Replimune Reports Fiscal Fourth Quarter and Year-Ended 2021 Financial Results and Provides Corporate Update

On May 20, 2021 Replimune Group, Inc. (NASDAQ: REPL), a biotechnology company developing a series of oncolytic immuno-gene therapies derived from its Immulytic platform, reported financial results for the fiscal fourth quarter and year ended March 31, 2021 and provided a business update (Press release, Replimune, MAY 20, 2021, View Source [SID1234580379]).

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"We have made good progress advancing our programs over the quarter and look forward to providing a data update with RP1 and RP2 at our virtual investor event being held in June as we continue to enroll into our registration-directed clinical trials in CSCC and anti-PD1 failed melanoma, with expected readouts in 2022," said Philip Astley-Sparke, CEO of Replimune. "Our vision extends well beyond our initial lead skin cancer indications, with the broader aim of becoming a cornerstone for immuno-oncology treatments across a wide spectrum of tumor types. With this in mind, and as we move closer to market, we recently expanded our senior management team with two key hires with a considerable track record of success in bringing multiple high profile programs to BLA approval and in overseeing the launch of new drugs in the immuno-oncology space."

Corporate Updates

Expanded management team with key hires to prepare for the transition to a commercial company. The Company hired Sushil Patel, Ph.D. as Chief Commercial Officer, arriving from Genentech where he was the head of their global oncology franchise for lung cancer, skin cancer and rare / agnostic tumor types, and previously the lifecycle leader in lung cancer for the multi-billion-dollar checkpoint blockade drug Tecentriq. In addition to hiring Dr. Patel, the Company also appointed Tanya Lewis as Chief Development Operations Officer. Ms. Lewis’s past accomplishments include successful negotiations related to registration trial designs, approval, and/or commercialization of XPOVIO, VELCADE, VARUBI, INTEGRILIN and ZEJULA.

Announced plans to amend the CERPASS clinical trial protocol to add complete response (CR) rate as an additional independent primary endpoint. The amendment is based on the depth and durability of responses and the manageable safety profile seen in patients with non-melanoma skin cancers treated with RP1 in combination with Opdivo to date. Under the modified clinical trial protocol for CERPASS, Replimune plans to add CR rate as an additional independent primary endpoint, in addition to overall response rate (ORR), and to reduce target enrollment from 240 patients to 180 patients. Secondary endpoints will continue to include duration of response, progression-free survival (PFS), and overall survival (OS). Replimune plans to submit the amended protocol to the FDA by the end of this quarter and is maintaining its guidance to expect primary data read out in 2022.

Held Type B meeting with the FDA to discuss the regulatory pathway for RP1 in combination with Opdivo (nivolumab) in anti-PD1 failed melanoma. Replimune recently held a Type B meeting with the FDA to discuss the design of the currently enrolling 125-patient registration-directed cohort of patients with anti-PD1 failed melanoma in the IGNYTE clinical trial. The FDA expressed that while a randomized controlled clinical trial would always be preferred for registration, if the clinical data is sufficiently compelling in this patient population with no clear standard of care, then the data could be submitted to the FDA for review under the accelerated approval pathway. The FDA also indicated that a randomized confirmatory trial would be needed as is required under the accelerated approval process.

Presented new biomarker and pre-clinical data for RP1 and RP2, at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data presented continues to confirm potent anti-tumor activity and activation of robust systemic immune activation by RP1 and RP2.

Virtual Investor Event to be held on Thursday, June 3, 2021. The Company will host a virtual investor event to present updated data from its Phase 2 skin cancer cohorts combining RP1 with Opdivo and data from its Phase 1 study of RP2 alone and in combination with Opdivo. The event will include presentations by the management team and Mark Middleton, Professor of Experimental Cancer Medicine in the Department of Oncology, consultant Medical Oncologist at the Oxford Cancer and Hematology Centre and Head of the Department of Oncology at the University of Oxford.

First RP1 batches produced and filled at state-of-the-art manufacturing facility. Release testing is underway for the first GMP RP1 batches produced at its 63,000-square-foot manufacturing facility in Framingham, MA, that was built to support the commercialization of all of its products. Technology transfer for RP2 has also commenced and is expected to complete in the next quarter.
Program Highlights and Upcoming Milestones

RP1 in combination with Libtayo (cemiplimab) in CSCC: CERPASS, the Company’s Phase 2, global, randomized, controlled, registration-directed clinical trial continues to actively enroll patients and remains on track for a primary data read out in 2022.

RP1 in combination with Opdivo in anti-PD-1 failed melanoma: The Company’s 125-patient cohort in the IGNYTE Phase 2 clinical trial of RP1 in combination with Opdivo, continues to actively enroll patients and remains on track to report the primary data read out in 2022.

RP1 in combination with Opdivo in melanoma and non-melanoma skin cancers (NMSC): Following a positive Phase 2 data update in October 2020 and enrollment of the initial melanoma cohort (including anti-PD1 naïve and anti-PD1 failed patients) being complete, the Company continues to enroll its 45-patient cohort evaluating RP1 in combination with Opdivo in NMSC. The Company plans to provide an update on these programs at its June 3rd investor event.

RP1 in anti-PD1 failed NSCLC: The first patient in the cohort of 30 anti-PD1 failed NSCLC patients treated with RP1 combined with Opdivo has been dosed, and the Company expects to report initial data in the second half of 2021.

RP1 as monotherapy in solid organ transplant recipients with CSCC: The Company is currently enrolling a 30 patient Phase 1b clinical trial (ARTACUS) assessing the safety and efficacy of RP1 in liver and kidney transplant recipients with CSCC. Enrollment in this immuno-compromised population has been hampered by COVID 19, but as the effects of the pandemic reduce the Company expects recruitment to increase. Early data from this clinical trial is intended to be presented in the second half of 2021.

RP1 in combination with Opdivo in MSI-H/dMMR tumors: The Company continues to expect to be able to decide whether to pursue RP1 for MSI-H/dMMR tumors into registration-directed development by the end of 2021.

RP2 alone and in combination with Opdivo: Following positive data with RP2 given as monotherapy that were presented in October 2020, the Company is actively enrolling a 30-patient cohort evaluating RP2 in combination with Opdivo. Updated data from this clinical trial, including an update on patients treated with RP2 monotherapy and initial data with RP2 in combination with Opdivo will be presented at the upcoming June 3rd investor event.

RP3 alone and in combination with anti-PD-1 therapy: The Phase 1 clinical trial evaluating RP3 alone and in combination with anti-PD1 therapy in solid tumor patients is actively enrolling and the Company remains on track to report initial data in the second half of 2021.

Target evaluation for new indications is currently underway: The Company remains on track to disclose part of its initial development plans for RP2 and/or RP3 in less immune responsive tumor types at the upcoming June 3rd investor event.
Financial Fiscal Quarter Four and Year End Highlights:

Cash Position: As of March 31, 2021, cash, cash equivalents and short-term investments were $476.3 million, as compared to $168.6 million as of March 31, 2020. This increase was primarily related to $372.5 million in net proceeds from financing activities offset by an increase in cash utilized in operating activities in advancing our expanded clinical development plan.

Based on the current operating plan, Replimune believes that existing cash and cash equivalents and short-term investments will fund operating expenses and capital expenditure requirements into the second half of 2024.
R&D Expenses: Research and development expenses were $16.2 million for the fourth quarter and $56.8 million for the fiscal year ended March 31, 2021, as compared to $11.2 million for the fourth quarter and $38.8 million for the fiscal year ended March 31, 2020. This increase was primarily due to clinical expenses driven by the company’s lead programs, expansion into additional studies, operating our dedicated manufacturing facility and related increased personnel costs. Research and development expenses included $2.0 million in stock-based compensation expenses for the fourth quarter and $5.7 million in stock-based compensation expenses for the fiscal year ended March 31, 2021.

G&A Expenses: General and administrative expenses were $6.0 million for the fourth quarter and $23.2 million for the fiscal year ended March 31, 2021, as compared to $5.2 million for the fourth quarter and $17.4 million for the year ended March 31, 2020. The increase was primarily driven by personnel related costs, professional fees, and facility expansion. General and administrative expenses included $1.5 million in stock-based compensation expenses for the fourth quarter and $6.0 million in stock-based compensation expenses for the fiscal year ended March 31, 2021.

Net Loss: Net loss was $21.5 million for the fourth quarter and $80.9 million for the fiscal year ended March 31, 2021, as compared to a net loss of $15.8 million for the fourth quarter and $52.6 million for the fiscal year ended March 31, 2020.
About CERPASS
CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial will enroll 180 patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD1 therapy. The trial will evaluate complete response (CR) rate and overall response rate (ORR) as its two primary efficacy endpoints as assessed by independent review, as well as duration of response, progression-free survival (PFS), and overall survival (OS) as its secondary endpoints. The study is being run under a clinical trial collaboration agreement with Regeneron in which the costs of the trial are shared and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi.
Libtayo is a registered trademark of Regeneron.

About IGNYTE
IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo. There are 4 tumor specific cohorts currently enrolling in this trial including a 125-patient extension cohort of RP1 combined with Opdivo in anti-PD-1 failed cutaneous melanoma. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same trial of approximately 30 patients with melanoma. The additional thirty patient cohorts are studying RP1 in combination with Opdivo in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in microsatellite instability high, or MSI-H/dMMR tumor types and anti-PD-1 failed non-small cell lung cancer, or NSCLC. This trial is being done under a collaboration and supply agreement with Bristol Myer Squibb.
Opdivo is a registered trademark of Bristol-Myers Squibb Company.

About RP1
RP1 is Replimune’s lead Immulytic product candidate and is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response.

About RP2 & RP3
RP2 and RP3 are derivatives of RP1 that express additional proteins. RP2 expresses an anti-CTLA-4 antibody-like molecule and RP3 additionally expresses the immune co-stimulatory pathway activating proteins CD40L and 4-1BBL. RP2 and RP3 are intended to provide targeted and potent delivery to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic immune-based efficacy on tumors and limiting off-target toxicity.

BostonGene Announces Publication in Cancer Cell Revealing Four Tumor Microenvironment Subtypes Predict Response to Immunotherapy Across Multiple Cancers

On May 20, 2021 BostonGene Corporation, a biomedical software company committed to defining optimal precision medicine-based therapies for cancer patients, reported a publication in Cancer Cell, a premier peer-reviewed scientific journal that publishes high impact results in cancer research and oncology (Press release, BostonGene, MAY 20, 2021, View Source [SID1234580396]). The manuscript, "Conserved pan-cancer microenvironment subtypes predict response to immunotherapy", describes a transcriptomic-based tumor classification platform that identifies distinct tumor microenvironment subtypes across a broad array of cancers, predicting prognosis and response to immune checkpoint blockade.

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"These findings reinforce the power of integrated analysis to discover unique and clinically applicable characteristics of the tumor microenvironment, a crucial factor in therapeutic response in multiple cancers"

"These findings reinforce the power of integrated analysis to discover unique and clinically applicable characteristics of the tumor microenvironment, a crucial factor in therapeutic response in multiple cancers," said Nathan Fowler, MD, Chief Medical Officer at BostonGene. "The results of our study provided the foundation for us to launch BostonGene Tumor Portrait TestsTM, which are designed to identify key drivers of each tumor, enabling doctors to personalize therapy for cancer patients. The study also represents a major milestone in the landmark research BostonGene is conducting and underscores the importance of innovative multi-platform analytics combined with cutting-edge software to improve patient outcomes."

For this research study, tumor transcriptome sequencing was analyzed to holistically describe and comprehensively characterize cancer cells and the surrounding tumor microenvironment. Transcriptomic data of over 10,000 cancer patients were analyzed, leading to the identification of four unique microenvironment subtypes that are conserved across 20 different cancers. These four subtypes are strongly associated with prognosis and response to immunotherapy in different cancers. Additionally, a personalized tumor map was created to visually depict key molecular and immune characteristics of each tumor. The microenvironment classification platform and tumor map provide a clinically useful and robust methodology for response prediction and incorporate precision medicine strategies across varied cancer types.

The BostonGene Tumor Portrait TestsTM reveal key drivers of each tumor, including immune microenvironment properties, actionable mutations, biomarkers of response to diverse therapies, and recommended therapies. Through these comprehensive analyses, BostonGene Tumor Portrait TestsTM generate a personalized roadmap for therapeutic decision-making for each cancer patient.

Immutep announces publication of TACTI-002 and INSIGHT-004 abstracts for ASCO 2021 Annual Meeting

On May 20, 2021 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a biotechnology company developing novel LAG-3 related immunotherapy treatments for cancer and autoimmune disease, reported that data from its TACTI-002 and INSIGHT-004 studies have been published in abstracts available via the links below on the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s (ASCO) (Free ASCO Whitepaper) 2021 Annual Meeting’s official website (Press release, Immutep, MAY 20, 2021, View Source [SID1234580430]).

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Poster presentations with new and updated data that are not part of the abstracts will be available on ASCO (Free ASCO Whitepaper).org from 9 am on 4 June 2021 US Eastern time and made available on Immutep’s website at www.immutep.com.

TACTI-002

Title: Results from a phase II study of eftilagimod alpha (soluble LAG-3 protein) and pembrolizumab in patients with PD-L1 unselected metastatic non-small cell lung carcinoma.

Abstract: View Source

Title: Results from a phase II study of eftilagimod alpha (soluble LAG-3 protein) and pembrolizumab in patients with PD-L1 unselected metastatic second- line squamous head and neck carcinoma.
Abstract: View Source

INSIGHT-004

Title: Phase I INSIGHT platform trial: Advanced safety and efficacy data from stratum D evaluating feasibility and safety of eftilagimod alpha (soluble LAG-3 protein) combined with avelumab in advanced solid tumors.
Abstract:  View Source;

About American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Conference
ASCO’s annual meeting represents the world’s largest gathering of oncology physicians, industry representatives, researchers, patient advocates, and investment analysts to discuss cutting-edge clinical research and therapeutics in oncology, and to gain insights for improving cancer care. For additional information on the 2021 ASCO (Free ASCO Whitepaper)’s Annual meeting, please visit View Source

About TACTI-002
TACTI-002 (Two ACTive Immunotherapies) is a Phase II clinical trial being conducted in collaboration with Merck & Co., Inc., Kenilworth, NJ, USA (known as "MSD" outside the United States and Canada). The study is evaluating the combination of efti with MSD’s KEYTRUDA (pembrolizumab) in up to 183 patients with second line head and neck squamous cell carcinoma or non-small cell lung cancer in first and second line.

About INSIGHT-004 (also known as INSIGHT Stratum D)
INSIGHT-004 is a Phase I clinical trial and is the fourth arm of the investigator-initiated INSIGHT trial which is being conducted by the Institute of Clinical Cancer Research IKF at Krankenhaus Nordwest in Frankfurt. It is being conducted under Immutep’s collaboration with Merck KGaA and Pfizer Inc., and is evaluating the safety, tolerability and recommended Phase II dose of efti when given in combination with avelumab, a human anti-PD-L1 antibody, in 12 patients with solid cancers. Avelumab is co-developed and co-commercialised by Merck KGaA, Darmstadt, Germany and Pfizer Inc.

Q1 2021 Report: Scandion Oncology is evolving

On May 20, 2021 Scandion Oncology A/S ("Scandion Oncology" or "the Company") reported the Q1 2021 Report for the period January 1 – March 31, 2021 (Press release, Scandion Oncology, MAY 20, 2021, View Source;scandion-oncology-is-evolving,c3350229 [SID1234580347]).

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Bo Rode Hansen, President & CEO, Scandion Oncology A/S comments:

"Scandion Oncology is evolving. Clinical progress and strengthening of the organization have marked the first quarter of 2021. With key hires in R&D, advisory roles, and business development we are building the organization with the competences needed to be ready for the upcoming milestones – the part 1 of the phase II CORIST study in Q2 2021, followed by a planned readout from the PANTAX Ib study in Q3-Q4 2021. 2021 is certainly an exciting year."

Reporting period January 1, 2021 – March 31, 2021

Net sales amounted to 0 TDKK (0)
Profit/loss before financial items amounted to -9,812 TDKK (-4,043)
Cash position amounted to 145,216 TDKK (11,013)
Earnings per share was -0.27 DKK (-0.20)
Highlights during Q1 2021

ON JANUARY 19, Scandion Oncology announced that the Company had applied for and received approval for admission to trading on Nasdaq First North Growth Market Sweden. The first day of trading was February 3, 2021.
ON JANUARY 23, Scandion Oncology announced that the Company had completed the first 12-patient cohort in the ongoing dose-range finding part of CORIST, the clinical phase II study with SCO-101 in combination with chemotherapy (FOLFIRI) in patients with drug resistant metastatic colorectal cancer.
The Company received the green light from the Data Safety Monitoring Board to move forward with the next treatment cohorts.

ON JANUARY 28, Scandion Oncology announced that the Company had submitted an amendment to the Danish Medicines Agency regarding the PANTAX study. The amendment is based on the learnings obtained from treating the first 12 patients in the CORIST study and will contribute to an optimization of the PANTAX clinical trials. The processing time for the amendment, on top of the current impact of the COVID-19 pandemic could delay the planned readout from the study into Q4, 2021.
Highlights after the end of the period

ON APRIL 21, Scandion Oncology announced that Dr. Richard L. Schilsky, a seasoned and highly profiled international leader, was appointed as member of Scandion Oncology’s clinical advisory board (CAB). Dr. Schilsky is the former CMO and Executive Vice President of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and a long-time faculty member of the University of Chicago.
The Q1 2021 Report is available on the Company’s website: www.scandiononcology.com.

Audiocast today, May 20 at 10:00 am CET
Scandion Oncology A/S will be hosting an audiocast on May 20 at 10:00 am CET to present results for Q1 2021 followed by a Q&A session. Representing the company will be President & CEO, Bo Rode Hansen and CFO, Carit Jacques Andersen.

Please login to the audiocast via View Source It is not required to dial in by phone to follow the presentation.

To listen and view the presentation and the Q&A, we recommend that you join via the webcast feature.

To ask questions, we recommend that you join via phone and you will find all the dial-in numbers via the link above.

For further information regarding Scandion Oncology, please contact:

This information is information that Scandion Oncology A/S is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, on May 20, 2021 CET at 8:30

Positive Primary Analysis from the Phase 2 OPTIC Study of Ponatinib (Iclusig®) in Chronic Phase-Chronic Myeloid Leukemia (CP-CML) to be Presented at the 2021 ASCO Annual Meeting

On May 20, 2021 Incyte (Nasdaq:INCY) reported that data from the primary analysis of the Phase 2 OPTIC (Optimizing Ponatinib Treatment In CML) trial will be presented during an oral session (Abstract #7000) at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, held virtually June 4-8, 2021 (Press release, Incyte, MAY 20, 2021, View Source [SID1234580364]). The OPTIC trial – an ongoing, randomized, open-label study prospectively evaluating response-based dosing regimens of ponatinib (Iclusig) over a range of three starting doses (45mg, 30mg, 15mg) followed by dose reduction to 15mg with the aim of optimizing efficacy and safety in patients with chronic-phase chronic myeloid leukemia (CP-CML) who are resistant to prior tyrosine kinase inhibitor (TKI) therapy – met its primary endpoint.

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The trial, sponsored by Takeda and co-funded by Incyte, evaluated treatment in patients with resistant disease, with and without mutations. The results show that the optimal benefit-risk profile for ponatinib in patients with CP-CML was achieved with a starting dose of 45mg/day, and upon response (achieving ≤1% BCR-ABL1IS), dose reduction to 15mg/day. The results also suggest a clinically-manageable safety profile for ponatinib. Data from the OPTIC interim analysis (cutoff date of July 2019), which evaluated 216 patients with a median follow-up of 21 months, were previously reported. The primary analysis (cutoff date of May 2020) evaluated 283 patients with a median follow-up of 32 months. All patients in the OPTIC trial were evaluable for the primary endpoint at the time of this analysis.

"The OPTIC primary analysis reinforces the positive response that is achievable with ponatinib in appropriate patients with CP-CML, and the ability that response-based regimens have to maximize efficacy, while maintaining a manageable safety profile," said Luca Marini, M.D., Regional Vice President, Head of European Medical Affairs, Incyte. "The OPTIC trial provides additional confirmation on the optimization of ponatinib dosing and reinforces its role as a meaningful treatment option for patients."

Key findings from the OPTIC primary analysis include:

The maximum rates of ≤1% BCR-ABL1IS at 12 months, the primary endpoint, were achieved in the 45mg/day starting dose cohort (44.1%), and 73.3% of patients in this cohort maintained response with the dose reduction to 15mg/day. The 30mg/day and 15mg/day starting dose cohorts also demonstrated benefit (29.0% and 23.1% ≤1% BCR-ABL1IS at 12 months, respectively), especially in patients with less-resistant disease and with a T315I mutation.
Positive survival outcomes were estimated in all three arms, with an 89.3% 36-month overall survival (OS) probability anticipated for the 45mg starting dose cohort and 73.3% progression-free survival (PFS) anticipated for the same cohort.
This indicates that the dose-reduction strategy did not impact OS regardless of prior second-generation TKI resistance or the presence of BCR-ABL1 mutations.
Rates of arterial occlusive events (AOEs) observed at the time of primary analysis (6% overall and 9.6% in the 45mg starting dose cohort) suggest a clinically manageable safety profile.
Safety data include:
Among all study participants (N=283), the most common treatment-emergent adverse events (TEAEs) Grade 3 or greater were thrombocytopenia (27%), neutropenia (17%) and anemia (7%).
Reported AOEs were 10%, 5% and 3% for the 45mg, 30mg, 15mg/day starting dose cohorts, respectively. Grade 3 or greater AOEs were 5%, 5% and 3% for the 45mg, 30mg and 15mg/day starting dose cohorts, respectively.
Reported serious AOEs were 4%, 4% and 3% for the 45mg, 30mg, 15mg/day starting dose cohorts, respectively. There were four deaths related to AEs (two sudden deaths and two pneumonia).
"As a physician, I am pleased by the results of the OPTIC trial evaluating patients with resistant CP-CML, who are in need of additional options to improve outcomes," said Dr. Gianantonio Rosti, M.D., Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori" (IRST S.r.l.) Istituto di Ricovero e Cura a Carattere Scientifico. "It is encouraging to see the positive benefit-risk profile that can be achieved with ponatinib through a response-based dosing regimen, therefore providing efficacy while also managing the risk for arterial occlusive events."

Incyte has an exclusive license from Takeda Pharmaceuticals International AG to commercialize ponatinib in the European Union and 29 other countries, including Switzerland, UK, Norway, Turkey, Israel and Russia. Iclusig is marketed in the U.S. by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited.

About the OPTIC Trial

OPTIC (Optimizing Ponatinib Treatment In CML) is a randomized, dose-ranging Phase 2 trial designed to evaluate three starting doses (15mg, 30mg, 45mg) of ponatinib in patients with resistant chronic-phase chronic myeloid leukemia (CP-CML) or who had documented history of presence of T315I mutation after receiving any number of prior tyrosine kinase inhibitors (TKIs). Dose reduction at response occurred per study protocol. The trial is expected to inform the optimal use of ponatinib in these patients. The primary endpoint of the trial is achieving ≤1% BCR-ABL1 at 12 months. A total of 283 patients were enrolled at clinical sites around the world. Dose reduction at response occurred per study protocol.

For more information about the OPTIC study, please visit View Source

About CML

CML – a rare malignancy – is one of four main types of leukemia; it is a result of a genetic mutation that takes place in early, immature versions of myeloid cells, which form red blood cells, platelets and most types of white blood cells. Subsequently, an abnormal gene called BCR-ABL1 forms, turning the damaged cell into a CML cell. CML typically progresses slowly, but it can change into a fast-growing acute leukemia that is hard to treat.

About Ponatinib (Iclusig) Tablets

Ponatinib (Iclusig) targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Click here to view the Iclusig EU Summary of Medicinal Product Characteristics.

Incyte has an exclusive license from Takeda Pharmaceuticals International AG to commercialize ponatinib in the European Union and 29 other countries, including Switzerland, UK, Norway, Turkey, Israel and Russia. Iclusig is marketed in the U.S. by Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited