On February 16, 2022 Invitae (NYSE: NVTA), a leading medical genetics company, reported the availability of FusionPlex Dx and LiquidPlex Dx in Europe, part of its industry-leading Anchored Multiplex PCR chemistry in-vitro diagnostic (IVD) products (Press release, Invitae, FEB 16, 2022, View Source [SID1234608168]). Invitae is delivering essential high quality innovation for precision oncology in the fight against cancer.

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With this announcement, Invitae provides the highest quality CE-IVD products to enable European pathologists and oncologists to efficiently provide vital information, in a timely manner, to guide cancer therapy for patients.

"This is a major step toward our mission to bring comprehensive genetic information into mainstream medicine," said Vishal Sikri, President of Oncology at Invitae. "Our best in class FusionPlex and LiquidPlex tests will enable more patients access to the right cancer therapies at the right time, which is especially critical in Europe where the majority of molecular testing is performed locally in decentralized hospital settings."

In line with ESMO (Free ESMO Whitepaper) guidelines for non-small cell lung cancer (NSCLC), Invitae’s FusionPlex Dx and LiquidPlex Dx allow comprehensive genomic profiling (CGP) and companion diagnostic (CDx) use for solid tumor neoplasms including NSCLC in tissue biopsy and where tissue is a limiting factor. The panels are intended to be used on Illumina’s Next Generation Sequencing (NGS) platforms for IVD testing.

"Increasing patient access to content on our instrument fleet through partnerships such as those with Invitae speaks to our mission to improve human health by unlocking the power of the genome," said Joydeep Goswami, Chief Strategy and Corporate Development Officer at Illumina. "We congratulate Invitae on its first CE-IVD cancer testing kits, a significant milestone."

FusionPlex Dx identifies structural variants including fusions in RNA derived from formalin-fixed, paraffin-embedded tumor specimens. FusionPlex Dx covers multiple actionable variants in a 41-gene panel to provide CGP for patients with solid malignant neoplasms. FusionPlex Dx is intended to be used as a CDx to aid in identifying patients diagnosed with NSCLC with mesenchymal-epithelial transition factor (MET) exon 14 (METex14) skipping alterations, anaplastic lymphoma kinase (ALK) fusions, ros proto-oncogene 1, receptor tyrosine kinase (ROS1) fusions, ret proto-oncogene (RET) fusions, neurotrophic receptor tyrosine kinase 1-3 (NTRK1, NTRK2, NTRK3) fusions, and for whom treatment with a targeted therapy may be beneficial.

LiquidPlex Dx identifies substitutions and insertion-deletion mutations in cell-free circulating tumor DNA (ctDNA) isolated from plasma derived from whole blood of cancer patients (liquid biopsy). LiquidPlex Dx interrogates 29 genes to provide CGP for patients with solid malignant neoplasms. LiquidPlex Dx is intended to be used as a CDx to aid in identifying patients diagnosed with NSCLC with METex14 skipping alterations, and for whom treatment with a targeted therapy may be beneficial.

"With Invitae’s simplified workflow and reporting solutions, both tests will enable any facility with sequencing technology, combined with our solutions, to accurately profile solid tumors using tissue or blood samples for therapy selection," said Sikri. "Precision medicine has increasingly delivered better outcomes for many cancer patients over the last several years. Molecular pathology has never been more important as the fight against cancer shifts towards precision oncology and targeted therapies based on genomic testing. Yet globally, access to the exponentially increasing therapy options is not guaranteed."

On February 16, 2022 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage, precision oncology biopharmaceutical company, reported that Susan Molineaux, Ph.D., the company’s founder, president and chief executive officer, will participate in a fireside chat at the 11th Annual SVB Leerink Global Healthcare Conference on Thursday, February 17 at 4:20 p.m. Eastern Time (Press release, Calithera Biosciences, FEB 16, 2022, View Source [SID1234608164]). An archived replay of the webcast will be available on the Company’s website for 30 days following the live presentation.

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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 BerGenBio ASA (OSE: BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for severe unmet medical needs, reported its results for the fourth quarter and full year 2021 (Press release, BerGenBio, FEB 16, 2022, View Source [SID1234608161]).

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A briefing by BerGenBio’s senior management team will take place at 10:00am CET today via a webcast presentation, followed by a Q&A session. Please see below for details.

Operational Highlights – fourth quarter 2021 (including post-period end)• Post-period end, BerGenBio announced its participation in the EU-SolidAct trial, part of EU-RESPONSE, a pan-European research project involved with the rapid and coordinated investigation of medications to treat COVID-19 during the ongoing pandemic allowingBerGenBio to potentially confirm encouraging clinical data at significantly reduced cost​. • Licensed key exclusive intellectual property rights from UT Southwestern Medical Center, which strengthen the intellectual property estate for the potential treatment of Non-Small Cell Cancer (NSCLC) patients with STK11 mutations.• Granted Fast Track designation by the FDA for bemcentinib in combination with an anti-PD-(L)1 agents as treatment for patients with STK11 altered advanced/metastatic NSCLC patients without actionable mutations.• Presented pre-clinical and clinical data on bemcentinib in STK11 mutated NSCLC at SITC (Free SITC Whitepaper) Annual Meeting 2021, indication that bemcentinib restores response to anti-PD-1 treatments in NSCLC patients with STK11 mutations.• Presented updated data from Phase II trial(BGBC003) of bemcentinib in combination with low dose cytarabine (LDAC) in older, relapsedand refractory AML patients at the 63rd Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting.• Anders Tullgren appointed as Chairman of the Board, bringing over 35 years of global experience in both large pharmaceutical and small/mid-size biotech environments.

Financial Highlights – fourth quarter and full year 2021(Figures in brackets = same period 2020 unless otherwise stated) • Revenue amounted to NOK 0.8 million (NOK 0.6million) for the fourth quarter and NOK 0.8million (NOK 0.6 million) for the full year 2021• Total operating expenses for the fourth quarter were NOK 68.1 million (NOK 72.4 million) and total operating expenses for the full year 2021amounted to 315.2 million (NOK 261.7 million)• The operating loss for the quarter came to NOK 67.3 million (NOK 71.8 million) and NOK 314.5 million (NOK 261.1 million) for the full year2021• Cash and cash equivalents amounted to NOK 436.4 million at the end of December 2021 (NOK 721.6 million by end of December 2020)

Martin Olin, Chief Executive Officer of BerGenBio, commented: "In the fourth quarter of 2021 we continued to make significant progress in defining our strategy and priorities to advance our two AXL development candidates. Our lead development candidate, bemcentinib, a potentially first-in-class selective AXL inhibitor currently undergoing Phase II clinical trials in NSCLC, AMLand respiratory infections (COVID-19), is well positioned for further advancement.

"Our strategy and priorities going forward will be anchored within a rigorous data-driven framework, where the scientific rationale, pre-clinical and clinical data define a compelling rationale for advancing our development candidates towards potential treatment modalities addressing high unmet medical needs.

"With a clear strategy in place, I believe BerGenBio is well positioned to progress and deliver on its potential. I look forward to providing you with further updates on our progress."

Presentation and Webcast Details

The live webcast link is available at www.bergenbio.com in the Investors/Financial Reports section. A recording will be available shortly after the webcast has finished.

The fourth quarter and full year report and presentation will be made available on the Company’s website in the Investors/Financial Reports section and a recording of the webcast will be made available shortly after the webcast has finished.

On February 15, 2022 Intellia Therapeutics Inc is to out-license rights to its ex vivo Crispr/Cas9 genome editing platform to ONK Therapeutics Ltd enabling the Irish company to develop engineered natural killer (NK) cell therapies for cancer (Press release, ONK Therapeutics, FEB 15, 2022, View Source [SID1234613904]). ONK is developing off-the-shelf NK cell therapies. In addition to the genome editing platform, ONK will also have access to the US company’s lipid nanoparticle delivery technologies. The agreement will enable the development of up to five allogeneic NK cell therapies.

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