On June 7, 2022 Novartis reported longer-term follow-up data from the Phase III ASCEMBL trial for patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors (TKIs), presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Novartis, JUN 7, 2022, View Source [SID1234615712]). In this analysis, the proportion of patients in the Scemblix (asciminib) arm (n=157) who achieved a major molecular response (MMR) at 96 weeks was more than double that in the Bosulif (bosutinib) arm (n=76) (37.6% vs. 15.8% [P=.001]), substantially increasing from previous analyses1,2. Additionally, the probability of maintaining MMR for at least 72 weeks for patients treated with Scemblix was 96.7% (95% CI, 87.4%–99.2%), reflecting long-term durability of efficacy1.

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Despite longer duration of exposure for patients in the Scemblix arm – with a median of 23.7 months vs. 7.0 months for patients in the Bosulif arm – the updated 96-week analysis showed the proportion of patients treated with Scemblix who discontinued treatment due to adverse events (AEs) continued to be more than three times lower than those treated with Bosulif (7.7% vs. 26.3%). No new on-treatment deaths were reported since the primary analysis at 24 weeks1,2.

"In a chronic cancer where resistance can develop to many of the existing therapies, or where patients can have their quality of life negatively impacted by treatment side effects over time, it’s encouraging to see sustained and increasing efficacy with consistent adequate tolerability for patients treated with Scemblix in the longer term," said Jorge E. Cortes, MD, Director, Georgia Cancer Center, Augusta University. "This 96-week data shows the potential of Scemblix and its unique mechanism of action to help change the treatment paradigm in CML."

Scemblix is the first FDA-approved CML treatment that works by binding to the ABL myristoyl pocket3. With this novel mechanism of action, it is also known in scientific literature as STAMP inhibitor, Scemblix can help address resistance to TKI therapy in patients with Ph+ CML-CP and overcome mutations at the defective BCR-ABL1 gene, which is associated with the over-production of leukemic cells2,4-10. Scemblix continues to be studied across multiple lines of treatment for CML-CP11-18.

In addition to durable responses consistent with the primary analysis, more patients treated with Scemblix than Bosulif had BCR::ABL1≤1% (45.1% vs 19.4%) at 96 weeks. The most frequent (>10% in any treatment arm) grade ≥3 AEs on Scemblix vs. Bosulif, respectively, were thrombocytopenia (22.4%, 9.2%), neutropenia (18.6%, 14.5%), diarrhea (0%, 10.5%), and increased alanine aminotransferase (0.6%, 14.5%)1. The values for these AEs were similar to the values reported at the 24 and 48 week analyses1,2,19.

"These longer-term results offer a more robust view of the promising potential of Scemblix, and will help support ongoing regulatory filings as we seek to bring this therapy to more patients across the globe," said Jeff Legos, Executive Vice President, Global Head of Oncology & Hematology Development, Novartis. "As leaders in CML treatment innovation, we believe that with Scemblix, we have the potential to once again transform the standard of care for people affected by this disease."

Visit View Source for the latest information from Novartis at ASCO (Free ASCO Whitepaper), including our bold approach to reimagining cancer care, and access to our ASCO (Free ASCO Whitepaper) data presentations. Additional updates on trials evaluating Scemblix in earlier lines of therapy – as well as for patients with the T315I mutation – will be presented at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress, with more information available at View Source

About Scemblix (asciminib)
Scemblix (asciminib) is FDA-approved for the treatment of adult patients with Ph+ CML-CP pre-treated with two or more TKIs, as well as adult patients with Ph+ CML-CP with the T315I mutation. The first indication is approved under the US FDA Accelerated Approval Program based on MMR rate at 24 weeks; continued approval for the first indication may be contingent upon verification and description of clinical benefit from confirmatory evidence3.

Scemblix represents an important development for patients who experience resistance and/or intolerance to currently available TKI therapies, and it is being studied across multiple treatment lines for CML-CP, both as monotherapy and in combination2,11-18. Specifically, the ASC4FIRST Phase III study (NCT04971226) evaluates Scemblix in newly-diagnosed adult patients with Ph+ CML-CP vs. an investigator-selected TKI, with recruitment proceeding ahead of plan12.

Regulatory reviews for Scemblix in multiple countries and regions across the globe are ongoing. These updated 96-week ASCEMBL results are being shared with regulatory authorities, as we seek to bring Scemblix to more patients in more countries across the globe.

On June 7, 2022 The Janssen Pharmaceutical Companies of Johnson & Johnson reported initial results from the pivotal Phase 2 RAGNAR study evaluating the investigational use of BALVERSA (erdafitinib), a fibroblast growth factor receptor (FGFR) kinase inhibitor, in patients with advanced solid tumors with prespecified FGFR alterations (Press release, Johnson & Johnson, JUN 7, 2022, View Source [SID1234615731]). At a planned interim analysis (IA), responses were observed across a variety of FGFR-driven solid tumors for patients who had exhausted standard treatment options prior to being treated with BALVERSA.1 These results will be featured in an oral presentation (Abstract #3007) today at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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RAGNAR (NCT04083976) is a Phase 2 clinical study designed to evaluate the efficacy and safety of BALVERSA in patients with advanced or metastatic solid tumors and prespecified FGFR gene alterations, regardless of tumor location or histology (tumor-agnostic). The IA was based on 178 patients with 32 distinct solid tumor histologies.1 Patients in the study were prospectively identified by local molecular testing or central next-generation sequencing (NGS); the most common tumor types were cholangiocarcinoma (bile duct cancer) (n=31), high-grade glioma (tumor of the brain or spinal cord) (n=29), breast cancer (n=14), pancreatic cancer (n=13) and squamous non-small cell lung cancer (n=11).1 The study also included tumors that occur less frequently in the real world such as salivary gland and parathyroid carcinomas (rare endocrine malignancies), as well as tumors of unknown primary origin.1 Study participants were heavily pretreated, with 74.7 percent (n=133) having received two or more prior lines of therapy.1

The primary endpoint of the RAGNAR study is the overall response rate (ORR) as assessed by an independent review committee (IRC). At the IA data cutoff, IRC assessed an ORR of 29.2 percent (95 percent confidence interval [CI], 22.7-36.5) and a disease control rate (DCR) of 72.5 percent (95 percent CI, 65.3-78.9) for the overall tumor-agnostic patient population.1 Investigators observed responses in 14 distinct tumor types. This included responses in hard-to-treat malignancies such as salivary gland cancer (100 percent ORR; treated n=5, responders n=5), pancreatic cancer (31 percent ORR; treated n=13, responders n=4) and glioblastoma (21 percent ORR; treated n=29, responders=6).1 Investigators also observed an overall 7.1-month median duration of response (DOR) (95 percent CI, 5.5-9.3)1. At the data cutoff, 51.1 percent (n=24) of patients who had responded to treatment continued to show a response.1 The primary analysis for all patients treated in this RAGNAR cohort, known as the broad panel cohort, is anticipated later this year.

The safety profile of BALVERSA observed in RAGNAR was consistent with the known safety profile of BALVERSA in metastatic urothelial carcinoma (mUC). Across tumor types, 44.9 percent of patients experienced adverse events of grade three or above.1 Adverse events were manageable with supportive care and treatment interruptions or reductions, when necessary.1 The discontinuation rate due to drug-related adverse events was 7.3 percent.1

"Diagnostic advances in the identification of FGFR gene alterations have opened the door to targeted, tumor-agnostic treatment approaches for patients," said Yohann Loriot, M.D., Ph.D., Institut Gustave Roussy and University of Paris-Saclay, and principal study investigator.‡ "Results from the RAGNAR study show that, through the targeted inhibition of FGFR receptors, we may be able to tailor treatment for patients with advanced FGFR-driven cancers, regardless of tumor location or histology."

In 2019, BALVERSA was granted accelerated approval by the U.S. Food and Drug Administration (FDA) as a targeted therapy for adult patients with locally advanced or mUC with susceptible FGFR2 or FGFR3 alterations and who have progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.2

"Janssen is committed to advancing precision medicine approaches for the treatment of patients with biomarker-driven cancers, an area of clear unmet need," said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. "RAGNAR, Janssen’s first tumor-agnostic study, demonstrates our commitment to understand the biology of disease, identify new treatment pathways and improve patient outcomes. We look forward to progressing the development of BALVERSA for these patients and sharing additional updates on this program in the future."

About FGFR Alterations
Fibroblast growth factor receptors are a family of receptor tyrosine kinases that help cells grow, survive and multiply; FGFRs play a key role in several biological processes including tissue repair, inflammatory response and metabolism.3,4,5 Fusions or mutations in the genes that control FGFR (known as FGFR1–4 alterations) may lead to the development and progression of certain cancers by increasing tumor cell growth and survival.4 Patients with advanced, FGFR-driven solid tumors who have exhausted standard treatment options typically face a poor prognosis.

About the RAGNAR Study
RAGNAR (NCT04083976) is a Phase 2 clinical trial evaluating the safety and efficacy of BALVERSA in patients with advanced solid tumors, regardless of cancer type or tumor location (tumor-agnostic), driven by FGFR1–4 alterations. Patients in the trial have progressed on or after at least one line of systemic therapy and have no alternative standard treatment options. Following screening by local molecular testing or central NGS, patients are enrolled in four separate cohorts: a broad panel cohort of patients with pathogenic FGFR mutations or gene fusions (tumor histologies evaluated include but are not limited to cholangiocarcinoma [bile duct cancer], high- and low-grade glioma [a tumor type occurring in the brain or spinal cord], breast, pancreatic, squamous and non-squamous non-small cell lung cancer, colorectal, endometrial, esophageal, salivary gland, ovarian, duodenal [cancer occurring in the first part of the small intestine], thyroid and cancer of unknown primary origin); an exploratory cohort of patients with other FGFR mutations; a cholangiocarcinoma expansion cohort; and a pediatric cohort of patients ages 6 to 17 with FGFR alterations.1

The primary endpoint of RAGNAR is IRC-assessed ORR. Key secondary endpoints include investigator-assessed ORR, DOR, DCR, clinical benefit rate, progression free survival, overall survival and incidence and severity of adverse events.

About BALVERSA
BALVERSA (erdafitinib) is a once-daily, oral FGFR kinase inhibitor that is approved by the U.S. FDA for the treatment of adults with locally advanced or mUC that has susceptible FGFR3 or FGFR2 genetic alterations and has progressed during or following at least one line of platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Patients are selected for therapy based on an FDA-approved companion diagnostic for BALVERSA. Information on FDA-approved tests for the detection of FGFR genetic alterations in urothelial cancer is available at: View Source This indication is approved under accelerated approval based on tumor response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.2,6

In addition to RAGNAR, BALVERSA is being studied in clinical trials including the Phase 3 THOR (NCT03390504) study evaluating BALVERSA versus standard of care, consisting of chemotherapy (docetaxel or vinflunine) or anti-PD-1 agent pembrolizumab, in participants with advanced urothelial cancer and selected FGFR aberrations with disease progression following one or two prior lines of therapy; and the randomized Phase 2 THOR-2 (NCT04172675) study examining BALVERSA versus investigator choice of intravesical chemotherapy in participants who received Bacillus Calmette-Guérin and recurred with high risk non-muscle-invasive bladder cancer.7,8

In 2008, Janssen Pharmaceutica NV entered into an exclusive worldwide license and collaboration agreement with Astex Pharmaceuticals to develop and commercialize BALVERSA.

For more information, visit www.BALVERSA.com.

BALVERSA IMPORTANT SAFETY INFORMATION

Warnings and Precautions
Ocular Disorders – BALVERSA can cause ocular disorders, including central serous retinopathy/retinal pigment epithelial detachment (CSR/RPED) resulting in visual field defect.

CSR/RPED was reported in 25% of patients treated with BALVERSA, with a median time to first onset of 50 days. Grade 3 CSR/RPED, involving central field of vision, was reported in 3% of patients. CSR/RPED resolved in 13% of patients and was ongoing in 13% of patients at the study cutoff. CSR/RPED led to dose interruptions and reductions in 9% and 14% of patients, respectively, and 3% of patients discontinued BALVERSA. Dry eye symptoms occurred in 28% of patients during treatment with BALVERSA and were Grade 3 in 6% of patients. All patients should receive dry eye prophylaxis with ocular demulcents as needed.

Perform monthly ophthalmological examinations during the first 4 months of treatment and every 3 months afterwards, and urgently at any time for visual symptoms. Ophthalmological examination should include assessment of visual acuity, slit lamp examination, fundoscopy, and optical coherence tomography. Withhold BALVERSA when CSR occurs and permanently discontinue if it does not resolve within 4 weeks or if Grade 4 in severity. For ocular adverse reactions, follow the dose modification guidelines [see Dosage and Administration (2.3)].

Hyperphosphatemia and Soft Tissue Mineralization – BALVERSA can cause hyperphosphatemia leading to soft tissue mineralization, cutaneous calcinosis, non-uremic calciphylaxis and vascular calcification. Increases in phosphate levels are a pharmacodynamic effect of BALVERSA [see Pharmacodynamics (12.2)]. Hyperphosphatemia was reported as adverse reaction in 76% of patients treated with BALVERSA. The median onset time for any grade event of hyperphosphatemia was 20 days (range: 8–116) after initiating BALVERSA. Thirty-two percent of patients received phosphate binders during treatment with BALVERSA. Cutaneous calcinosis, non-uremic calciphylaxis and vascular calcification have been observed in 0.3% of patients treated with BALVERSA.

Monitor for hyperphosphatemia throughout treatment. In all patients, restrict phosphate intake to 600-800 mg daily. If serum phosphate is above 7.0 mg/dL, consider adding an oral phosphate binder until serum phosphate level returns to <5.5 mg/dL. Withhold, dose reduce, or permanently discontinue BALVERSA based on duration and severity of hyperphosphatemia [see Dosage and Administration (2.3), Table 2: Dose Modifications for Adverse Reactions].

Embryo-fetal Toxicity – Based on the mechanism of action and findings in animal reproduction studies, BALVERSA can cause fetal harm when administered to a pregnant woman. In a rat embryo-fetal toxicity study, erdafitinib was embryotoxic and teratogenic at exposures less than the human exposures at all doses studied. Advise pregnant women of the potential risk to the fetus. Advise female patients of reproductive potential to use effective contraception during treatment with BALVERSA and for one month after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with BALVERSA and for one month after the last dose [see Use in Specific Populations (8.1, 8.3) and Clinical Pharmacology (12.1)].

Most common adverse reactions including laboratory abnormalities ≥20%:
Phosphate increased (76%), stomatitis (56%), fatigue (54%), creatinine increased (52%), diarrhea (47%), dry mouth (45%), onycholysis (41%), alanine aminotransferase increased (41%), alkaline phosphatase increased (41%), sodium decreased (40%), decreased appetite (38%), albumin decreased (37%), dysgeusia (37%), hemoglobin decreased (35%), dry skin (34%), aspartate aminotransferase increased (30%), magnesium decreased (30%), dry eye (28%), alopecia (26%), palmar-plantar erythrodysesthesia syndrome (26%), constipation (28%), phosphate decreased (24%), abdominal pain (23%), calcium increased (22%), nausea (21%), and musculoskeletal pain (20%). The most common Grade 3 or greater adverse reactions (>1%) were stomatitis (9%), nail dystrophy*, palmar-plantar erythrodysesthesia syndrome (6%), paronychia (3%), nail disorder*, keratitis†, onycholysis* (10%), and hyperphosphatemia.

*Included within onycholysis. †Included within dry eye.

An adverse reaction with a fatal outcome in 1% of patients was acute myocardial infarction.
Serious adverse reactions occurred in 41% of patients, including eye disorders (10%).
Permanent discontinuation due to an adverse reaction occurred in 13% of patients. The most frequent reasons for permanent discontinuation included eye disorders (6%).
Dosage interruptions occurred in 68% of patients. The most frequent adverse reactions requiring dosage interruption included hyperphosphatemia (24%), stomatitis (17%), eye disorders (17%), and palmar-plantar erythrodysesthesia syndrome (8%).
Dose reductions occurred in 53% of patients. The most frequent adverse reactions for dose reductions included eye disorders (23%), stomatitis (15%), hyperphosphatemia (7%), palmar-plantar erythrodysesthesia syndrome (7%), paronychia (7%), and nail dystrophy (6%).
Drug Interactions

Moderate CYP2C9 or strong CYP3A4 Inhibitors: Consider alternative agents or monitor closely for adverse reactions. (7.1)
Strong CYP2C9 or CYP3A4 inducers: Avoid concomitant use with BALVERSA. (7.1)
Moderate CYP2C9 or CYP3A4 inducers: Increase BALVERSA dose up to 9 mg. (7.1)
Serum phosphate level-altering agents: Avoid concomitant use with agents that can alter serum phosphate levels before the initial dose modification period. (2.3, 7.1)
CYP3A4 substrates: Avoid concomitant use with sensitive CYP3A4 substrates with narrow therapeutic indices. (7.2)
OCT2 substrates: Consider alternative agents or consider reducing the dose of OCT2 substrates based on tolerability. (7.2)
P-gp substrates: Separate BALVERSA administration by at least 6 hours before or after administration of P-gp substrates with narrow therapeutic indices. (7.2)
Use in Specific Populations
Lactation – Because of the potential for serious adverse reactions from erdafitinib in a breastfed child, advise lactating women not to breastfeed during treatment with BALVERSA and for one month following the last dose.

Please see the full Prescribing Information for BALVERSA.

On June 7, 2022 Elevar Therapeutics has reported the Phase II clinical trial results of its small-molecule tyrosine kinase inhibitor (TKI) rivoceranib in progressive recurrent or metastatic adenoid cystic carcinoma (R/M ACC) patients (Press release, Elevar Therapeutics, JUN 7, 2022, View Source [SID1234615922]).

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The open-label Study RM-202 of the orally administered TKI was carried out at 11 sites in the US and South Korea.

It has been designed to investigate the therapy’s safety and efficacy in patients with progressive R/M ACC.

Study RM-202 evaluated rivoceranib in 80 patients, including 53 (66.3%) based in the US.

Within six months before the trial, all the participants demonstrated tumor progression.

An overall response rate (ORR) of 15.1% was observed in the trial, and the remaining 85% of patients had a tumour size reduction, though not an endpoint of the trial.

Elevar Therapeutics CEO Saeho Chong said: "With every participant exhibiting a recent growing lesion upon entering this Phase II trial of rivoceranib, these results demonstrate significant clinical effectiveness and rivoceranib’s promise as a potential new treatment for patients with R/M ACC.

"Our entire Elevar team is greatly encouraged by these results, and we are fully focused on advancing rivoceranib through the regulatory process."

In this trial, 52% of subjects reported a response according to CHOI (size or density), which is believed to be more correlated with median overall survival (mOS) than RECIST v1.1 (size only).

Irrespective of previous vascular endothelial growth factor (VEGFR) therapy, median progression-free survival (mPFS) of nine months was observed versus published data of a baseline of 2.8 months for R/M ACC.

On June 7, 2022 ImmunOs Therapeutics AG, a biopharmaceutical company leveraging its HLA-based technology platform to develop first-in-class therapeutics for the treatment of cancer and autoimmune diseases, reported the closing of an oversubscribed Series B financing round totaling $74 million (Press release, ImmunOs Therapeutics, JUN 7, 2022, View Source [SID1234615697]). The round was led by new investors Samsara BioCapital, Lightspeed Venture Partners, and Gimv, and joined by new investors Mission BioCapital, GL Capital, PEAK6 Strategic Capital, and Fiscus Financial, as well as existing investors Pfizer Ventures, BioMed Partners, and Schroder Adveq. In connection with the financing, Shelley Chu, MD, PhD, Partner at Lightspeed, Marcos Milla, PhD, Venture Partner at Samsara, and Andreas Jurgeit, PhD, Partner at Gimv, will join the Company’s Board of Directors, which currently includes Reinhard Ambros, PhD, Chairperson and former Head of the Novartis Venture Fund, Daniel Vasella, MD, Vice Chairperson and former Chairperson and CEO of Novartis AG, Markus Hosang, PhD, General Partner at BioMed Partners, Michael Baran, PhD, Partner at Pfizer Ventures, and Sean R. Smith, CEO of ImmunOs.

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The proceeds of the Series B will be used to fund the clinical development of ImmunOs’ lead program, IOS-1002 (formerly iosH2), through Phase 2 clinical trials and to progress additional oncology and autoimmune disease programs within the Company´s HLA-based pipeline towards clinical development. IOS-1002 is a first-in-class, multi-functional agent based on a naturally occurring human leukocyte antigen (HLA) that targets key components of the innate immune system, including LILRB1 (ILT2), LILRB2 (ILT4), and KIR3DL1, and synergizes with the adaptive immune system, thereby leading to profound anti-tumor activity. A Phase 1 clinical trial is planned to start in the second half of 2022.

Additionally, ImmunOs has established a U.S. subsidiary to further expand its international reach, execute future U.S. clinical trials, and strengthen its transatlantic operations and team.

"We are delighted to close this significant financing round led by a group of top-tier U.S. and European investors," said Sean R. Smith, CEO of ImmunOs Therapeutics. "The raise underlines our promising, first-in-class immunotherapy approach for the treatment of both solid and liquid tumors. The funding allows us to advance our lead program, IOS-1002, into clinical trials, expand our pipeline in oncology and autoimmune disease, and strengthen our transatlantic operations. We are honored that renowned investors such as Samsara, Lightspeed, Gimv, Mission BioCapital, GL Capital, PEAK6, and Fiscus have joined our investor base."

"We are excited to invest in ImmunOs Therapeutics with its innovative HLA-based technology platform, expert leadership team, and strong syndicate of investors," said Shelley Chu, MD, PhD, Partner at Lightspeed Venture Partners. "The Company is ideally positioned to advance its potentially first-in-class immunotherapies for the benefit of patients with limited therapeutic options. We look forward to supporting ImmunOs’ success."

"Myeloid checkpoint therapeutics are potentially the next major breakthrough in immuno-oncology, and we believe ImmunOs Therapeutics will contribute exciting medicines in this rapidly evolving field to help patients suffering from cancer," continued Marcos Milla, PhD, Venture Partner at Samsara. "We look forward to generating data from the initial clinical trials of IOS-1002."

"ImmunOs’ approach of identifying HLA molecules with naturally optimized affinity and specificity profiles is not just elegant but also truly differentiated – a novel modality leveraging the co-evolution of immune-regulatory mechanisms," stated Andreas Jurgeit, PhD, Partner at Gimv. "We are extremely excited by the progress being made at ImmunOs and proud to support the team towards clinical validation for the benefit of patients."

On June 7, 2022 Genexine (KOSDAQ: 095700) a publicly traded, clinical stage biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, reported top-line results from the KEYNOTE-899 phase 1b/2 clinical trial of GX-I7 (efineptakin alfa) in combination with pembrolizumab (Press release, Genexine, JUN 7, 2022, View Source [SID1234615713]). The results indicated that GX-I7 in combination with pembrolizumab was safe and well tolerated and demonstrated promising early anti-tumor activity in patients with R/R metastatic TNBC. Genexine presented these data in a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting taking place from June 3-7, 2022.

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Top-line data showed that GX-17 in combination with pembrolizumab was safe and well tolerated in the overall phase 1b/2 trial. For the phase 2 expansion cohort GX-I7 was administered at a dose of 1,200 µg/kg in nine-week intervals combined with pembrolizumab at 200 mg administered every three weeks.

Observed ORRs with GX-17 in combination pembrolizumab were 15.7% (8/51) for phase 1b and 21.2% (7/33) for phase 2. Of the 25 patients who had an evaluable PD-L1 from a biopsy sample, 40.0% (10/25) were PD-L1 positive (CPS≥10). Notably, the ORR in patients who were PD-L1 positive was 60% (6/10). Absolute lymphocyte count and the number of CD4+ and CD8+ T cells were significantly increased, while NLRs and proportion of Treg in CD4+ T cells were significantly decreased in patients receiving GX-I7 720 µg/kg or higher.

"The results from this study are quite encouraging and indicate that GX-I7 (efineptakin alfa) in combination with a check point inhibitor could become an important therapy for patients with r/r metastatic TNBC." said Professor Sohn Ju-hyuk the primary investigator of the trial and professor of Medical Oncology at Yonsei Severance Hospital. "GX-I7 represents a new class of potential therapy for cancer patients and could become the first therapy to manage lymphopenia in cancer patients. Although this is a retrospective analysis, I am encouraged by the 60% ORR in PD-L1 positive patients especially considering there was no response in the PD-L1-negative group with previous study. I believe further trials with PD-L1 positive TNBC patients are warranted."

"GX-I7 represents a potential first-in-class treatment for cancer patients. We are pleased with the observed safety and tolerability profile and are encouraged by the efficacy shown in these two trials," said Neil Warma, CEO of Genexine. "We plan on carefully analyzing the complete data set but are highly encouraged by the CPS>10 cohort that showed a robust 60% ORR. Although this was observed in a modest sample size, we believe it warrants further review. GX-I7 is one of our flagship products and Genexine has embarked on a global development program in multiple cancers with our partners, NeoImmune Tech in the U.S. and I-Mab in China."

GX-I7 (efineptakin alfa), discovered and developed by Genexine, is a hybrid Fc-fused long-acting recombinant human IL-7 which plays an essential role in the development and homeostasis of T-cells. T-cells play an important role in fighting cancer by recognizing cancer cells and killing them directly or indirectly by communicating with other immune cells. As a T-cell amplifier, GX-I7 may boost the immune system and help eradicate tumor cells more effectively. GX-I7 may modulate multiple steps in the cancer immunity cycle to overcome resistance to current immunotherapy. By working synergistically with leading and emerging immuno-oncology therapeutics, GX-I7 may broaden, deepen, and prolong anti-tumor responses in cancer patients.