Onco-Summaries: Daily Oncology Updates at a Glance
- Oncofocus Team
- Jul 31
- 4 min read
Updated: Aug 4
30/07/2026
NMPA grants priority review to sacituzumab tirumotecan for first‑line TNBC (Ref)
Kelun-Biotech announced that China's National Medical Products Administration (NMPA) has accepted a new indication application for its drug sacituzumab tirumotecan (sac-TMT), a TROP2-targeted antibody drug conjugate. The new application seeks approval for first-line use in patients with recurrent or metastatic triple-negative breast cancer (TNBC) who have low PD-L1 expression or who relapsed after earlier immunotherapy.
The application is based on Phase III OptiTROP‑Breast03 trial: randomized, open‑label, multicenter study comparing sac‑TMT vs investigator’s choice chemotherapy in advanced TNBC.
Results showed statistically significant and clinically meaningful efficacy improvements over chemotherapy
Previously, sac-TMT was granted Breakthrough Therapy Designation (BTD) by the NMPA for the first-line treatment of unresectable locally advanced, recurrent or metastatic PD-L1-negative TNBC
The new application is under priority review, expected to accelerate approval
FDA Advisory Committee Backs RP1's Efficacy Data in Advanced Melanoma (Ref)
Replimune announced that the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) met to review the BLA resubmission for RP1 (vusolimogene oderparepvec) combined with nivolumab in advanced melanoma patients who progressed on prior anti-PD-1 therapy.
The committee voted 10 to 3 that the efficacy results from the IGNYTE study are evaluable and clinically meaningful, a favorable outcome for the resubmission.
The committee examined two questions: whether the single-arm IGNYTE study design allows reliable determination of response rate and durability, and whether the observed responses reflect clinically meaningful, systemic antitumor activity attributable to RP1
Patients and physicians spoke at the meeting, emphasizing the high unmet need in advanced melanoma and the importance of new options like RP1
CEO Sushil Patel called the outcome encouraging and said the company looks forward to continuing to work with the FDA as it completes its BLA review
Upcoming milestone: The FDA's target action date for this Class 1 resubmission is Aug 2, 2026
FDA Grants Orphan Drug Designation to Enzomenib for Acute Lymphoblastic Leukemia (Ref)
Sumitomo Pharma America announced that the FDA granted orphan drug designation to enzomenib (DSP-5336; oral small-molecule inhibitor targeting the menin-KMT2A protein interaction) for treating acute lymphoblastic leukemia (ALL)
This is enzomenib's second orphan drug designation from the FDA. It previously received the designation for acute myeloid leukemia (AML) back in June 2022
The drug also holds FDA Fast Track Designation (June 2024) for relapsed/refractory AML with KMT2A rearrangement or NPM1 mutation, plus an orphan designation from Japan's MHLW (September 2024) for the same subtype
Ongoing/upcoming milestones:
Enzomenib is currently in a Phase 1/2 dose-escalation/dose-expansion trial (NCT04988555) in relapsed/refractory acute leukemia patients
It is also being evaluated in a registrational Phase 2 trial called "Horizen-1," targeting relapsed/refractory AML and ALL patients with KMT2A rearrangements or NPM1 mutations
REVTORPYK (gedatolisib) Added to NCCN Guidelines for HR+/HER2- Breast Cancer (Ref)
Celcuity announced that its recently FDA-approved drug REVTORPYK [gedatolisib; a pan-PI3K/mTOR ("PAM") inhibitor] has been included in the NCCN Clinical Practice Guidelines as a preferred Category 1 second-line-plus therapy for HR+/HER2- locally advanced or metastatic breast cancer
REVTORPYK was approved on July 14, 2026 for HR+/HER2- locally advanced or metastatic breast cancer without a PIK3CA mutation, after progression on endocrine therapy (Ref)
Supporting data:
REVTORPYK is used in combination with fulvestrant, with or without palbociclib, for patients without a PIK3CA mutation who progressed after at least one line of endocrine therapy, and it's the only approved inhibitor targeting class I PI3K isoforms plus mTORC1/2
Approval was based on the Phase 3 VIKTORIA-1 trial: the triplet regimen (REVTORPYK + palbociclib + fulvestrant) showed median progression-free survival of 9.3 months versus 2.0 months for fulvestrant alone, with a 32% objective response rate; the doublet (REVTORPYK + fulvestrant) showed 7.4 months PFS versus 2.0 months, with a 28% response rate
Notable safety concerns include stomatitis (up to 72% incidence), rash, and hyperglycemia, requiring monitoring and preventive measures like prophylactic mouthwash.
Celcuity has set up a patient support and expanded access program ahead of commercial availability.
Upcoming milestones:
Commercial launch in the US is expected in late Q3 2026
Karyopharm to file sNDA in Aug 2026, for selinexor + ruxolitinib in Myelofibrosis under accelerated approval pathway (Ref)
Karyopharm announced plans to submit a supplemental New Drug Application (sNDA) in August 2026 for the combination of selinexor (XPO1 inhibitor) + ruxolitinib (JAK1/2 inhibitor) for the treatment of patients with Myelofibrosis, seeking Priority Review under the accelerated approval pathway
Regulatory Strategy
FDA confirmed SVR35 (≥35% spleen volume reduction) is an acceptable surrogate endpoint predictive of overall survival
Preliminary overall survival data from the Phase 3 SENTRY trial supports accelerated approval
Long‑term OS follow‑up will serve as confirmatory evidence
If approved, regimen represents the first combination therapy for Myelofibrosis
Selinexor Phase 3 Endometrial cancer trial shows non-significant PFS trend (Ref)
Karyopharm announced topline results from its Phase 3 XPORT-EC-042 trial evaluating selinexor (XPO1 inhibitor) vs placebo as maintenance therapy in endometrial cancer
The study did not meet its primary endpoint of progression-free survival (PFS) with statistical significance, though a clinically meaningful trend was observed
Primary endpoint: Median PFS was 12.75 months with selinexor vs 7.43 months with placebo in the modified intent-to-treat population (HR=0.76; one-sided p=0.0791)
Safety: Consistent with prior selinexor data; no new safety signals reported
Strategic Implications
Karyopharm will deprioritize endometrial cancer investment
Focus will shift to myelofibrosis and multiple myeloma programs, including submission of an sNDA and upcoming Phase 2 SENTRY-2 data in myelofibrosis (expected 2H 2026)
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