On February 16, 2022 Pebble Global Holdings (aka "Pebble Life Sciences" or "Pebble"), a privately held Texas-based leader in non-psychotropic cannabinoid research for drug development, reported that a sponsored research program entitled "Evaluation of Multi-Cannabinoid Formulations in Ovarian Cancer" was initiated in 2020 and presently exists between Pebble and MD Anderson Cancer Center ("MD Anderson") (Press release, MD Anderson, FEB 16, 2022, View Source [SID1234608196]). MD Anderson is one of the world’s most respected centers devoted exclusively to cancer patient care, education, prevention, research and ranked No. 1 in cancer in the U.S. News & World Report’s 2021-2022 annual "Best Hospitals."

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Despite being one of the most researched, ovarian cancer is the leading cause of gynecological cancer in the US and second most globally. It is the fifth leading cause of cancer death in US women, with 22,000 diagnosed in 2021, resulting in 14,000 deaths. 75% of patients are "advanced" at initial diagnosis, with 1 in 6 dying within 90 days and treatment limited by severe adverse effects from the current standard of care.

"Our team has thoroughly enjoyed working with MD Anderson the past two years on this novel therapy. We’re excited to continue with MD Anderson, to improve cancer treatment standard of care. Pebble’s non-THC, non-psychotropic approach permits a regulatory drug pathway that allows both domestic and global distribution, so we can help as many suffering women as possible," says Pebble Founder & CEO – Patrick Moran.

On February 16, 2022 Blueprint Medicines Corporation (NASDAQ: BPMC) reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2021 (Press release, Blueprint Medicines, FEB 16, 2022, View Source [SID1234608162]).

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"Following a year of outstanding execution across our portfolio, we are well positioned in 2022 to drive significant year-over-year product revenue growth, achieve multiple anticipated clinical data milestones for our precision therapies in systemic mastocytosis and lung cancer, and expand our portfolio with new therapeutic candidates, all supported by a strong cash position," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, as we execute on this near-term vision, we will put in place drivers of future growth, including the expansion of our research platform to incorporate targeted protein degradation, as well as plans for targeted sourcing of external innovation. Together, these efforts have the potential to significantly strengthen and broaden our impact as a leading global precision therapy company."

Fourth Quarter 2021 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): systemic mastocytosis (SM) and gastrointestinal stromal tumor (GIST)

Recorded global net product revenues of $53.0 million and $20.0 million for the full year and the fourth quarter of 2021, respectively, representing approximately 150 percent year-over-year growth based on strong initial U.S. demand in advanced SM.
Received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use for AYVAKYT for the treatment of adult patients with advanced SM, including aggressive SM, SM with an associated hematological neoplasm, or mast cell leukemia, after at least one systemic therapy. Read the press release here.
Announced two publications in Nature Medicine on the registration-enabling EXPLORER and PATHFINDER trials highlighting AYVAKIT’s robust efficacy and safety data in advanced SM. Read the press release here.
GAVRETO (pralsetinib): RET-altered cancers

Following the transition of certain responsibilities from Blueprint Medicines to Roche in the third quarter of 2021, Roche recorded and reported $9.8 million in U.S. net end user product revenues for the second half of 2021. Full year 2021 U.S. net end user product revenues for GAVRETO were $14.5 million, including sales booked by Blueprint Medicines in the first half of 2021.
Received, via the collaboration with Roche, conditional marketing authorization by the European Commission for GAVRETO for the treatment of adults with RET fusion-positive non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor.
BLU-945, BLU-701 and BLU-451 (formerly LNG-451): EGFR-driven NSCLC

Treated the first patient in the Phase 1/2 HARMONY trial of BLU-701 in EGFR-driven NSCLC.
Presented new preclinical data supporting the development of BLU-945 and BLU-701 combination therapy in EGFR-driven NSCLC at the British Thoracic Oncology Group annual conference. Read the poster presentation here.
Multiple abstracts accepted for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, including initial Phase 1/2 SYMPHONY trial dose escalation data for BLU-945 in EGFR-driven NSCLC.
Announced a strategic collaboration and license agreement with Zai Lab Limited for the development and commercialization of BLU-945 and BLU-701 for the treatment of EGFR-driven NSCLC in Greater China. Read the press release here.
Completed the acquisition of Lengo Therapeutics and its lead compound BLU-451, a potential best-in-class precision therapy for EGFR exon 20 insertion-positive NSCLC. Read the press release here.
Received clearance from the U.S. Food and Drug Administration (FDA) for an investigational new drug (IND) application for BLU-451 in EGFR exon 20 insertion-positive NSCLC.
BLU-222: ovarian, breast and other cyclin E-CDK2 aberrant cancers

Received FDA clearance for an IND application for BLU-222 for cyclin E-CDK2 aberrant cancers.
Corporate

Announced Kate Haviland, who has served as Chief Operating Officer and previously Chief Business Officer since 2016, has been appointed by the company’s Board of Directors to succeed Jeff Albers as President and Chief Executive Officer, effective April 4, 2022. At that time, Mr. Albers will transition from his current role as Chairman, President and Chief Executive Officer to Executive Chairman of the Board of Directors. In addition, Ms. Haviland will join the Board of Directors. Read the press release here.
Announced promotions for Christina Rossi to Chief Operating Officer and Philina Lee, Ph.D., to Chief Commercial Officer. In addition, announced a role expansion for Helen Ho, Ph.D., to Chief Business Officer. Read the press releases here and here.
Announced the appointment of Daniella Beckman, Chief Financial Officer of Tango Therapeutics, to the company’s Board of Directors. Read the press release here.
Key Upcoming Milestones

The company plans to achieve the following milestones by mid-2022:

Initiate a Phase 1/2 trial of BLU-222 in cyclin E-CDK2 aberrant cancers in the first quarter of 2022.
Initiate a Phase 1/2 trial of BLU-451 in EGFR exon 20 insertion-positive NSCLC in the first quarter of 2022.
Present initial clinical data from the Phase 1/2 SYMPHONY trial of BLU-945 in the second quarter of 2022.
Present preclinical data supporting the development of BLU-451 in EGFR exon 20 insertion-positive NSCLC in the second quarter of 2022.
Present preclinical data supporting the development of BLU-222 in cyclin E-CDK2 aberrant cancers in the second quarter of 2022.
Launch AYVAKYT in advanced SM in Europe in the second quarter of 2022.
Report topline data from the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced SM in mid-2022.
Fourth Quarter and Year End 2021 Results

Revenues: Revenues were $107.0 million for the fourth quarter of 2021, including $20.0 million of net product revenues from sales of AYVAKIT and $87.0 million in collaboration revenues. Revenues for the year ended December 31, 2021 were $180.1 million, including $53.0 million of net product revenues from sales of AYVAKIT, $4.7 million of net product revenues from sales of GAVRETO and $122.4 million in collaboration revenues. Blueprint Medicines recorded $34.1 million and $793.7 million in revenues in the fourth quarter and year ended December 31, 2020, respectively.
Cost of Sales: Cost of sales was $7.5 million for the fourth quarter of 2021 and $17.9 million for the year ended December 31, 2021, as compared to $0.1 million for the fourth quarter of 2020 and $0.4 million for the full year ended December 31, 2020. This increase was primarily driven by lower margin drug product sold to our collaboration partners during the fourth quarter of 2021.
R&D Expenses: Research and development expenses were $356.9 million for the fourth quarter of 2021 and $601.0 million for the year ended December 31, 2021, as compared to $77.4 million for the fourth quarter of 2020 and $326.9 million for the year ended December 31, 2020. Research and development expense for the fourth quarter of 2021 included $260.0 million incurred to acquire in-process research and development compounds through the acquisition of Lengo Therapeutics which was the primary driver of the increase in expenses over the prior year. Research and development expenses also included $10.0 million in stock-based compensation expenses for the fourth quarter of 2021 and $39.7 million in stock-based compensation for the year ended December 31, 2021.
SG&A Expenses: Selling, general and administrative expenses were $54.2 million for the fourth quarter of 2021 and $195.3 million for the year ended December 31, 2021, as compared to $42.5 million for the fourth quarter of 2020 and $157.7 million for the year ended December 31, 2020. This increase was primarily due to increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT. Selling, general and administrative expenses included $12.7 million in stock-based compensation expenses for the fourth quarter of 2021 and $52.0 million in stock-based compensation for the year ended December 31, 2021.
Net Income (Loss): Net loss was $(318.7) million for the fourth quarter of 2021 and $(644.1) million for the year ended December 31, 2021, or a diluted net loss per share of $(5.40) and diluted net loss per share of $(11.01), respectively, as compared to a net loss of $(85.7) million for the fourth quarter of 2020 and a net income of $313.9 million for the year ended December 31, 2020, or a diluted net loss per share of $(1.53) and a diluted net income per share of $5.59, respectively.
Cash Position: As of December 31, 2021, cash, cash equivalents and marketable securities were $1,034.6 million, as compared to $1,549.7 million as of December 31, 2020.
2022 Financial Guidance

Blueprint Medicines reported it anticipates approximately $180M to $200M in total revenues in 2022, including approximately $115M to $130M in AYVAKIT net product revenues. The company continues to expect that its existing cash, cash equivalents and investments, together with anticipated future product revenues, will provide sufficient capital to enable the company to achieve a self-sustainable financial profile.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss fourth quarter and full year 2021 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international), and referring to conference ID 936793. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conferences

Blueprint Medicines will participate in two upcoming investor conferences:

11th Annual SVB Leerink Global Healthcare Conference on Friday, February 18, 2022 at 10:40 a.m. ET.
Cowen 42nd Annual Health Care Conference on Tuesday, March 8, 2022 at 9:10 a.m. ET.
A live webcast of each presentation will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcasts will be archived on Blueprint Medicines’ website for 30 days following each presentation.

On February 16, 2022 BioCanRx reported Dr. Carl H. June as our distinguished keynote speaker for the 2022 Summit for Cancer Immunotherapy (Summit4CI) taking place November 19-21, 2022 (Press release, BioCanRx, FEB 16, 2022, View Source;utm_medium=rss&utm_campaign=carl-h-june-keynote-speaker-2021-summit4ci-2 [SID1234608181]).

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Dr. June is the Richard W. Vague Professor in Immunotherapy, Director of the Center for Cellular Immunotherapies and Director of the Parker Institute for Cancer Immunotherapy in the Perelman School of Medicine at the University of Pennsylvania. The impact of his work has become widely recognized as a major turning point that is delivering on the long-held promise of cancer gene therapy. Dr. June is a pioneer in immunotherapy research who spearheaded the development of chimeric antigen receptor (CAR) T-cell therapy, a new tool for treating cancer that involves extracting a patient’s immune cells and genetically engineering them so they can attack and kill their cancer. This treatment received FDA approval in August 2017, followed by more approvals for varying countries and indications. Dr. June has published over 350 manuscripts and has received multiple awards and honours.

Summit4CI 2022 is taking place in Montréal, Quebec, at the Fairmont the Queen Elizabeth. Please join us Saturday, November 19 to Monday, November 21 as we explore the latest progress in cancer immunotherapy from scientific, clinical, industry and patient perspectives during BioCanRx’s sixth scientific conference. In addition to our feature keynote speaker Dr. June, this year’s Summit4CI will feature internationally recognized speakers, networking and social events, an HQP Development Day, scientific presentations, poster sessions, a public forum, an Oxford-style debate, and more. We hope to see you at the conference! Learn more here.

On February 16, 2022 Electra Therapeutics, Inc., a clinical stage biotechnology company developing antibody therapies that target signal regulatory proteins (SIRP), reported a $84 million Series B financing co-led by Westlake Village BioPartners and OrbiMed (Press release, Electra Therapeutics, FEB 16, 2022, View Source [SID1234608197]). Other participating investors include Redmile Group, Cormorant Asset Management, Cowen Healthcare Investments, RA Capital, and New Leaf Venture Partners. Electra was previously funded by its parent company, Star Therapeutics.

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Electra is building a pipeline of novel therapies targeting SIRP, a family of cell surface receptors on various immune cell types. The company is taking a first-in-class approach to engage SIRP beyond the current drug development efforts in cancer, by targeting specific SIRP proteins to deplete targeted pathological immune cells. ELA026, the company’s lead product candidate, exhibits this novel mechanism of action and is currently in Phase 1 clinical studies, including a trial in secondary hemophagocytic lymphohistiocytosis (sHLH), a life-threatening inflammatory disease. ELA026 is also advancing in other immunological diseases. In addition, the company’s pipeline includes preclinical programs in immunology and immuno-oncology.

"We are thrilled to introduce Electra and share our novel approach to targeting SIRP," said Adam Rosenthal, PhD, CEO and Co-founder of Electra. "Our team has made rapid progress advancing our lead product candidate, ELA026, from an idea to the clinic in less than 3 years, and we are excited by each of our three pipeline programs targeting numerous diseases with significant unmet need."

Proceeds from the financing will be used to continue advancing ELA026 in clinical trials for sHLH and other immunological diseases. The funding will also support advancement of an additional preclinical pipeline program targeting SIRP in immunology, as well as Electra’s immuno-oncology platform that leverages SIRP to promote targeted depletion of tumor cells.

"It is inspiring to see the rapid progress and success of Electra’s pioneering work in targeting SIRP to develop therapies for immunological diseases. ELA026 represents a novel mechanism for targeting diseases driven by aberrant myeloid and T cell activity, and has enormous potential to address life-threatening diseases like sHLH, as well as other severe inflammatory diseases," said Beth Seidenberg, MD, Founding Managing Director at Westlake Village BioPartners.

Electra’s Board of Directors consists of Beth Seidenberg, MD, Founding Managing Director at Westlake Village BioPartners; Carl Gordon, PhD, CFA, Managing Partner at OrbiMed; Nancy Stagliano, PhD, Executive Board Chair, and Adam Rosenthal, PhD, CEO and Co-founder of Electra. Electra’s Board observers are Amrit Nagpal, Managing Director at Redmile Group and Vijay Lathi, Managing Director at New Leaf Venture Partners.

"We are impressed by the bold vision of the Electra team which has deep insights into drug discovery and development, and we are pleased to support Electra as they translate their approach with SIRP to patients in need of innovative treatment options," said Carl Gordon, PhD, CFA, Managing Partner at OrbiMed.

About Secondary Hemophagocytic Lymphohistiocytosis (sHLH)

Secondary hemophagocytic lymphohistiocytosis (sHLH) is a life-threatening hyperinflammatory condition for which there is no approved treatment. sHLH can be triggered by cancer, immunotherapy, infection, or an autoimmune disease. Once triggered, sHLH requires immediate intervention. Without treatment, it can rapidly progress from symptoms such as persistent fever, hepatomegaly and/or splenomegaly, and cytopenias, to multi-organ failure and death. Even with the current use of off-label treatments that have toxicity challenges and limited efficacy, sHLH remains fatal in approximately 60% of adults within 3.5 years.

Amgen has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission .

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