On January 11, 2021 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 a corporate update and highlighted priorities for 2021 (Press release, Scholar Rock, JAN 11, 2021, View Source [SID1234573843]).

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"2020 was a transformative year for Scholar Rock with significant progress made both clinically and operationally with positive interim data from the TOPAZ trial providing initial proof-of-concept of apitegromab’s potential in SMA and showing the potential benefit of inhibiting the latent forms of growth factors," said Tony Kingsley, President and CEO of Scholar Rock. "In 2021, we will further elucidate the potential of our product candidates through data from our TOPAZ Phase 2 trial in SMA and our DRAGON Phase 1 trial in cancer immunotherapy as well as build upon our scientific platform towards additional indications."

2021 Expected Milestones:

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

Top-line Efficacy and Safety Data from TOPAZ Phase 2 Trial in SMA Anticipated in 2Q21. A total of 58 patients were enrolled across the three cohorts of the TOPAZ clinical trial with one discontinuation to date. Positive six-month interim analysis data were announced in October 2020, demonstrating apitegromab’s potential to improve motor function in patients with Type 2 and Type 3 SMA. The top-line 12-month efficacy and safety data are expected in the second quarter of 2021.
As of January 8, 2021, 56 patients have completed the 12-month study and all 56 have opted into the extension period.

Identification of Second Indication for Apitegromab Planned for 2021. With the demonstration of initial proof-of-concept from the TOPAZ interim analysis, Scholar Rock is actively evaluating multiple other disorders for which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 is a potent and highly selective inhibitor of latent transforming growth factor beta 1 (TGFβ1) activation being developed with an aim of overcoming resistance to and increasing the number of patients who may benefit from, checkpoint inhibitor therapy.

Initial Clinical Response and Safety Data from the DRAGON Trial are Anticipated in the Second Half of 2021. SRK-181 is being evaluated in the two-part DRAGON Phase 1 trial in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Dose escalation in Part A of the DRAGON trial is progressing with the highest planned dose of 2400 mg now being evaluated in Part A1 (SRK-181 as a single-agent) and the 800 mg dose being currently evaluated in Part A2 (SRK-181 in combination with an approved anti-PD-(L)1 therapy).
The DRAGON trial is anticipated to advance to Part B in the second quarter of 2021, which is expected to encompass multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients with locally advanced or metastatic solid tumors for which they have been treated with an approved anti-PD-(L)1 therapy and have demonstrated primary resistance. Patients in Part B will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy.

"We enter 2021 with positive momentum across our programs. We believe we’ve demonstrated the potential of our novel scientific approach to treating a wide array of disease areas for which growth factors play a critical role in disease progression," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "Our strong balance sheet, which can fund operations into 2023, provides flexibility as we continue to invest in developing important therapies for patients in need."

On January 11, 2021 Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) ("Takeda") reported that it will present virtually at the 39th Annual J.P. Morgan Healthcare Conference at 5:20 p.m. ET on Monday, January 11, 2021 / 7:20 a.m. JT on Tuesday, January 12, 2021 (Press release, Takeda, JAN 11, 2021, View Source [SID1234573842]). Investors and the general public are invited to listen to the live webcast with Christophe Weber, president and chief executive officer, here. A replay of the webcast will also be archived at the same location. A link to the webcast and slides for download will also be available on Takeda’s website at View Source

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On January 11, 2021 EQRx, a company committed to developing and delivering important new medicines at lower prices, reported that it has raised $500 million in Series B financing (Press release, EQRx, JAN 11, 2021, View Source [SID1234573841]). Since launching a year ago, EQRx has raised approximately $750 million to advance its mission of bringing new medicines to treat life-threatening and chronic diseases to patients and healthcare systems around the world at prices that are a fraction of the cost of today’s leading therapies.

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The Series B financing includes participation from all Series A investors, life science specialists, world-class mutual funds and private equity funds, sovereign wealth and family offices, and market-leading payers and health systems that cover more than 20% of insured lives in the United States.

"There is an urgent need for change in the industry’s approach to drug pricing, and although challenging, changing the model is not only possible, it is critical," said Alexis Borisy, founder, chairman and chief executive officer of EQRx. "The price of a drug should never be the rate-limiting factor to patient access. EQRx is confronting this issue head-on by employing a disruptive, market-based approach to create true, sustainable access to great medicines at fair prices and reestablish trust and transparency between drug developers and the people who need these medicines."

EQRx is building a highly competitive pipeline of important new drug candidates to address diseases like cancer and inflammatory conditions that combined are projected to represent nearly 40% of the drug spend among the highest cost diseases globally by 2025. By leveraging proven druggable targets and a relentless focus on efficiency, together with deep strategic partnerships with health systems and payers, EQRx anticipates a higher probability of regulatory success, a lower risk-adjusted cost of drug development and a more streamlined access model. The Company estimates that its broad pipeline of innovative therapies has the potential to save the U.S. healthcare system between 50-70% of its current drug spend across the respective therapeutic areas.

In oncology, EQRx has disclosed several late-stage drug candidates currently in development that show promise in the treatment of some of the most common and devastating types of cancers globally—lung cancer, breast cancer and other solid tumors—including:

sugemalimab, a PD-L1 antibody with compelling phase 3 clinical data in non-small cell lung cancer (NSCLC), as well as potential additional applications in gastric cancers, esophageal cancers, and lymphomas. Notably, sugemalimab has received Breakthrough Drug Designation and Orphan Drug Designation from the United States Food and Drug Administration (FDA) for the treatment of certain lymphomas;
almonertinib, an EGFR inhibitor, to treat certain types of NSCLC, which is already approved in China and is in late-stage clinical trials;
EQRx’s PD-1 antibody (formerly known as CS1003), which has received Orphan Drug Designation from the FDA for a type of primary liver cancer; and
lerociclib, a CDK4/6 inhibitor, which targets HR+/HER2- breast cancer, the most common type of the disease.
EQRx’s business model drives cost savings both through the potential use of these medicines as monotherapies, and by enabling biopharma companies to create high-quality, lower-cost combination regimens including EQRx’s drugs through partnerships.

The Company has also entered several strategic collaborations with national and regional health plans and large integrated delivery networks and health systems. "EQRx is the ultimate convener, bringing healthcare stakeholders together in meaningful strategic partnerships to modernize traditional drug manufacturer-to-patient access models globally," added Melanie Nallicheri, co-founder, president and chief operating officer of EQRx. "Our shared goal is to enable effortless prescribing for providers and equal access to important new medicines for people by eliminating barriers, especially high drug costs. With this Series B financing, EQRx is well-poised to make sustainable drug pricing a reality for people, payers and health systems."

On January 11, 2021 -bluebird bio, Inc. (Nasdaq: BLUE) reported its intent to separate its severe genetic disease and oncology businesses into differentiated and independent publicly traded companies (Press release, bluebird bio, JAN 11, 2021, View Source [SID1234573840]). bluebird bio, Inc. will retain focus on severe genetic disease (SGD) and will launch its oncology business ("Oncology Newco") as a new entity. bluebird bio’s Board of Directors approved the intent to separate into two companies and it is anticipated that the spin out of Oncology Newco is to be tax-free to shareholders, subject to receipt of a favorable Internal Revenue Service (IRS) ruling.

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Upon completion of the separation, current chief bluebird, Nick Leschly, will lead Oncology Newco as Chief Executive Officer and will take on the role of Executive Chair for bluebird bio, Inc. Current President of the SGD business, Andrew Obenshain will continue his leadership as Chief Executive Officer of bluebird bio, Inc. Further, current Chair of bluebird’s Board of Directors, Daniel Lynch, will become Chair of the Board for Oncology Newco.

"We are excited and energized to begin this new year with so much opportunity ahead. Over the last decade, bluebird bio has pioneered development of gene and cell therapies for severe genetic diseases and oncology – delivering transformative outcomes for patients. Through the tenacity and incredible work of our bluebirds, our first commercial product is now approved in Europe and we are now on the cusp of several potential product approvals with a strong pipeline of earlier oncology research candidates on the horizon. This is a position few biotech companies have been able to attain," said Nick Leschly, chief bluebird. "After careful strategic review, it is clear to us that the two businesses are best served by independent leadership and teams to drive distinct strategic and operational objectives. Specifically, we believe it is the right time to double down on the respective businesses to fully enable and optimize the continued innovation, development and deployment of transformative gene and cell therapies for the patients we serve."

"In close collaboration with the Board of Directors, bluebird bio leadership has conducted a thorough assessment of the business overall and examined a range of options for the future," said Daniel Lynch, Chair of bluebird bio’s Board of Directors. "Based on this review, we collectively believe this strategic decision is in the best interest of patients, employees, investors and other stakeholders. We are committed to working together through this transformative process to ensure each company is optimized with the right teams in place for progressing these therapies through the regulatory process into commercialization, harnessing the power of the pipeline to continue creation of innovative medicines, establishing and rapidly growing product revenue, and creating value for shareholders."

Launching Severe Genetic Disease and Oncology for Bold Futures

bluebird bio intends to ensure both SGD and Oncology Newco are established as independent organizations with enhanced therapeutic focus and strong financial foundations. The company believes this approach will provide both entities with the ability to achieve the following:

Enhanced resource allocation and capital considerations for each company
Therapeutic expertise and focus to more effectively execute and deliver on milestones
Streamlined and simplified operations
Tailored investment theses to attract an appropriately suited shareholder base
Sustained patients first culture and innovation mindset
Increased strategic flexibility
By establishing this foundation in two new environments, the company believes each entity will be in a stronger position to deliver on their goals:

Severe Genetic Disease

Focus on delivery of Core 3 therapies in β-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the United States and Europe
Expand access and reimbursement for our commercial product, ZYNTEGLO (betibeglogene autotemcel), in Europe
Increase addressable patient populations through geographic expansion, label expansions, and product profile enhancement
Build on our expertise in gene therapy manufacturing through commercialization, significant process enhancements, and next generation technologies
Continue to explore innovative tools and technologies to ultimately bring these transformative medicines to more patients
Oncology Newco

Support commercial success of investigational B-cell maturation antigen (BCMA) directed chimeric antigen receptor (CAR) T cell therapy, idecabtagene vicleucel (ide-cel), in multiple myeloma and continued development of investigational bb21217 product candidate; advancing into earlier lines and continuing to innovate
Deliver on the oncology pipeline of cellular therapies with a focus on non-Hodgkin’s lymphoma, acute myeloid leukemia, next-generation multiple myeloma and solid tumors
Advance next generation product cycling engine designed to rapidly build, test, learn and improve with an overarching goal of 1-2 investigational new drugs (INDs) in each of 2021 and 2022
bluebird bio Adds Additional Oncology Expertise to Board of Directors

As bluebird bio continues to build out therapeutic expertise within its Board of Directors, the company has appointed Ramy Ibrahim, M.D. to its Board of Directors. Dr. Ibrahim is a recognized leader in clinical development in immunotherapy and cell therapy. He is currently serving as a consultant for the Parker Institute for Cancer Immunotherapy (PICI) where he was recently the Chief Medical Officer and built the clinical capabilities within the institute as well as worked with renowned cell therapy experts to support building world class cell therapy startups. Before joining PICI, Dr. Ibrahim was the vice president and Global Therapeutic area head for Immuno-Oncology clinical development for AstraZeneca/MedImmune, leading the global clinical team developing multiple immunotherapies. In addition, as a member of the Bristol-Myers Squibb Immuno-oncology program, he served on the Yervoy (ipilimumab) clinical team supporting the program from early phase II through multiple global launches of the first FDA-approved immune checkpoint inhibitor. In addition to his engagement with investment firms, Dr. Ibrahim also serves on the Scientific Advisory Board of Harpoon and on the Board of Directors for Surface Oncology.

bluebird bio also acknowledges the significant and impactful contributions of Dr. David Schenkein, who after eight years, is stepping down from the bluebird bio Board of Directors.

Financial Summary

bluebird bio preliminary and unaudited cash, cash equivalents and marketable securities balance as of December 31, 2020 was approximately $1.3B. At the time of separation, bluebird bio plans to capitalize each business with sufficient cash runway to achieve value creating milestones. In preparation for the separation, bluebird bio will continue to prudently and carefully manage the cost structure of each business while evaluating dedicated financial and strategic funding sources. bluebird bio expects to incur increased transactional and separation expenses through the completion of the transaction as it works to separate and transition the two businesses. bluebird bio will provide additional financial details closer to the date of separation.

Transition and Timing

Specific details regarding the companies including financial statements, the name of Oncology Newco as well as executive management teams and the respective Board of Directors (BOD) for each company will be provided at a later date. Expected executive team, employee and BOD transitions will be effective as of the closing of the separation anticipated to be in the Q4 2021 timeframe. bluebird bio anticipates both companies will be headquartered in Cambridge, Mass. European operations will remain with bluebird bio and the SGD business. Facilities, research and manufacturing operations in Seattle, Wash. and Durham, N.C. will migrate with the Oncology Newco. The separation is subject to customary closing conditions, including the effectiveness of a Form 10 registration statement with the U.S. Securities and Exchange Commission, receipt of a private letter ruling from the IRS and tax opinion from counsel, and final approval by bluebird bio’s Board of Directors. There can be no assurance regarding the ultimate timing of the separation or that the separation will ultimately occur.

Goldman Sachs & Co. LLC is serving as exclusive financial adviser to bluebird bio and Goodwin Procter LLP is serving as its legal counsel.

Webcast Information

bluebird bio will hold a conference call to discuss the news on Monday, January 11 at 8:00 a.m. ET. Investors may listen to the call by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 225-6277.

In addition, members of the management team will participate in the 39th Annual J.P. Morgan Healthcare Conference, Monday, January 11 at 2:50 p.m. ET.

To access the live webcast of bluebird bio’s presentations, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source Replays of the webcast will be available on the bluebird bio website for 90 days following the event.

On January 11, 2021 Novocure (NASDAQ: NVCR) reported operating statistics and preliminary, unaudited net revenues and cash balances for the fourth quarter and full year 2020 (Press release, NovoCure, JAN 11, 2021, View Source [SID1234573839]). Novocure plans to discuss these results with investors at the 39th Annual J.P. Morgan Virtual Healthcare Conference. Novocure also plans to highlight key clinical and product development programs.

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(1) The preliminary, unaudited results described in this press release are estimates only and are subject to revision until the company reports its full, audited financial results for the fourth quarter and full year 2020 on or about Feb. 25, 2021.

(2) An "active patient" is a patient who is receiving treatment under a commercial prescription order as of the measurement date, including patients who may be on a temporary break from treatment and who plan to resume treatment in less than 60 days.

(3) A "prescription received" is a commercial order for Optune or Optune Lua that is received from a physician certified to treat patients for a patient not previously on Optune or Optune Lua. Orders to renew or extend treatment are not included in this total.

"Our track record of execution extended throughout 2020 with significant achievements furthering our three overarching priorities," said William Doyle, Novocure’s Executive Chairman. "We sustained our commercial momentum with $494 million in net revenues, representing 41% annual revenue growth; launched three new clinical trials, expanding our development pipeline to include eight ongoing global studies involving nearly 3,000 patients across seven solid tumor cancers; and, increased our investments in product development."

"We continue to build on twenty years of innovation and execution to pioneer an emerging modality in cancer care," said Asaf Danziger, Novocure’s CEO. "Through our clinical and product development efforts, we believe we are just beginning to unlock the potential of the Tumor Treating Fields platform to extend survival in some of the most aggressive forms of cancer. We look forward to providing a full company update and discussing our 2020 financial results on our February conference call."

Fourth quarter 2020 operating statistics and preliminary financial highlights

On a preliminary, unaudited basis, for the quarter ended December 31, 2020, net revenues were $144.0 million, representing 45% growth compared to the fourth quarter 2019, and full year 2020 net revenues were $494.4 million, representing 41% growth compared to full year 2019.

In the United States, net revenues totaled $97.7 million in the quarter ended December 31, 2020, representing 48% growth compared to the same period in 2019.
In Germany and other EMEA markets, net revenues totaled $33.8 million in the quarter ended December 31, 2020, representing 31% growth compared to the same period in 2019.
In Japan, net revenues totaled $7.9 million in the quarter ended December 31, 2020, representing 42% growth compared to the same period in 2019
In Greater China, net revenues totaled $4.5 million in the quarter ended December 31, 2020, representing 132% growth compared to the same period in 2019.
We recorded $9 million and $36 million in revenues from Medicare fee-for-service beneficiaries billed under the coverage policy effective on September 1, 2019 for the three and twelve month periods ended December 31, 2020, respectively. We have gained a good understanding of how to ensure timely processing of Medicare claims and have sufficient experience to recognize approximately two-thirds of the expected contribution from Medicare beneficiaries. In the fourth quarter of 2020, we also recognized approximately $11 million in incremental net revenues compared to the first half of 2020 resulting from the successful appeal of previously denied claims for Medicare fee-for-service beneficiaries billed prior to established coverage.

There were 3,411 active patients at December 31, 2020, representing 17% growth compared to December 31, 2019, and one percent growth compared to September 30, 2020.

In the United States, there were 2,193 active patients at December 31, 2020, representing 12% growth compared to December 31, 2019.
In Germany and other EMEA markets, there were 953 active patients at December 31, 2020, representing 25% growth compared to December 31, 2019.
In Japan, there were 265 active patients at December 31, 2020, representing 38% growth compared to December 31, 2019.
Additionally, 1,411 prescriptions were received in the quarter ended December 31, 2020, representing two percent growth compared to the same period in 2019, and three percent growth compared to the quarter ended September 30, 2020. In the quarter ended December 31, 2020, 1,160 Optune prescriptions were written for patients with newly diagnosed glioblastoma.

In the United States, 962 prescriptions were received in the quarter ended December 31, 2020, representing a four percent decrease compared to the same period in 2019.
In Germany and other EMEA markets, 349 prescriptions were received in the quarter ended December 31, 2020, representing 22% growth compared to the same period in 2019.
In Japan, 100 prescriptions were received in the quarter ended December 31, 2020, representing eight percent growth compared to the same period in 2019.
At December 31, 2020, on a preliminary, unaudited basis, Novocure had $235 million in cash and cash equivalents and $608 million in short-term investments, for a total balance of $843 million in cash, cash equivalents and short-term investments. This represents an increase of $608 million in cash and investments since September 30, 2020.

Anticipated clinical milestones

Data from phase 2 pilot HEPANOVA trial in advanced liver cancer (Q1 2021)
Data from phase 2 pilot EF-31 trial in gastric cancer (2021)
Interim analysis of phase 3 pivotal LUNAR trial in non-small cell lung cancer (2021)
Interim analysis of phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2021)
Interim analysis of phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2021)
Data from phase 3 pivotal METIS trial in brain metastases (2022)
Data from phase 2 pilot EF-33 trial with high-intensity arrays in recurrent glioblastoma (2022)
Final data from phase 3 pivotal LUNAR trial in non-small cell lung cancer (2023)
Final data from phase 3 pivotal PANOVA-3 trial in locally advanced pancreatic cancer (2023)
Final data from phase 3 pivotal INNOVATE-3 trial in recurrent ovarian cancer (2023)
Fourth quarter and full year 2020 financial results conference call

Novocure will host a conference call and webcast to discuss fourth quarter and full year 2020 financial results at 8 a.m. EST on Thursday, February 25, 2021. 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 3965899.

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.