On February 13, 2020 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that polatuzumab vedotin in combination with bendamustine and rituximab (hereafter, BR therapy) achieved the primary endpoint of complete response rate (CRR) by PET-CT at the timing of Primary Response Assessment (PRA) in the Japanese Phase II study (JO40762/P-DRIVE study) (Press release, Chugai, FEB 13, 2020, View Source [SID1234554250]). P-DRIVE is an open label, single-arm study to evaluate the combination therapy of polatuzumab vedotin with BR therapy as a treatment for people with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Combination of polatuzumab vedotin and BR therapy observed no new safety signals in the study compared with the previous studies for polatuzumab vedotin.

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"We are very pleased that polatuzumab vedotin in combination with BR therapy showed efficacy in treating relapsed or refractory DLBCL patients," said Chugai’s Executive Vice President, Co-Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. "About 40% of patients experience relapse of the disease after standard therapy and subsequent treatment options are limited. Chugai is committed to file for approval based on these results to provide patients with this potential treatment option as early as possible."

The Ministry of Health, Labour and Welfare granted the Orphan Drug designation for polatuzumab vedotin in DLBCL in November, 2019. Polatuzumab vedotin was granted accelerated approval in the US in June, 2019 and conditional marketing authorization in EU in January, 2020 respectively. In addition to the P-DRIVE study, the global phase III POLARIX study in patients with untreated DLBCL is ongoing in Japan.

Chugai Receives Orphan Drug Designation for Polatuzumab vedotin in Diffuse Large B-Cell Lymphoma from the MHLW (Press release issued by Chugai on November 20, 2019)
View Source

About JO40762 (P-DRIVE) study
JO40762 (P-DRIVE) is an open label, single-arm study investigating polatuzumab vedotin in combination with BR therapy in 35 patients with relapsed or refractory DLBCL. Primary endpoint is investigator’s assessment of CRR by PET-CT at the timing of PRA (six to eight weeks after last administration of the investigational drugs). Patients received treatment for one cycle of three weeks and was administered up to a total of 6 cycles.

About polatuzumab vedotin
Polatuzumab vedotin is a first-in-class anti-CD79b antibody-drug conjugate (ADC), comprising the anti-CD79b humanized monoclonal antibody and a tubulin polymerization inhibitor attached together using a linker. The CD79b protein is expressed specifically in the majority of B-cells, making it a promising target for the development of new therapies1, 2). Polatuzumab vedotin binds to CD79b and destroys these B-cells through the delivery of an anti-cancer agent, which is thought to suppress the effects on normal cells3, 4). Polatuzumab vedotin is being developed by Roche using Seattle Genetics’ ADC technology and is currently being investigated for the treatment of several types of non-Hodgkin’s lymphoma.

About diffuse large B-cell lymphoma (DLBCL)
DLBCL is one of the histologic subtypes of non-Hodgkin’s lymphoma (NHL), which is categorized as aggressive disease that progresses on a monthly basis. DLBCL is the most common form of NHL, accounting for 30-40 percent of NHL5-7). DLBCL frequently occurs in middle-aged and older people, mainly in their 60’s8). The median age at diagnosis has been reported to be 649).

The combination of rituximab and chemotherapy is the standard therapy for untreated DLBCL; however, recurrence has been observed in about 40% of the patients due to insufficient therapeutic effect10). In addition, although autologous stem cell transplantation (ASCT) is recommended in eligible patients with recurrent or refractory DLBCL, ASCT cannot be performed in about half of these patients due to failure of salvage chemotherapy prior to ASCT11). Furthermore, no standard therapy has been established for patients ineligible for ASCT due to reasons including age or complications12).

Salvage chemotherapy: A therapy mainly used in patients with hematologic malignancy who experienced no therapeutic effects (refractory), or recurrence/relapse of the disease is referred to as a salvage chemotherapy or salvage therapy. Applicable treatment may vary depending on the type of cancer, most of which will be combination therapies consisting of multiple drugs including anticancer agents13).

About orphan drugs
Based on Pharmaceuticals and Medical Devices Law, orphan drugs are designated by the Minister of Health, Labour and Welfare and granted priority review. The designation criteria are as follows: The number of patients who may use the drug is less than 50,000 in Japan; The drug is indicated for the treatment of serious diseases and there is a significant medical value such as no alternative appropriate drug or treatment, or high efficacy or safety expected compared to existing products; there is a theoretical rationale for using the product for the targeted disease and the development plan is reasonable.

On February 13, 2020 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) reported business highlights and financial results for the fourth quarter and year ended December 31, 2019 (Press release, Agios Pharmaceuticals, FEB 13, 2020, View Source [SID1234554271]). In addition, Agios highlighted key 2020 corporate milestones and data presentations for its clinical development programs.

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"On the heels of a busy and productive 2019, I’m more confident than ever in the strength of our team and our ability to make a meaningful impact on the lives of patients through great science, a deep pipeline and differentiated therapies," said Jackie Fouse, Ph.D., chief executive officer at Agios. "In 2020, our clinical development team is focused on advancing our Phase 3 PK deficiency studies in order to submit a new drug application in 2021, finalizing our pivotal development plan for the PK activation program in thalassemia and establishing proof-of-concept in sickle cell disease. In addition, we are driving enrollment in several Phase 3 studies for our IDH inhibitors in both malignant hematology and solid tumors. Our commercial team is focused on achieving an ambitious revenue target for TIBSOVO and increasing market development activities in preparation for a potential launch in PK deficiency."

ANTICIPATED 2020 KEY MILESTONES

Agios expects the following key milestones in 2020:

Hematologic Malignancies

Deliver full-year U.S. revenue for TIBSOVO of $105-115 million

Receive European Medicines Agency CHMP opinion for TIBSOVO in relapsed or refractory acute myeloid leukemia (AML) with an IDH1 mutation by year-end

Complete enrollment of the Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1 mutant AML by year-end
enrollment of the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1 mutant advanced hematologic malignancies by year-end

Solid Tumors

File supplemental new drug application (sNDA) for TIBSOVO in previously treated IDH1 mutant cholangiocarcinoma by year-end

Rare Genetic Diseases

Announce topline data for ACTIVATE and ACTIVATE-T pivotal trials for mitapivat in adults with pyruvate kinase (PK) deficiency by year-end

Submit updated data from the Phase 2 study of mitapivat in thalassemia for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June and finalize pivotal development strategy by year-end

Achieve proof-of-concept for mitapivat in sickle cell disease by mid-2020

Receive investigational new drug (IND) clearance for AG-946, a next generation PKR activator, and initiate a first-in-human study in healthy volunteers in the first half of 2020

Research

Achieve at least one new development candidate by year-end

FOURTH QUARTER AND FULL YEAR 2019 FINANCIAL RESULTS

Revenue: Total revenue for the fourth quarter of 2019 was $35.4 million, which includes $12.9 million in collaboration revenue, $19.6 million of net product revenue from sales of TIBSOVO and $3.0 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to $30.0 million for the fourth quarter of 2018, which included $18.4 million in collaboration revenue, $9.4 million of net product revenue from U.S. sales of TIBSOVO and $2.2 million in royalty revenue from net global sales of IDHIFA. Total revenue for the year ended December 31, 2019 was $117.9 million compared to $94.4 million for the year ended December 31, 2018. The increase in 2019 revenue was primarily driven by net U.S. sales of TIBSOVO and were offset by a decline in collaboration revenue due to the recognition of a milestone from Celgene and the upfront payment from CStone in 2018.

Cost of Sales: Cost of sales were $0.3 million for the fourth quarter of 2019 compared to $0.7 million for the fourth quarter of 2018, and $1.3 million for the year ended December 31, 2019 compared to $1.4 million for the comparable period in 2018.

Research and Development (R&D) Expenses: R&D expenses were $106.2 million for the fourth quarter of 2019 compared to $93.8 million for the fourth quarter of 2018 and $410.9 million for the year ended December 31, 2019 compared to $341.3 million for the comparable period in 2018. The increase in R&D expense was primarily attributable to clinical trial activity for mitapivat in PK deficiency and thalassemia; start-up costs for the vorasidenib Phase 3 INDIGO study in low-grade glioma, including required clinical pharmacology studies and companion diagnostic development; and ongoing enrollment in the TIBSOVO Phase 3 AGILE and HOVON frontline AML combination studies. R&D expense also increased as a result of ongoing research efforts across our discovery platform programs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $34.8 million for the fourth quarter of 2019 compared to $31.9 million for the fourth quarter of 2018, and $132.0 million for the year ended December 31, 2019 compared to $114.1 million for the year ended December 31, 2018. The increase in SG&A expense was primarily attributable to increased investment in marketing activities in preparation for the potential launch of mitapivat and personnel costs related to increased headcount to support growing operations.

Net Loss: Net loss was $102.4 million for the fourth quarter of 2019 compared to $91.8 million for the fourth quarter of 2018, and $411.5 million for the year ended December 31, 2019 compared to a net loss of $346.0 million for the year ended December 31, 2018.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of December 31, 2019 were $717.8 million compared to $805.4 million as of December 31, 2018. The change in cash was primarily driven by expenditures to fund operations of $464.4 million offset by the net proceeds of $277.2 million from the November follow-on offering and cash inflows of $99.3 million from product sales, stock option exercises, royalty revenue, and collaboration reimbursements and milestones. The company expects that its cash, cash equivalents and marketable securities as of December 31, 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 collaboration-related payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2021.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 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 referring to conference ID 4195413. 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.

On February 13, 2020 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported the closing of a $60 million senior secured loan facility with Oxford Finance LLC, a specialty finance firm providing senior debt to life sciences and healthcare service companies (Press release, Syros Pharmaceuticals, FEB 13, 2020, View Source [SID1234554295]).

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"The initial $20 million tranche of this financing extends our expected cash runway into 2022, beyond key planned clinical data readouts for SY-1425 and SY-5609 in multiple cancer patient populations," said Joseph Ferra, Syros’ Chief Financial Officer. "By providing access to additional capital, this facility also increases our financial flexibility as we continue to advance our clinical programs and earlier-stage pipeline with the aim of bringing small-molecule medicines to market that provide a profound benefit for patients with cancer and monogenic diseases."

Syros plans to use the proceeds of the financing to advance its lead product candidates SY-1425 and SY-5609, for which expected clinical readouts include potential proof-of-concept data for SY-1425 in RARA-positive relapsed or refractory acute myeloid leukemia patients in the fourth quarter of 2020, initial dose-escalation data for SY-5609 in select solid tumors in the fourth quarter of 2020, and additional dose-escalation data, including clinical activity data, for SY-5609 in mid-2021. Syros also expects to use proceeds from the financing to advance its preclinical programs toward the potential nomination of its next development candidate by the end of 2021, as well as for general corporate purposes.

The non-dilutive financing agreement provides Syros with up to $60 million in borrowing capacity in three tranches, with the initial tranche of $20 million available immediately. Syros is required to make monthly interest-only payments on each tranche prior to the amortization date of March 1, 2023. The debt facility will mature on February 1, 2025.

On February 13, 2020 SOPHiA GENETICS, leader in Data-Driven Medicine, reported the appointment of Philippe Menu, MD-PhD, MBA, as Chief Medical Officer (Press release, Sophia Genetics, FEB 13, 2020, View Source [SID1234554311]).

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Dr. Philippe Menu brings a unique blend of medical expertise across multiple areas including clinical medicine, fundamental research in molecular biology, and management consulting. He spent the last eight years at McKinsey & Company where he co-led the McKinsey Cancer Center and served dozens of clients in the biopharma sector. Dr. Menu advised global pharmaceutical companies, mid-size players and biotech alike across the entire value chain, with a major focus on innovative therapies and diagnostics in oncology and rare diseases.

"The potential linked to new-generation health data is limitless and it is our goal to continue expanding the scope of new clinical applications for our community of more than 1,000 hospitals across 82 countries," affirmed Jurgi Camblong, CEO and Co-founder of SOPHiA GENETICS. "Philippe’s background and track record across different sectors of life sciences, including biopharma, will help the execution of new clinical-grade applications. In turn, this will allow the longitudinal monitoring of patients through multi-modal data approaches and the optimization of drug development."

"It is a privilege to be joining SOPHiA GENETICS as Chief Medical Officer," said Dr. Menu. "I am incredibly inspired by what the SOPHiA team has already achieved by analyzing half a million patients’ genomic profiles across the world through its unique and growing global network of hospital partners. Looking ahead, I am most impressed by the full potential to positively impact patients’ lives that still lies ahead of us through the application of SOPHiA’s multi-modal data approach. SOPHiA is uniquely positioned to help deliver transformative progress for patients around the world: we can help discover new biomarkers to develop new therapies, match the right treatment to the right patients in clinical trials as well as in routine clinical care, and follow patients longitudinally through a multi-omics approach to help predict who will most benefit from which therapies and why. I look forward to working closely with our hospital, biopharma and other healthcare ecosystem partners to help accelerate the adoption of Data-Driven Medicine."

On February 13, 2020 Theravance Biopharma, Inc. (NASDAQ: TBPH), a diversified biopharmaceutical company primarily focused on the discovery, development and commercialization of organ-selective medicines, reported that it will report its fourth quarter and full year 2019 financial and operating results and provide a business update after market close on Monday, February 24, 2020 (Press release, Theravance, FEB 13, 2020, View Source [SID1234554327]). An accompanying conference call and simultaneous webcast will be hosted at 5:00 p.m. ET (2:00 p.m. PT/10:00 p.m. GMT) that day.

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Conference Call Information

To participate in the live call by telephone, please dial (855) 296-9648 from the US or (920) 663-6266 for international callers, using the confirmation code 5775588. Those interested in listening to the conference call live via the internet may do so by visiting Theravance Biopharma’s website at www.theravance.com, under the Investor Relations section, Presentations and Events.

A replay of the conference call will be available on Theravance Biopharma’s website for 30 days through March 25, 2020. An audio replay will also be available through 8:00 p.m. ET on March 2, 2020 by dialing (855) 859-2056 from the US, or (404) 537-3406 for international callers, and then entering confirmation code 5775588.