On October 3, 2019 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that it has opened for enrollment a Phase 1b expansion cohort of its Phase 1/2 clinical trial of cirmtuzumab, a ROR1-targeted monoclonal antibody, combined with ibrutinib, in patients with mantle cell lymphoma (MCL) (Press release, Oncternal Therapeutics, OCT 3, 2019, View Source [SID1234540042]). The decision to open an expansion cohort in MCL of the ongoing Phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial was based on favorable interim results from the dose-finding cohort of the trial, including that the combination was well-tolerated and that complete responses were observed in two heavily pre-treated patients who had received and failed multiple chemotherapy regimens and an autologous transplant, as well as either an allotransplant or CAR-T therapy, prior to participating in this clinical trial.

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In June, the Company presented interim data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, including the preliminary results from the first six patients with MCL treated in the CIRLL clinical trial. One patient with MCL, who had relapsed following an allogeneic stem cell transplant, experienced a confirmed complete response (CR) after three months of cirmtuzumab plus ibrutinib treatment, including complete resolution of a large mediastinal mass. This CR appears to be sustained and has been confirmed to be ongoing after completing 12 months of cirmtuzumab plus ibrutinib treatment. Following ASCO (Free ASCO Whitepaper), a second confirmed CR occurred in a patient who had progressive disease after failing several different chemotherapy regimens, autologous transplant and CAR-T therapy. Additional data from this clinical trial will be presented at a future medical conference.

"It is encouraging to see that the drug has been well tolerated as well as the early signal of efficacy of cirmtuzumab with ibrutinib in MCL, particularly the rapid and durable complete responses of the heavily pre-treated patients after three months of therapy, which is an unusually fast response in this patient population," said Hun Lee, M.D., Assistant Professor of Medicine in the Department of Lymphoma & Myeloma at the University of Texas MD Anderson Cancer Center, who is an investigator on the CIRLL clinical trial.

The CIRLL clinical trial is supported by a grant from the California Institute for Regenerative Medicine (CIRM) and is being conducted in collaboration with the University of California San Diego (UC San Diego).

"We are pleased to be opening the expansion cohort portion of the CIRLL clinical trial for patients with MCL, and continue to be encouraged by the interim results from this study for both patients with MCL and patients with chronic lymphocytic leukemia, for whom a randomized Phase 2 portion of the trial was opened in August," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO.

About the CIRLL Clinical Trial

The CIRLL clinical trial (CIRM-0001) is a Phase 1/2 trial evaluating cirmtuzumab in combination with ibrutinib in separate groups of patients with chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL). Part 1 of the clinical trial was a Phase 1 dose-finding portion designed to determine the Phase 2 dose, or recommended dosing regimen (RDR). Part 2 is a Phase 1b expansion cohort to confirm the RDR. Additional information about the CIRM-0001 clinical trial and other clinical trials of cirmtuzumab may be accessed at ClinicalTrials.gov.

About Cirmtuzumab

Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of CLL and MCL, in a collaboration with the University of California San Diego School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, an investigator-initiated Phase 1 clinical trial of cirmtuzumab in combination with paclitaxel for women with metastatic breast cancer is being conducted at the UC San Diego School of Medicine. CIRM has also provided funding to support development programs for cirmtuzumab and a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors.

ROR1 is a potentially attractive target for cancer therapy because it is an oncofetal antigen – a protein that confers a survival and fitness advantage when reactivated and expressed by tumor cells. When expressed by hematologic malignancies such as CLL and MCL, ROR1 acts as a receptor for the tumor growth factor Wnt5a. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to inhibiting Wnt5a activation, specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. Cirmtuzumab is in clinical development and has not been approved by the U.S. Food and Drug Administration for any indication.

On October 3, 2019 Biodesix, Inc., reported it has been issued U.S. Patent Number 10,422,729, which covers the Biodesix Collection Device (BCD) (Press release, Biodesix, OCT 3, 2019, View Source [SID1234540040]). The new device offers improved ease of use in blood sample collection for diagnostic testing, compared to current standard methods.

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"This device is intuitive and very easy to use," remarked Susan Garwood, M.D., Pulmonary and Advanced Bronchoscopy Lung Cancer Specialist with Centennial Medical Center in Nashville, part of the HCA TriStar division. "The Biodesix Collection Device enables the opportunity for a fast, simple collection process. Enhancing patient comfort while retaining accuracy is of top concern for all professionals drawing patient blood samples."

The BCD, currently in use with the Biodesix Nodify XL2 test, replaces the need for processing samples used for proteomic testing on-site and shipping blood tubes, which often requires cold-chain logistics during transit to the testing laboratory. This reduces the need for processing equipment such as centrifuges in the clinic, improves sample stability during transit and eliminates the need for single-use, bulky shipping containers containing materials such as Styrofoam that are not only costly but also have a negative environmental impact.

"The BCD is designed for exceptional laboratory test performance and surpasses similar collection methods by combining multiple sample processing steps, including specimen collection and reproducible sample separation with ambient shipping. Specimen stability has been demonstrated with analytic measures of proteins using highly sensitive mass spectrometry methods," said Gary Pestano, Ph.D., chief development officer at Biodesix.

"Our commitment to innovation continues to improve the customer experience," commented Scott Hutton, chief operating officer at Biodesix. "We are as focused as ever on supporting healthcare professionals and their patients by providing increasingly valuable information to physicians using a noninvasive blood collection. The new BCD significantly reduces cost and environmental impact over current sample collection practices, and we are excited to not only use this for our own tests but to make it available for broad use across the industry."

Biodesix is planning to use the new BCD in all current and future proteomic testing and also make the device available for purchase by labs and diagnostic companies wishing to use this novel technology for their own sample processing and testing. To see how the BCD is used to collect specimens for the Nodify XL2 test visit this link.

On October 3, 2019 Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, reported a poster highlighting new preclinical duvelisib (COPIKTRA) data that will be presented at the 5th International Conference on New Concepts in Lymphoid Malignancies, which is hosted by the European School of Haematology (ESH), and is taking place October 3-5, 2019, in Estoril, Portugal (Press release, Verastem, OCT 3, 2019, View Source [SID1234540039]). The poster features preclinical research that compared duvelisib with idelalisib in preclinical models of mantle cell lymphoma (MCL). COPIKTRA is not approved for use in MCL, diffuse large B-cell lymphoma (DLBCL), or marginal zone lymphoma (MZL).

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"These data highlight MCL as a B-cell malignancy which expresses both PI3K-delta and PI3K-gamma in the malignant B cells, in addition to the role of PI3K-gamma which has already been established in cells of the supportive tumor microenvironment," said Jonathan Pachter, PhD, Chief Scientific Officer of Verastem Oncology. "Accordingly, the data show superior anti-cancer activity of the dual PI3K-delta/gamma inhibitor duvelisib compared to the PI3K-delta inhibitor idelalisib in these preclinical MCL models. At Verastem Oncology, we are working to expand into additional lymphoid malignancy indications and these data provide additional support for the future study of duvelisib through clinical trials in patients with MCL."

Duvelisib in Preclinical Models of MCL

MCL is a rare, aggressive B-cell non-Hodgkin lymphoma (NHL) that is incurable. The purpose of this research was to gain a functional understanding of the distinctive features associated with the malignant cells in MCL, with the goal of identifying potential new treatment strategies. PI3K-delta is known to be equally expressed in all B-cell malignancies and this research revealed that primary MCL cells show elevated expression of PI3K-gamma relative to primary cells from other B-cell malignancies, including chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) and marginal zone lymphoma (MZL). These preliminary data suggest that high levels of PI3K-gamma in MCL cells correlate with poorer disease outcomes.

The researchers then examined the effects of inhibition of specific PI3K isoforms, including PI3K-alpha, PI3K-delta (idelalisib), PI3K-gamma, and dual PI3K-delta/gamma (duvelisib), on the migration and proliferation of malignant lymphocytes from patients with MCL. These preclinical models showed that inhibition of both PI3K-delta and gamma reduced the proliferation of MCL cells whereas inhibition of PI3K-delta alone had no effect. The functional role of PI3K-delta and gamma in MCL cells was further differentiated from that of PI3K-delta alone. PI3K-delta and gamma more effectively inhibited CCL21-induced migration of MCL cell lines and primary MCL cells than did PI3K-gamma inhibition alone. Inhibition of PI3K-delta or PI3K-alpha did not affect CCL21-induced migration of MCL cells. These data suggest that when PI3K-gamma is aberrantly expressed by MCL cells, these cells become reliant on this PI3K isoform for chemokine-induced migration and tissue residency. Thus, duvelisib may have potential in MCL therapy by restricting entry, retention, and proliferation of the malignant cells within the mantle zone.

Details for the ESH 2019 poster presentation are as follows:

Title: Aberrantly Expressed PI3Kγ Contributes to Cell Proliferation and Co-Operates with PI3δ to Mediate CCL21-Induced Migration in Mantle-Cell Lymphoma: Therapeutic Implications for Duvelisib
Lead author: Kathy Till, University of Liverpool
Date and time: Thursday, October 3, 2019, beginning at 7am through the end of the conference
Location: Poster Area

A PDF copy of this poster presentation will be available here after the meeting.

SELECT IMPORTANT SAFETY INFORMATION

This does not include all information needed to use COPIKTRA (duvelisib) safely and effectively. See full Prescribing Information.

WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS

See full Prescribing Information for complete boxed warning

Fatal and/or serious infections occurred in 31% of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% of COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.
WARNINGS AND PRECAUTIONS

Hepatotoxicity: Monitor hepatic function.
Neutropenia: Monitor blood counts.
Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception.
ADVERSE REACTIONS

The most common adverse reactions (≥20%) are diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.

To report Adverse Reactions, contact FDA at 1-800-FDA-1088 (1-800-332-1088) or www.fda.gov/medwatch and Verastem Oncology at 1-877-7RXVSTM (1-877-779-8786).

DRUG INTERACTIONS

CYP3A inducers: Avoid co-administration with strong CYP3A inducers.
CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15 mg twice daily when co-administered with strong CYP3A4 inhibitors.
CYP3A substrates: Monitor for signs of toxicities when co-administering COPIKTRA with sensitive CYP3A substrates.
USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed.

Please see accompanying full Prescribing Information, including Boxed Warning.

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status, and is being investigated in combination with other agents through investigator-sponsored studies.4 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

On October 3, 2019 AngioDynamics, Inc. (NASDAQ: ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, peripheral vascular disease, and oncology, reported financial results for the first quarter of fiscal year 2020, which ended August 31, 2019 (Press release, AngioDynamics, OCT 3, 2019, View Source [SID1234540038]).

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"Our top-line and gross-margin performance in the quarter was in line with our expectations," commented Jim Clemmer, President and Chief Executive Officer of AngioDynamics, Inc. "We continue to build momentum and remain focused on driving growth by creating a suite of disruptive and differentiated technology. The divestiture of the NAMIC Fluid Management business allowed us to begin the year with a strong balance sheet, enabling strategic acquisitions like Eximo Medical, Ltd., which we announced this morning. Additionally, we continue to make steady progress with site initiation in our NanoKnife DIRECT study for pancreatic cancer."

First Quarter 2020 Financial Results

Net sales for the first quarter of fiscal 2020 were $66.0 million, an increase of 3.3%, compared to $63.9 million a year ago and representing a decline of 1.0% organically. Excluding the impact of Asclera, sales of which were discontinued during fiscal year 2019, net sales grew 5.6% year over year and grew 1.3% organically. Foreign currency translation did not have a significant impact on the Company’s sales in the quarter.

Oncology net sales were $14.0 million, an increase of 20.9% from $11.6 million a year ago, led by sales of the Company’s BioSentry Tract Sealant System, the Alatus and IsoLoc balloon products, NanoKnife, and Solero.
Vascular Interventions and Therapies ("VIT") net sales were $28.9 million, an increase of 1.1%, compared to $28.6 million a year ago. Excluding last year’s Asclera Sales of $1.4 million in the first quarter, VIT grew 6.4%, as strong growth in sales of the Company’s AngioVac and Thrombolytic product line offerings was partially offset by a decline in the overall Core VIT business.
Vascular Access net sales were $23.2 million, a decrease of 2.7% from $23.8 million a year ago, as lower sales of Ports and PICCs negatively offset growth in the Midlines and Dialysis businesses.
U.S. net sales in the first quarter of fiscal 2020 were $52.9 million, an increase of 2.8% from $51.5 million a year ago, and International net sales were $13.1 million, an increase of 5.3% from $12.4 million a year ago.

Gross margin for the first quarter of fiscal 2020 was 57.9%, an increase of 170 basis points compared to the first quarter of fiscal 2019, driven primarily by productivity and supply chain improvements as well as positive product mix.

The Company recorded a net loss from continuing operations of $1.3 million, or a loss of $0.03 per share, in the first quarter of fiscal 2020. This compares to a net loss from continuing operations of approximately $5.7 million, or a loss of $0.15 per share, a year ago.

Excluding the items shown in the non-GAAP reconciliation table below, adjusted net income for the first quarter of fiscal 2020 was $3.2 million, or $0.08 per share, compared to adjusted net income of $0.7 million, or $0.02 per share, in the first quarter of fiscal 2019.

Adjusted EBITDAS in the first quarter of fiscal 2020, excluding the items shown in the reconciliation table below, was $7.3 million, compared to $5.4 million in the first quarter of fiscal 2019.

In the first quarter of fiscal 2020, the Company used $6.5 million in operating cash flow and had capital expenditures of $1.4 million. As of August 31, 2019, the Company had $83.6 million in cash and cash equivalents and no debt outstanding.

Fiscal Year 2020 Financial Guidance

The Company continues to expect fiscal year 2020 net sales to be in the range of $280 to $286 million and gross margin to be in the range of 58% to 59%, inclusive of the Eximo acquisition.

Separately, the Company is updating its expectations for full-year adjusted earnings per share to account for investments related to the full-market launch of the products acquired from Eximo, anticipated in the second half of fiscal year 2020, and now expects adjusted earnings per share in the range of $0.10 to $0.15.

Conference Call

The Company’s management will host a conference call today at 8:00 a.m. ET to discuss its fiscal 2020 first quarter results.

To participate in the conference call, dial 1-877-407-0784 (domestic) or +1-201-689-8560 (international) and refer to the passcode 13694372.

This conference call will also be webcast and can be accessed from the "Investors" section of the AngioDynamics website at www.angiodynamics.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A recording of the call will also be available from 11:00 a.m. ET on Thursday, October 3, 2019, until 11:59 p.m. ET on Thursday, October 10, 2019. To hear this recording, dial 1-844-512-2921 (domestic) or +1-412-317-6671 (international) and enter the passcode 13694372.

On October 3, 2019 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing potential new therapies for cancer, reported updated data from the ongoing Phase 1b study of investigational ME-401, a selective oral inhibitor of PI3K delta (Press release, MEI Pharma, OCT 3, 2019, View Source2019-10-03-MEI-Pharma-Announces-Updated-Clinical-Data-from-ME-401-Phase-1b-Study-in-Patients-with-Indolent-B-cell-Malignancies" target="_blank" title="View Source2019-10-03-MEI-Pharma-Announces-Updated-Clinical-Data-from-ME-401-Phase-1b-Study-in-Patients-with-Indolent-B-cell-Malignancies" rel="nofollow">View Source [SID1234540036]). These new data will be presented at MEI’s Investor and Analyst Event being held tomorrow, October 4, 2019, at 8:00 am ET.

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Highlights of the ME-401 updated data include:

Overall response rates of 78% in relapsed or refractory (r/r) follicular lymphoma (FL) and 89% in r/r chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL).
Rates of Grade 3 adverse events of special interest related to ME-401 exposure were observed in <10% of patients dosed on an intermittent schedule (IS).
Median duration of response not yet reached in patients with FL or CLL/SLL on the IS regimen. Median follow-up for FL and CLL/SLL patients is 9.2 months (range 3.4-20.7 months) and 7.4 months (range 2.6-14.7 months), respectively.
At the Investor and Analyst event MEI will also review progress across the pipeline of its four clinical-stage oncology candidates with a focus on voruciclib, a cyclin-dependent kinase (CDK) inhibitor with potent CDK9 inhibition. Voruciclib is in a Phase 1b study in patients with r/r B-cell malignancies or Acute Myeloid Leukemia (AML) after failure of prior standard therapies. In addition, we plan to evaluate voruciclib in combination with venetoclax (marketed as Venclexta), a B-cell lymphoma (Bcl) 2 inhibitor, in patients with r/r AML.

"ME-401 continues to exceed expectations in the Phase 1b study; the overall response rate remains high at 81% with 73 evaluable FL and CLL/SLL patients being followed for treatment," Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "Currently, our primary focus is the ME-401 Phase 2 TIDAL study evaluating patients with relapsed or refractory follicular lymphoma, which may support an accelerated approval of a marketing application with FDA, as well as the continuing exploration of various combination regimens with ME-401."

Dr. Gold continued: "More broadly, MEI has a strong foundation to build value through advancing our pipeline, evaluating drug combination opportunities across the B-cell malignancy landscape, and continuing to create and explore strategic opportunities to most effectively leverage the potential of the pipeline. Each of our four clinical-stage oncology candidates is well positioned for continued clinical development."

Investor and Analyst Event Information

Date: Friday, October 4, 2019
Time: 8 am -11 am ET

The investor event will feature presentations from the following MEI Pharma executives:

Dan Gold, Ph.D., President and Chief Executive Officer, MEI Pharma.

Robert Mass, M.D., Chief Medical Officer, MEI Pharma.

Richard Ghalie, M.D., Senior Vice President, Clinical Development, MEI Pharma.
The event will also feature presentations by the following guest speakers:

Lewis C. Cantley, Ph.D., Meyer Director of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medical College, Professor of Cancer Biology in Medicine.

Nishitha M. Reddy, M.D., M.B.B.S., Associate Professor of Medicine at Vanderbilt University Medical Center.

Matthew J. Matasar, M.D., Medical Director, Memorial Sloan Kettering Bergen.
You can access the live webcast under the investor relations section of MEI’s website on the "Events and Presentation" page at: www.meipharma.com. A replay of the webcast will be archived for at least 30 days after the conclusion of the live event.

The full Investor and Analyst Event presentation will be available on the home page of the Investor Relations section of MEI Pharma’s website at: View Source

About MEI’s Clinical-Stage Oncology Pipeline

About ME-401
ME-401 is an oral, once-daily, selective phosphatidylinositol 3-kinase (PI3K) delta inhibitor in clinical development for the treatment of B-cell malignancies. MEI owns worldwide rights in all geographies except Japan, which we licensed to Kyowa Kirin Company (formerly "Kyowa Hakko Kirin Co., Ltd.") in 2018.

MEI is conducting two ongoing studies evaluating ME-401. The first is a Phase 2 clinical trial evaluating ME-401 as a monotherapy for the treatment of adults with relapsed or refractory follicular lymphoma ("FL") after failure of at least two prior systemic therapies including chemotherapy and an anti-CD20 antibody. Subject to the results, upon completion of the Phase 2 clinical trial, we are planning a submission with the FDA to support an accelerated approval of a marketing application under 21 CFR Part 314.500, Subpart H. The second is a multi-arm, open-label, Phase 1b dose escalation and expansion trial evaluating ME-401 as a monotherapy and in combination with other therapies or investigational agents in patients with relapsed or refractory B-cell malignancies.

About Voruciclib
Voruciclib is an orally administered cyclin-dependent kinase (CDK) inhibitor differentiated by its potent in vitro inhibition of CDK9, in addition to CDK6, 4 and 1. Voruciclib is currently being evaluated in a Phase 1b dose ranging trial in patients with relapsed and/or refractory B-cell malignancies or acute myeloid leukemia (AML) after failure of prior standard therapies.

About ME-344
ME-344 is our novel and tumor selective, isoflavone-derived mitochondrial inhibitor drug candidate. It directly targets the OXPHOS complex 1, a pathway involved in ATP production in the mitochondria. ME-344 was recently studied in an investigator-initiated, multi-center, randomized clinical trial in combination with the vascular endothelial growth factor (VEGF) inhibitor bevacizumab (marketed as Avastin ) in a total of 42 patients with HER2 negative breast cancer. The data established significant biologic activity in the ME-344 treatment group as measured by Ki67 reductions (a measure of cell proliferation that is highly correlated with tumor response).

About Pracinostat
Pracinostat is an oral histone deacetylase (HDAC) inhibitor being evaluated in a pivotal Phase 3 global registration clinical trial for the treatment of adults with newly diagnosed AML who are unfit to receive intensive chemotherapy. Pracinostat is also being evaluated in a Phase 2 trial in patients with high or very high-risk myelodysplastic syndrome (MDS). In August 2016, we entered into an exclusive worldwide license, development, manufacturing and commercialization agreement with Helsinn Healthcare SA, a Swiss pharmaceutical corporation for pracinostat in AML, MDS and other potential indications.