, Sanofi has optioned a potential first-in-class molecule against a challenging dual target, while Genentech has advanced an unexplored neuroscience target from our maps. These are different programs, but they are outputs of the same AI-native engine, providing evidence of repeatability. We will keep concentrating capital where the evidence is strongest and turning innovation into impact for patients who are waiting."

Internal Pipeline Highlights

Positive Phase 2 results from ongoing TUPELO trial of REC-4881 in FAP highlight activity across GI tract and on larger polyps

FAP is a genetically defined driver of GI cancer, affecting >50,000 patients in the US and EU5 who face major surgeries and life-long surveillance with no approved therapies, representing a potential $15 billion opportunity, based on Company estimates. Among the approximately 35,000 post-colectomy patients, upper GI disease represents the highest unmet need in FAP, due to risks of bleeding and perforation from polyp removal, high complication and morbidity rates from surgery, and higher cancer risk.

REC-4881, a potential first-in-class oral therapy granted FDA Orphan Drug and Fast Track designations, is designed to address both Wnt/β-catenin initiation and MAPK evolution pathways. The opportunity was identified through unbiased phenotypic screening using Recursion’s proprietary AI foundation models, which revealed MEK1/2 inhibition as a potential way to reverse cellular phenotypes associated with APC loss, a previously unappreciated synthetic rescue relationship.

On November 2, Recursion announced further positive data from its ongoing Phase 1b/2 TUPELO trial of REC-4881, an investigational, oral MEK1/2 inhibitor in development for FAP. These data were presented by Lisa Boardman, M.D., Professor of Medicine at Mayo Clinic, in a presidential plenary session at The Collaborative Group of the Americas on Inherited Gastrointestinal Cancer (CGA-IGC) Annual Meeting in Denver, Colorado.

•Deep, durable reductions in total polyp burden, strengthened in the expanded adult FAP population (18+): After 12 weeks of REC-4881 (4 mg QD), 87% of patients (13 of 15) had a reduction in total polyp burden, with a 47% median reduction; approximately 1 in 3 achieved reductions of ≥65%. Twelve weeks after stopping treatment, 85% of evaluable patients (11 of 13) remained below baseline.
•Activity across the upper and lower GI tract, including near-clearance: After 12 weeks of treatment, median polyp burden reductions were 55% in the upper GI and 50% in the lower GI, supporting REC-4881’s potential as a single oral therapy to address FAP across the GI tract.
•Upper GI, the area of greatest unmet need in FAP (results first reported Oct. 15): 92% of patients (12 of 13) had reductions after 12 weeks of treatment, with two patients achieving ≥98% reductions; half (6 of 12) improved by at least one Spigelman stage; and 92% (11 of 12) remained below baseline 12 weeks after stopping treatment.
•New lower GI results highlight breadth of activity: 9 of 11 patients (82%) with measurable lower GI polyp burden at baseline had reductions after 12 weeks of treatment, with a 50% median reduction and 1 in 3 achieving ≥65%; 70% of evaluable patients remained below baseline 12 weeks after stopping treatment.
•New analysis shows substantial reductions in larger, higher-risk polyps: 6 of 8 patients with polyps >5 mm at baseline had fewer of these polyps after 12 weeks of treatment. Four achieved reductions of ≥80%, including two whose counts fell from more than 90 polyps to one. Six of eight remained below their baseline count 12 weeks after stopping treatment.
•No new safety signals observed: Favorable chronic dosing safety profile. The highest-grade treatment-related adverse event was Grade 1 or 2 in 21 of 26 patients (81%) and Grade 3 in four patients (15%); one patient had no TRAEs. No Grade 4 or 5 TRAEs, and no additional discontinuations since the previous data cutoff.

The Company anticipates initiating a registrational trial in the second half of 2027, pending ongoing FDA discussions, and will provide additional details later this year.

Early data from the Phase 1 dose-escalation portion of DAHLIA, our ongoing open-label Phase 1/2 study of REC-1245, show the following (data cutoff: August 21, 2026):

REC-1245 is a potential first-in-class oral molecular glue designed to exploit a synthetic-lethal vulnerability in cancers by degrading RBM39, a splicing factor that regulates genes involved in DNA repair and stress response. The Recursion platform contributed each of the three core elements of the program: the target, the molecule and the initial hypotheses on which patients are most likely to benefit. The target emerged from Recursion’s platform, which found that degrading RBM39 mimics the loss of CDK12, while sparing CDK13-associated liabilities. REC-1245 was identified within 18 months after screening only 204 compounds using Recursion’s design platform. The same platform-derived biology pointed to tumors intrinsically primed by replication stress and reliance on accurate splicing, including hard-to-treat cancers such as endometrial, hepatocellular and ovarian cancers and transcriptionally driven tumors.

•Three partial responses per RECIST 1.1 criteria (2 confirmed, 1 unconfirmed) in heavily pretreated patients with endometrial cancer and hepatocellular carcinoma, all at doses of 45 mg once daily (QD) or higher, with a disease control rate of 91.7% at those doses (n=12).
•Recursion has selected 45 mg QD for exploratory expansion cohorts, including endometrial cancer, where limited second-line treatments are approved; at the 45 mg QD dose (n=10), no dose-limiting toxicities or treatment-related serious adverse events were observed, and treatment-related adverse events were predominantly Grade 1 or 2. Most frequent TRAEs were Grade 1 or Grade 2 fatigue and anemia (30% each).
•Preliminary dose expansion data in endometrial cancer are expected in the second half of 2027. Dose escalation will also continue in other solid tumor indications.

Other Portfolio Updates: REC-7735 is an orally bioavailable, precision-designed small molecule inhibitor of PI3Kα H1047R generated using the Recursion OS. In the second half of 2026, Recursion initiated the Phase 1/2 ZINNIA clinical study for patients with select PIK3CA H1047R-mutant solid tumors.

As announced in October 2026, given the promising data in REC-4881 and REC-1245, Recursion plans to continue to focus resources on its high-impact partnerships and internal programs including REC-4881 (MEK1/2 – familial adenomatous polyposis), REC-1245 (RBM39 – endometrial cancer and other solid tumors), REC-4539 (LSD1 – solid tumors and hematology), REC-7735 (PI3Kα H1047R – solid tumors) and REC-102 (ENPP1 – hypophosphatasia). Recursion intends to pursue partnership opportunities to advance REC-617 (CDK7) and REC-3565 (MALT1).

Partnered Discovery Highlights Recursion’s 9th Milestone Achieved in Past Two Years

Sanofi options potential first-in-class immunology development candidate

Sanofi has optioned the first development candidate from the two companies’ multi-program research collaboration. The candidate has now been added to Sanofi’s development pipeline and is expected to enter Phase 1 clinical trials in 2027, with Sanofi funding and leading further development and commercialization to advance the program toward patients.

The option triggered a $10 million milestone payment to Recursion, bringing total upfront and milestone payments achieved from Sanofi under the collaboration to $144 million; Recursion is eligible for more than $300 million in additional milestones under the program, with double digit royalties on net product sales, if approved.

The potential first-in-class oral small molecule was designed to achieve a differentiated profile against a challenging dual target implicated in inflammatory disease. Optimizing for this target pair required navigating complex and competing chemistry constraints to achieve the desired balance of drug-like properties. The candidate was delivered by the combination of Recursion’s integrated AI-enabled drug design platform and small-molecule chemistry capabilities with Sanofi’s deep expertise in drug discovery and immunology. Recursion applied multiple computational design approaches across iterative cycles of prediction, chemical design, synthesis, and experimental validation to rapidly explore and optimize a complex chemical space.

With the first development candidate optioned, the collaboration has now achieved six progress-based milestones across five programs. Additional programs across the collaboration continue to advance. Over the next 12 months, other programs in the joint portfolio have the potential to reach development candidate and other progress-based milestones.

To learn more about this milestone, read the press release.

Genentech advanced first neuroscience target into early discovery program

In 3Q26, Genentech exercised the first Validated Target Option under the companies’ neuroscience collaboration, advancing a previously unexplored neuroscience target.

The milestone provides additional early evidence that Recursion’s AI-native platform can both discover and play a key role experimentally validating unexplored therapeutic targets in neuroscience, one of medicine’s most challenging therapeutic areas, where decades of research have largely focused on a limited number of well-studied targets. Predictions generated from the first whole-genome CRISPR knockout map, which was built by Recursion in partnership with Roche and Genentech from a subset of over 1 trillion internally manufactured iPSC-derived neuronal cells, were rigorously jointly validated with Genentech. Only targets that consistently demonstrated compelling evidence across pathway, functional, and disease validation progress into a validation package.

Next steps will include advancing the target through small molecule design, hit generation, and validation using Recursion’s AI-native chemistry platform. More broadly, the neuronal and microglial maps of biology remain reusable assets capable of being collaboratively utilized with biological, genetics, and computational expertise to generate and experimentally validate additional therapeutic hypotheses. To date, Recursion has achieved $216 million in upfront and milestone payments from the Roche and Genentech collaboration. The collaboration includes up to 40 potential small molecule discovery programs, each carrying the potential for more than $300 million in development, commercialization, and net sales milestones as well as tiered royalties up to high single digits per small molecule program for Recursion.

Potential upcoming milestones across partnered discovery:
•Potential for additional differentiated AI-enabled oral molecules to reach development candidate and late-stage discovery milestones with Sanofi over the next 12 months
•Translating AI-driven insights from maps of biology into new potentially novel targets from reusable high-dimensional data/maps with Roche and Genentech
•Using Recursion’s chemistry platform to design potential first-in-class molecules for the collaboration’s first neuroscience target with Genentech

Fred Hassan appointed Chair of the Board
Recursion announced that its Board of Directors has appointed Fred Hassan as Chair, effective October 31, 2026.

Mr. Hassan brings more than four decades of pharmaceutical leadership spanning company transformation, drug development, commercialization, investing and Board service. As Chairman and Chief Executive Officer of Schering-Plough, he led a transformation that restored sales growth and strong cash flow while expanding the company’s Phase 3 pipeline from three new molecular entities to eight. He previously served as Chairman and Chief Executive Officer of Pharmacia.

Mr. Hassan also brings extensive perspective from across the biotechnology and investment ecosystem. He serves as Lead Independent Director of BridgeBio Pharma, where his Board tenure has spanned the FDA approval and U.S. launch of Attruby, and as Executive Chair of Norstella, the pharmaceutical intelligence company behind Citeline, MMIT and Evaluate. His prior Board roles include Amgen and Prometheus Biosciences. He is also a Director at Warburg Pincus, where he previously served as Partner and Managing Director.

Third Quarter 2026 Financial Results

•Cash Position: Cash, cash equivalents and restricted cash were $561.5 million as of September 30, 2026. Including net proceeds of approximately $187.2 million from the Company’s underwritten offering which completed on October 16, 2026, pro forma cash, cash equivalents and restricted cash were approximately $748.7 million. Based on current operating plans with no additional financing, the Company expects its cash runway to extend into 2029.
•Revenue: Total revenue, consisting primarily of revenue from collaboration agreements, was $18.6 million for the third quarter of 2026, compared to $5.2 million for the third quarter of 2025. The increase was driven primarily by the Company’s collaboration with Sanofi, under which the Company achieved a $10.0 million milestone for an immunology small molecule program during the quarter.
•Research and Development Expenses: Research and development expenses were $99.1 million for the third quarter of 2026, compared to $121.1 million for the third quarter of 2025. The decrease was driven primarily by $22.8 million of in-process research and development costs related to the acquisition of full rights to REC-102, Recursion’s ENPP1 inhibitor for the treatment of hypophosphatasia (HPP), from its joint venture with Rallybio that were recognized in the third quarter of 2025 and did not recur in the current period.
•General and Administrative Expenses: General and administrative expenses were $32.6 million for the third quarter of 2026 compared to $41.6 million for the third quarter of 2025. The decrease in general and administrative expense compared to the prior period was primarily driven by a decrease in infrastructure technology expenses and professional service costs.
•Net Loss: Net loss was $119.0 million for the third quarter of 2026, compared to a net loss of $162.3 million for the third quarter of 2025.
•Operational cash flows: Net cash used in operating activities was $77.4 million for the three months ended September 30, 2026, compared to net cash used in operating activities of $117.4 million for the three months ended September 30, 2025. The decrease in cash used in operating activities was primarily driven by operating efficiencies across the company and working capital movements.
•Cash Operating Expense: Cash operating expense, excluding partnership inflows and transaction costs, for the nine months ended September 30, 2026 was $271.5 million compared to $323.2 million for the nine months ended September 30, 2025.

(Press release, Recursion Pharmaceuticals, NOV 2, 2026, View Source [SID1234674036])

On November 2, 2026 Kyowa Hakko Kirin reported its results for the third quarter ending September’ 2026.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

(Press release, Kyowa Hakko Kirin, NOV 2, 2026, View Source [SID1234674035])

On November 2, 2026 Karyopharm Therapeutics Inc. (Nasdaq: KPTI) ("Karyopharm" or the "Company"), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported it has entered into a Restructuring Support Agreement with its existing lenders and noteholders and other key stakeholders, which provides for a merger between Ayrmid Ltd. ("Ayrmid") and a reorganized Karyopharm (the "Transaction"). During this process, Karyopharm remains focused on its commitment to advancing its multiple myeloma and myelofibrosis programs and improving the lives of the patients Karyopharm serves and will continue to operate in the ordinary course of business.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

To implement the Transaction, Karyopharm has filed voluntary petitions under Chapter 11 of the U.S. Bankruptcy Code in the United States Bankruptcy Court for the Southern District of Texas. The Company anticipates completing the Transaction through a Chapter 11 plan, and the Company will seek Court approval of bidding procedures, which will allow other parties to submit competing proposals and bids for the Company’s assets. The Transaction is expected to close in the first quarter of 2027, subject to the conditions described in the Chapter 11 plan.

"Following a comprehensive review of available alternatives, we believe this process provides a clear framework to pursue a value maximizing transaction for our stakeholders while continuing our important work of advancing therapies for patients with multiple myeloma and myelofibrosis," said Richard Paulson, President and Chief Executive Officer at Karyopharm. "We are pleased to have signed an agreement with Ayrmid, a company committed to advancing meaningful therapies for patients with hematological disorders. Ayrmid’s proposal underscores the strategic interest in our programs and establishes a strong foundation as we move through this process designed to maximize value. Throughout, we will remain focused on supporting our employees, maintaining continuity across the business, and advancing our mission to improve the lives of patients."

"We see a strong strategic fit between Ayrmid and Karyopharm, with complementary portfolios, established commercial capabilities and a shared commitment to addressing significant unmet needs in hematology," said Dr. Joe Wiley, Chairman and CEO of Ayrmid. "We see considerable potential in Karyopharm’s marketed products, development pipeline and experienced team, and believe that, together, we can build a broader, stronger commercial-stage hematology business with meaningful opportunities for future growth. Subject to the successful completion of the transaction, we look forward to working with the Karyopharm team to realize that potential and continue delivering important therapies to patients in need."

In connection with this process, Karyopharm has secured commitments for up to $55 million in debtor-in-possession ("DIP") financing from its existing secured lenders. Once approved by the Court, this DIP financing, combined with cash generated from the Company’s ongoing operations, is expected to support the go-forward business during the Chapter 11 process and to meet its financial obligations arising subsequent to the filing date.

Karyopharm is prepared to move through this process quickly and efficiently without any disruption to either its ordinary course operations or the partnerships essential to supporting Karyopharm’s patients and advancing its programs.

Parties interested in participating in Karyopharm’s sale process should contact Centerview Partners at [email protected].

Additional Information

Additional information about Karyopharm’s restructuring process is available at View Source, or by contacting Stretto, the Company’s noticing and claims agent, at (866) 270-4922 (toll-free) and (949) 561-0471 (international).

Karyopharm is advised in this matter by Sidley Austin LLP as legal counsel, Centerview Partners LLC as investment banker and FTI Consulting Inc. as financial and communications advisor.

An ad hoc group of term lenders and noteholders is advised in this matter by Latham & Watkins LLP and Haynes and Boone LLP as legal counsel and Houlihan Lokey Capital, Inc. as investment banker and financial advisor.

Ayrmid is advised in this matter by Paul Hastings LLP as legal counsel.

(Press release, Karyopharm, NOV 2, 2026, View Source [SID1234674034])