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Agios Reports Business Highlights and Second Quarter 2019 Financial Results

On August 1, 2019 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the second quarter ended June 30, 2019 (Press release, Agios Pharmaceuticals, AUG 1, 2019, View Source [SID1234537982]).

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"In the second quarter we demonstrated our ability to execute across all areas of our business. Our commercial team continues to deliver on the AML launch for TIBSOVO and prepare for our first potential solid tumor launch on the heels of the positive ClarIDHy study in cholangiocarcinoma," said Jackie Fouse, Ph.D., chief executive officer at Agios. "On the clinical side, we continued to broaden our IDH program into earlier lines of AML therapy and expand our PKR programs into new disease areas while enrolling the PK deficiency pivotal studies. In addition, we completed dose escalation in our AG-270 Phase 1 trial and are on track to initiate the next phase of development. The great progress we made during the quarter keeps us on track to deliver on our remaining 2019 milestones and drive further value across our portfolio."

SECOND QUARTER 2019 HIGHLIGHTS & RECENT PROGRESS

Received approval from the U.S. Food and Drug Administration (FDA) on May 2, 2019 for single agent TIBSOVO for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) with an IDH1 mutation who are ≥ 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy.
Announced the Phase 3 ClarIDHy trial of TIBSOVO in advanced previously treated cholangiocarcinoma patients with an IDH1 mutation met its primary endpoint, demonstrating improvement in progression-free survival by independent radiology review compared with patients who received placebo.
Presented updated data from the Phase 1 studies of TIBSOVO in frontline AML, both as a monotherapy and in combination with azacitidine, and the first data from the perioperative study of TIBSOVO and vorasidenib in glioma at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data presentations can be found here.
Began dosing patients in the Phase 1 dose-escalation trial of AG-636, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH), in advanced lymphoma.
Completed the single agent dose-escalation portion of the ongoing Phase 1 study of AG-270 in methylthioadenosine phosphorylase (MTAP)-deleted tumors.
Appointed Orlando Oliveira to the role of senior vice president and general manager, international. Mr. Oliveira will be responsible for building and leading the company’s operations outside of the U.S. in support of the expected launch of TIBSOVO in Europe and potentially other select markets.
KEY UPCOMING MILESTONES

The company plans to achieve the following key milestones in the remainder of 2019:

Oncology:

Submit a supplemental new drug application to the FDA for TIBSOVO for advanced previously treated IDH1 mutant cholangiocarcinoma by year-end.
Initiate a registration-enabling Phase 3 study of vorasidenib in low-grade glioma with an IDH mutation by year-end.
Initiate expansion phase for the Phase 1 study of AG-270 in MTAP-deleted tumors, including two combination arms with AG-270 and taxanes in non-small cell lung cancer and pancreatic ductal adenocarcinoma, in the third quarter.
Rare Genetic Diseases:

Complete enrollment in two global pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end 2019:
ACTIVATE-T: A single-arm trial of up to 40 regularly transfused patients
ACTIVATE: A 1:1 randomized, placebo-controlled trial of 80 patients who do not receive regular transfusions
Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
ANTICIPATED 2019 DATA PRESENTATIONS

Full data from the Phase 3 ClarIDHy study of TIBSOVO in IDH1 mutant advanced previously treated cholangiocarcinoma have been submitted for presentation at the European Society for Medical Oncology Congress taking place in Barcelona, Spain from September 27-October 1, 2019.
Data from the single agent dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with MTAP-deleted tumors have been submitted to the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) taking place in Boston from October 26-30, 2019.
Data from IDH and PKR programs have been submitted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place in Orlando, Fla. from December 7-10, 2019, including new data from the extension phase of the Phase 2 DRIVE PK study of mitapivat in adults with PK deficiency and important translational data from the Phase 1 combination study of TIBSOVO and azacitidine in frontline AML.
SECOND QUARTER 2019 FINANCIAL RESULTS

Revenue: Total revenue for the second quarter of 2019 was $26.2 million, which includes $13.7 million of net product revenue from U.S. sales of TIBSOVO, $9.0 million in collaboration revenue and $2.7 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to revenue of $40.4 million for the second quarter of 2018, which included recognition of a $15 million milestone from Celgene related to Celgene’s filing of an MAA to the EMA for IDHIFA and $12.4 million from the signing of the CStone collaboration.

Cost of Sales: We began U.S. sales of TIBSOVO in the third quarter of 2018. Cost of sales were $0.3 million for the second quarter of 2019.

Research and Development (R&D) Expenses: R&D expenses were $107.4 million for the second quarter of 2019 compared to $86.7 million for the second quarter of 2018. The increase in R&D expense was primarily attributable to vorasidenib Phase 3 low grade glioma trial start-up costs, the mitapivat pivotal program in PK deficiency and Phase 2 study in thalassemia, and clinical trial activity related to the ongoing Phase 1 trials for AG-270 and AG-636.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $32.4 million for the second quarter of 2019 compared to $26.6 million for the second quarter of 2018. The increase in SG&A expense was primarily attributable to costs to support commercialization of TIBSOVO and personnel costs related to increased headcount.

Net Loss: Net loss was $109.9 million for the second quarter of 2019 compared to $68.7 million for the second quarter of 2018.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2019 were $624.0 million compared to $805.4 million as of December 31, 2018. The net decrease of $181.4 million in cash position was primarily driven by net expenditures to fund operations, including a onetime cash expense of $19.2 million for bonus payouts during the first quarter. The company expects that its cash, cash equivalents and marketable securities as of June 30, 2019, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2019 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 9319039. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

Xynomic Received China Approval to Start 2 Pivotal Lymphoma Clinical Trials

On August 1, 2019 Xynomic Pharmaceuticals Holdings, Inc. ("Xynomic", stock ticker: XYNO), a clinical stage US-China oncology drug development company, reported that Xynomic has received approval from China’s National Medical Products Administration ("NMPA") to start two pivotal clinical trials in China (Press release, Xynomic Pharmaceuticals, AUG 1, 2019, View Source [SID1234537980]). In these two trials, Xynomic will test its lead drug candidate abexinostat (as a single agent) as a third-line treatment of diffuse large B-cell lymphoma ("DLBCL") and as a third-line treatment of follicular lymphoma ("FL"), respectively.

According to China’s National Cancer Center and China’s National Health Commission, DLBCL is the most common aggressive non-Hodgkin’s lymphoma (NHL) subtype with an estimated 50,000 new cases per year in China and FL is the most common indolent NHL subtype with an estimated 10,000 new cases per year in China. DLBCL and FL combined represent approximately 45-60% of China’s NHL market.

According to China’s Center for Drug Evaluation’s database, Xynomic’s abexinostat is the only drug candidate currently under clinical development as third-line treatment of DLBCL in China. Xynomic plans to complete patient enrollment for these two trials in 12 months or sooner. These trials will be conducted in about 24 leading hematological cancer hospitals across China.

"We are truly excited to receive the approval from NMPA of China, world’s second largest market for oncology drugs. Currently in China there are virtually no approved treatment options for DLBCL and FL patients once the patients’ disease progress after R-CHOP treatment. Our abexinostat has demonstrated both safety and efficacy in these settings in prior Phase 1/2 trials. We look forward to completing these trials expeditiously and have also started preparation for commercial launch," said Y. Mark Xu, Xynomic’s Chairman and CEO.

ImCheck Therapeutics Awarded €2 Million From Bpifrance to Advance its First-in-class Immunotherapy Program Into the Clinic

On August 1, 2019 ImCheck Therapeutics, a biotech company on the cutting edge of the next wave of immunotherapies against cancer and autoimmune diseases, reported the award of €2 million from Bpifrance to contribute to the development of its most advanced novel cancer immunotherapy program. With this additional financing, ImCheck has obtained a total of €3.7 million in non-dilutive funding since its €20 million Series A in 2017 (Press release, ImCheck Therapeutics, AUG 1, 2019, View Source [SID1234537964]).

The financing is part of the ‘Deeptech’ fund from Bpifrance and represents both grants and a reimbursement loan. The support will be dedicated to conducting initial clinical studies of ICT01, a novel therapeutic anti-BTN3A activating monoclonal antibody designed to promote an anti-tumor response by γ9δ2 T cells. To date, ICT01 has demonstrated activity in a range of in vitro and in vivo tumor models.

ImCheck Therapeutics plans on completing all preclinical confirmatory studies before the end of the year to enable the start of clinical development of ICT01 during the first quarter of 2020. The first study will be a Phase I dose escalation trial using the anti-BTN3A antibody as a single agent in solid and hematologic malignancies, followed by a combination study with anti-PD-1/L1 antibodies.

"With the Deeptech program, Bpifrance supports companies in the research and development phase of a breakthrough innovation closely linked to public/private research with high barriers to entry and highly differentiated, before its industrial and commercial launch. Bpifrance supports several companies in the highly-innovative and highly-competitive field of immuno-oncology. We view ImCheck’s projects as unique and very well-positioned and we are pleased to continue to support ICT01’s development and its entry in the clinic. This €2m grant follows a first €1m funding awarded by Bpifrance in July 2017 as part of our Award for Innovation Development program," said Françoise Marchand, Project Innovation Officer at Bpifrance.

"This new financial support from Bpifrance is a validation of the potential of our pipeline and recognition of the progress we have achieved in rapidly advancing our first antibody candidate toward clinical evaluation. We remain on target to begin the trial at the start of 2020. Benefiting from the "Deeptech" fund is also an important step for ImCheck as it underlines our innovative position in immuno-oncology," concluded Pierre d’Epenoux, CEO of ImCheck Therapeutics.

Chimerix Announces Exclusive Worldwide License of Phase 3 Ready CX-01 for Development in Acute Myeloid Leukemia

On July 31, 2019 Chimerix (NASDAQ: CMRX), reported the completion of an exclusive worldwide license of CX-01 from Cantex Pharmaceuticals, Inc.Chimerix intends to move quickly into Phase 3 development of CX-01 for the treatment of Acute Myeloid Leukemia (AML) in the first-line setting (Press release, Chimerix, JUL 31, 2019, View Source [SID1234568985]). CX-01 has received Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration for the treatment of AML.

"We are pleased to have made such rapid progress in repositioning the company and transforming our pipeline with this important cancer therapy. We are excited to advance this promising product candidate in AML as it has shown compelling activity across multiple endpoints in first-line patients as opposed to later lines of therapy where most of the recent advances in this disease area have occurred. With more than 21,000 new cases of AML diagnosed annually in the U.S. alone and a five-year survival rate of less than 30%, the patient need is clear. CX-01’s mechanism of action, targeting multiple proteins involved in protecting and supporting the growth of cancer cells, provides opportunities for potential development across a range of hematologic malignancies," stated Mike Sherman, Chief Executive Officer of Chimerix. "This transaction exemplifies our commitment to pursuing and accelerating programs where we can quickly address unmet patient needs with a meaningful clinical benefit."

"While several new agents have been recently approved for AML, a backbone of cytotoxic chemotherapy continues to be necessary for treatment with curative intent. If our results are confirmed, combining CX-01 with chemotherapy has the potential to have a significant impact on the outcomes of patients suffering from one of the most challenging hematologic malignancies," said Paul Shami, MD, clinical investigator at Huntsman Cancer Institute and Professor of Medicine at the University of Utah.

CX-01 is a new chemical entity derived from unfractionated heparin with very low anticoagulant activity. CX-01 targets key protein pathways important for AML blast cell migration to the bone marrow and retention of these cells in the marrow where they are protected from chemotherapy. CX-01 also binds with proteins involved in chemotherapy resistance and the delay in platelet recovery after chemotherapy. Together, these activities are understood to sensitize AML blasts to chemotherapy and improve clinical responses. These mechanisms of action support the potential for development in myelodysplastic syndrome, multiple myeloma, and lymphomas.

In a recently completed Phase II study, 75 patients over 60 years of age with newly diagnosed AML were randomized 1:1:1 to one of two doses of CX-01 (0.125 mg/kg/hr or 0.250 mg/kg/hr) plus standard 7+3 chemotherapy (7 days of cytarabine, 3 days of anthracycline) or to the control arm of standard 7+3 chemotherapy alone. Data presented at the 2019 ASCO (Free ASCO Whitepaper) conference indicated an advantage across multiple endpoints for patients treated with 0.25 mg/kg/hr CX-01. In the evaluable patient population, results of the 0.25 mg/kg/hr CX-01 arm compared favorably to the control arm: complete response rate (complete response or complete response without complete hematologic recovery) of 89% vs. 58% (p=0.03), median event-free survival of 23.4 months vs. 9.0 months (p=0.011), and median overall survival which had not yet been reached in the CX-01 arm vs. 11.2 months (p=0.042). These data were consistent with a single arm pilot study of first line therapy in patients with AML (n=12), including a complete response rate of 92%.

CX-01 was well tolerated with adverse events similar across all treatment arms. The most common serious adverse event was febrile neutropenia with three cases in each CX-01 treatment group and one case in the control group.

Stephen Marcus, M.D., CEO of Cantex, stated, "We are very pleased to be partnering with Chimerix and their world-class scientists. We believe that Chimerix management’s track record in developing novel cancer therapeutics makes Chimerix the perfect partner to aggressively advance the development of CX-01 for the treatment of AML and other hematologic malignancies."

Transaction Terms

Under the terms of the agreement, Chimerix has exclusive worldwide rights to develop and commercialize CX-01. Chimerix will make an upfront payment of $30 million to Cantex. In addition, Chimerix has issued 10 million shares of Chimerix common stock to Cantex. Cantex is eligible for regulatory and commercial milestones of up to $587.5 million, and tiered royalties starting at 10%.

Conference Call
Chimerix management will host a conference call today at 8:30AM ET. To participate, please dial:
US and Canada: (877) 354-4056
International: (678) 809-1043
Conference ID: 9558159
A live, listen-only webcast of the conference call may also be accessed by visiting the Investors section of the Chimerix website, www.chimerix.com.

Kaleido Biosciences to Present at the 39th Annual Canaccord Genuity Growth Conference

On July 31, 2019 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a chemistry-driven approach to leveraging the potential of the microbiome organ to treat disease and improve human health, reported that management will participate in a fireside chat at the 39th Annual Canaccord Genuity Growth Conference in Boston, MA on Thursday, August 8, 2019 at 1:00 p.m. ET (Press release, Kaleido Biosciences, JUL 31, 2019, View Source [SID1234540066]).

A live audio webcast will be available through the Investors & Media section of Kaleido’s website at View Source An archived replay will be accessible for 90 days following the event.