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Novocure Announces Preliminary Full Year and Fourth Quarter 2021 Net Revenues and Provides Company Update

On January 10, 2022 Novocure (NASDAQ: NVCR) reported preliminary unaudited financial results and operational updates for the quarter and full year ended December 31, 2021 (Press release, NovoCure, JAN 10, 2022, View Source [SID1234598529]).* Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields (TTFields). TTFields are electric fields that disrupt cancer cell division.

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"Since our founding over 20 years ago, we have generated tremendous scientific data, technological knowledge and commercial experience supporting the clinical impact of Tumor Treating Fields," said William Doyle, Novocure’s Executive Chairman. "With over a half a billion dollars in global annual revenues and numerous late-stage trials nearing completion, Novocure is approaching a critical inflection point for our company and cancer patients."

Financial and operational updates:

Total preliminary net revenues for the year ended December 31, 2021, were $535.0 million, up 8% over the prior year.
Total preliminary net revenues for the fourth quarter 2021 were $133.2 million. In the fourth quarter, the company did not recognize material revenue from its Medicare backlog. This compares to $11 million received from successful appeal of previously denied Medicare claims in the fourth quarter of 2020.
Preliminary fourth quarter 2021 net revenues from the United States, EMEA and Japan contributed $92.0 million, $26.5 million and $8.8 million, respectively. Revenue in Greater China from Novocure’s partnership with Zai Lab totaled $5.8 million.
The number of active patients on therapy and the amount of net revenue recognized per active patient are our principal revenue drivers.
In the fourth quarter 2021, Novocure initiated contract negotiations with several large German payers that drove an update to the net revenue recognized per active patient, which negatively impacted fourth quarter revenue by approximately $4.0 million. Novocure believes this negotiated pricing is sustainable, will reduce the burden of case-by-case appeals and can be leveraged as the company expands into additional European markets.
As of December 31, 2021, there were 3,587 active patients on therapy. Active patients from North America, EMEA and Japan contributed 2,272, 1,008 and 307, respectively.
In the quarter ended December 31, 2021, 1,430 prescriptions were received. Prescriptions from North America, EMEA and Japan contributed 966, 349 and 115, respectively.
In 2022, the company expects to achieve active patient growth rates of 2% to 5%, in-line with the growth rate experienced in the fourth quarter 2021. Longer term, the company continues to expect further adoption in its core glioblastoma business.
Cash, cash equivalents and short-term investments were $937.7 million as of December 31, 2021.
Clinical and Product Development:

Given the limited total number of events seen to date in the fast-recruiting INNOVATE-3 pivotal trial in platinum-resistant ovarian cancer, Novocure anticipates the independent Data Monitoring Committee (DMC) will conduct its interim analysis in early Q2 2022. Timing of final data is unchanged and expected in 2023.
Today, Novocure is announcing its next generation array designed to increase Tumor Treating Fields dose delivery while limiting heat generation, thereby potentially increasing clinical efficacy. The next generation array design is complete, a healthy volunteer study is underway, and the company is working towards a limited market release in the EU later this year.
Novocure reiterates 2022 guidance for final data from its phase 3, pivotal LUNAR trial in non-small cell lung cancer and its phase 2 pilot EF-31 trial in gastric cancer.
Fourth quarter and full year 2021 financial results conference call

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2021 financial results at 8 a.m. EST on Thursday, February 24, 2022. Analysts and investors can participate in the conference call by dialing 855-442-6895 for domestic callers and 509-960-9037 for international callers, using the conference ID 8879093.

The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

* The unaudited results in this press release are preliminary and subject to the completion of the Company’s annual independent audit and, therefore, are subject to adjustment.

Syros Announces Clinical Updates and 2022 Goals to Support its Advancement to a Fully Integrated Biopharmaceutical Company

On January 10, 2022 Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported an update on its clinical development programs and outlined its strategic priorities and upcoming expected milestones (Press release, Syros Pharmaceuticals, JAN 10, 2022, View Source [SID1234598528]).

"2022 promises to be a transformative year as we continue to advance our studies of tamibarotene, SY-2101, and SY-5609. We are looking forward to three clinical data readouts this year as well as pivotal data from the SELECT-MDS-1 trial in late 2023 or early 2024," said Nancy Simonian, M.D., Syros’ Chief Executive Officer. "Additionally, in the second half of this year we expect to nominate our new development candidate from our CDK12 program, highlighting the productivity of our gene control discovery engine and our expertise in CDK inhibition. Together, we believe these upcoming milestones will provide insight into the clinical potential of our development-stage assets and lay the foundation for our long-term growth as we advance Syros into a fully integrated biopharmaceutical company."

Dr. Simonian continued, "We are pleased with our recent interactions with the U.S. Food and Drug Administration on the Phase 3 clinical trial design of SY-2101, which we now expect to initiate in the first quarter of 2023. In addition, based on preclinical data that supports CDK7 inhibition’s potential across a range of hematologic malignancies, we expect to start in the second half of this year a Phase 1 single agent study of SY-5609 in patients with relapsed blood cancers, including B-cell lymphomas, prior to moving into specific indications. We are excited to be the first company to advance a CDK7 inhibitor into hematology clinical development. The trial results have potential to benefit a broader patient population as well as demonstrate CDK7 inhibition as a novel approach for many difficult-to-treat hematologic cancers."

CLINICAL PROGRAM UPDATES AND UPCOMING MILESTONES

Targeted Hematology

Tamibarotene: Oral RARa agonist

Syros is evaluating tamibarotene in patients with RARA-positive newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS). The Company expects to report data from the ongoing SELECT-MDS-1 Phase 3 trial evaluating tamibarotene in combination with azacitidine in HR-MDS in the fourth quarter of 2023 or first quarter of 2024, with a potential NDA filing expected in 2024.

Syros is also evaluating tamibarotene for the treatment of patients with RARA-positive newly diagnosed unfit acute myeloid leukemia (AML). The Company expects to report data from the safety lead-in portion of the ongoing SELECT-AML-1 Phase 2 trial evaluating tamibarotene in combination with azacitidine and venetoclax in the second half of 2022.

SY-2101: Oral arsenic trioxide (ATO)

Syros is evaluating SY-2101 in patients with newly diagnosed acute promyelocytic leukemia (APL). The Company expects to report PK and safety data from its ongoing dose confirmation trial in mid-2022. The feedback from a Type C meeting with the U.S. Food and Drug Administration (FDA) in November 2021 continues to support molecular complete response rate as the primary endpoint for accelerated approval and event free survival as the primary endpoint for full approval, in each case compared to historic IV ATO data. Additionally, based on the feedback, Syros now expects the trial to enroll approximately 215 patients randomized two to one to receive SY-2101 or intravenously administered (IV) ATO. The IV ATO arm will allow safety and tolerability comparisons. Syros now expects to initiate the Phase 3 trial in the first quarter of 2023 and to announce data in 2025.

Selective CDK Inhibition

SY-5609: Oral CDK7 inhibitor

In the fourth quarter of 2021, Syros initiated the expansion cohort evaluating SY-5609 in combination with chemotherapy in patients with second-line metastatic pancreatic cancer. The cohort is expected to enroll approximately 50 pancreatic cancer patients who have progressed following first-line treatment with FOLFIRINOX. Patients will receive either SY-5609 in combination with gemcitabine, or SY-5609 in combination with gemcitabine and nab-paclitaxel, at the approved doses of the combination agents. The study will evaluate safety and tolerability, as well as efficacy measures such as disease control rate and progression free survival. Syros expects to report safety lead-in data of SY-5609 in combination with chemotherapy in the second half of 2022.

Syros also plans to evaluate the potential of SY-5609 in hematologic tumors. Based on mechanistic rationale and preclinical data, which support the potential of CDK7 inhibition in a broad range of blood cancers, Syros will evaluate the maximum tolerated dose of SY-5609 in patients with relapsed hematologic malignancies, including B-cell lymphomas, such as mantle cell lymphoma, before starting a focused expansion cohort. The Phase 1 trial is expected to begin in the second half of 2022, with data expected mid-2023, which will inform further development in specific hematologic cancers.

In August 2021, Syros entered into an agreement with Roche to explore SY-5609 in combination with atezolizumab in patients with BRAF-mutant colorectal cancer (CRC), and Roche plans for this arm of its ongoing Phase 1/1b INTRINSIC trial to be open for enrollment in the first half of this year. Under the terms of this agreement, Roche is the sponsor of the trial and Syros is supplying SY-5609.

Gene Control Discovery Engine

Syros announced today that the next development candidate from its gene control discovery engine will be a CDK12 inhibitor. Syros plans to nominate this candidate in the second half of 2022.

Syros also announced today that small molecule inhibitors of CDK11 and WRN are the focus of two additional oncology programs in discovery.

Financial Guidance

Based on its current operating plans, Syros expects that its existing cash, cash equivalents and marketable securities will be sufficient to fund its anticipated operating expenses and capital expenditure requirements into 2023.

Arrakis Therapeutics to Present at 40th Annual J.P. Morgan Healthcare Conference

On January 10, 2022 Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, reported that Michael Gilman, Ph.D., Chief Executive Officer, will present a corporate overview at the 40th Annual J.P. Morgan Healthcare Conference on Thursday, January 13, 2022 at 8:30 a.m. ET (Press release, Arrakis Therapeutics, JAN 10, 2022, View Source [SID1234598527]).

Takeda to Acquire Adaptate Biotherapeutics to Develop Novel Gamma Delta (γδ) T Cell Engager Therapies Targeting Solid Tumors

On January 10, 2022 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported the exercise of its option to acquire Adaptate Biotherapeutics ("Adaptate"), a UK company focused on developing antibody-based therapeutics for the modulation of variable delta 1 (Vδ1) gamma delta (γδ) T cells (Press release, Takeda, JAN 10, 2022, View Source [SID1234598526]). Through the acquisition, Takeda will obtain Adaptate’s antibody-based γδ T cell engager platform, including pre-clinical candidate and discovery pipeline programs. Adaptate’s γδ T cell engagers are designed to specifically modulate γδ T cell-mediated immune responses at tumor sites while sparing damage to healthy cells.

The planned acquisition of Adaptate follows Takeda’s recently exercised option to acquire GammaDelta Therapeutics ("GammaDelta") and is intended to further accelerate the development of innovative γδ T cell-based therapies. Combining GammaDelta’s cell therapy-based platform and Adaptate’s antibody-based γδ T cell engager platform with Takeda’s strong research and development organization positions Takeda to be at the leading edge in deploying the full potential of γδ T cells in the fight against cancer. The planned acquisition complements Takeda’s ongoing efforts to research and develop cell engagers for solid tumor applications, bolstered by the novel T cell engager COBRA platform, which was acquired from Maverick Therapeutics in another successful build-to-buy collaboration.

"Partnering with early-stage innovators to access cutting-edge platforms in the fight against cancer is at the center of our R&D strategy," said Christopher Arendt, Ph.D., Head of Oncology Cell Therapy and Therapeutic Area Unit of Takeda. "Adaptate’s γδ T cell engager platform and the team’s deep understanding of γδ T cell biology gives us an opportunity to develop a new class of therapeutics that tap into powerful innate immune mechanisms. The planned acquisition will strengthen our immuno-oncology R&D efforts as part of our ongoing pursuit of life-transforming medicines for patients with cancer."

Adaptate was formed in 2019 as a spin-out company from GammaDelta with investment from Abingworth LLP and Takeda, in which Takeda received an exclusive right to purchase Adaptate for a pre-negotiated upfront payment. The acquisitions of Adaptate and GammaDelta are expected to be finalized in Q1 of Takeda’s fiscal year 2022, pending completion of review under applicable antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.

"Our acquisition by Takeda recognizes the tremendous work put in over the last two years by Adaptate’s incredibly talented team," said Dr. Natalie Mount, CEO of Adaptate. "We have rapidly demonstrated, in preclinical models, the therapeutic potential of our novel Vδ1-targeting antibodies, and this move brings us an exciting step closer to realizing the full potential for Vδ1 T cell targeted therapies to improve treatment outcomes for cancer patients."

In addition, Tim Haines, Chair & Managing Partner at Abingworth noted, "Having played an instrumental role in creating Adaptate, we are delighted to see the impressive developments of its γδ T cell therapeutic antibody portfolio to date, under the leadership of Natalie Mount. We look forward to seeing Takeda progress Adaptate’s very promising therapeutic antibodies into the clinic."

Takeda’s oncology pipeline focuses on novel strategies that leverage the power of the immune system, with a focus on innate immunity. Innate immune responses serve as the body’s first defense mechanism against disease and involve the orchestration of a broad arsenal of mechanisms and cell types, including γδ T cells and natural killer (NK) cells, that may help to overcome cancer’s ability to evade immune recognition. Adaptate has discovered a unique set of antibodies that selectively modulate γδ T cell activity in the tumor microenvironment. The antibodies provide a precisely targeted signal to the immune system, thereby offering the opportunity for superior efficacy and safety compared to conventional immuno-oncology approaches in solid tumors.

Takeda’s Commitment to Oncology

Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it’s with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com

Scholar Rock Provides Corporate Update and Highlights Priorities for 2022

On January 10, 2022 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported that recent corporate updates and highlighted upcoming priorities for its pipeline programs in 2022 (Press release, Scholar Rock, JAN 10, 2022, View Source [SID1234598525]).

"2021 was another transformative year for Scholar Rock, with positive data from both of our clinical programs, including from the TOPAZ Phase 2 trial for apitegromab, which is being developed for the improvement of motor function in patients with SMA; and Part A of the DRAGON Phase 1 proof-of-concept trial for SRK-181, being developed to overcome resistance to check point inhibitor therapy in cancer patients," said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. "In 2022, we are thrilled to be advancing a pivotal Phase 3 trial of apitegromab and advancing our SRK-181 program to test our hypothesis that this highly selective and potent molecule can overcome resistance to checkpoint inhibitors thereby increasing the number of patients who may benefit from cancer immunotherapy. In addition, the preclinical pipeline has received a major boost as we have regained rights to assets discovered and developed during our research partnership with Gilead that have novel pharmacological profiles relevant to TGFβ-mediated diseases."

2022 Priorities:

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

Robust Enrollment of the Phase 3 SAPPHIRE Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients. Scholar Rock has initiated the SAPPHIRE study. The study design plans for approximately 156 patients aged 2-12 years old with non-ambulatory Type 2/3 SMA to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks added on top of background SMN treatment.
Progress TOPAZ Long-Term Extension to Two Year Readout. As of January 6, 55 of 57 patients remain in the long-term extension trial of apitegromab in Type 2 and 3 SMA.
Advance Development Activities to Include Patients with Type 1 and Ambulatory SMA.
SRK-181 is a potent and highly selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Advance Progress in Part B of DRAGON Phase 1 Proof-of-Concept Trial. Based on the safety and pharmacokinetic data from Part A of the DRAGON Phase 1 trial, Scholar Rock has initiated the Part B dose expansion portion of the trial, which is evaluating SRK-181 dosed 1500 mg every three weeks (Q3W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q3W and 1000 mg every two weeks (Q2W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q2W. Part B will enroll and dose patients in multiple proof of concept cohorts conducted in parallel, including;
Urothelial carcinoma (UC),
Cutaneous melanoma (MEL),
Non-small cell lung cancer (NSCLC),
Clear cell renal cell carcinoma (ccRCC),
Other solid tumors.
Each cohort is expected to enroll up to 40 patients with various locally advanced or metastatic solid tumors who have demonstrated primary resistance to anti-PD-(L)1 therapy. Early efficacy and safety data are anticipated in 2022.

Advancing assets gained from the Gilead collaboration. In December 2018, Gilead Sciences and Scholar Rock entered into a three-year collaboration to discover and develop therapeutics that target TGFβ-driven signaling, a central regulator of fibrosis. Under the collaboration, Gilead had exclusive options to license worldwide rights to antibodies from certain TGFβ programs being developed by Scholar Rock. Scholar Rock received $80.0 million in proceeds upon signing the agreement and an additional $25.0 million preclinical milestone was achieved in December 2019 for the successful demonstration of efficacy in preclinical in vivo proof-of-concept studies. As of December 19, 2021 the collaboration period has concluded and on January 6, 2022, Gilead agreed that its option exercise period for all programs has been terminated.

Scholar Rock regains rights to a suite of antibodies with novel pharmacological profiles that were discovered over the course of the collaboration.
Of particular note, Scholar Rock has discovered antibodies that selectively inhibit the activation of latent TGFβ1 in the context of fibrotic extracellular matrix and that avoid perturbing TGFβ1 presented by cells of immune system. Such antibodies demonstrated significant antifibrotic activity in a variety of preclinical rodent models and safety at all doses tested in a non-GLP mouse safety study that we intend to publish in 2022.
"The novel anti-fibrotic antibodies discovered during this collaboration demonstrate the unique capabilities of the discovery platform we have built at Scholar Rock," said Gregory Carven, CSO of Scholar Rock. "We are excited to continue the advancement of these assets as a part of the company’s growing preclinical pipeline."

"We made great progress across our portfolio in 2021 and we’re carrying that momentum into 2022," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We recently strengthened our balance sheet through the use of our ATM and taking the $25 million second tranche of our debt facility with Silicon Valley Bank and Oxford Finance so that we have greater flexibility to continue to advance our clinical and pre-clinical programs. We have high conviction in our platform based on the exciting clinical data to date and we believe this puts us in a unique position as we advance our programs to serve patients’ needs."