On December 1, 2021 2seventy bio, Inc. (Nasdaq: TSVT), an emerging immuno-oncology company, reported financial results and recent highlights for the third quarter ended September 30, 2021 (Press release, 2seventy bio, DEC 1, 2021, View Source [SID1234596339]).

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"We launched 2seventy bio a few short weeks ago and I’m highly encouraged by the way our team has taken this challenge head on," said Nick Leschly, chief kairos officer. "Our urgency to bring transformative cell therapies forward is driven by the ultimate goal, which is to provide more time to patients and their families devastated by cancer. We have hit the ground running, with the first IND acceptance for our program in AML, and another anticipated in the bNHL program by the end of the year. Exciting times are ahead, and we are fired up for 2022 as ABECMA continues to deliver for MM patients and our product engine drives more disruptive next-gen cell therapies forward in both liquid and solid tumors."

RECENT HIGHLIGHTS

COMPANY LAUNCH – On November 4, 2021, 2seventy bio launched as an independent, publicly traded company with a robust cell therapy pipeline across a range of hematologic and solid tumors including the SC-DARIC33 and bbT369 candidates that are planned, subject to IND acceptance, to enter the clinic by the first half of 2022. 2seventy bio’s portfolio also includes a development and 50/50 U.S. commercialization partnership with Bristol Myers Squibb (BMS) for ABECMA, a first-in-class B-cell maturation antigen (BCMA)-directed CAR T cell immunotherapy, in relapsed or refractory multiple myeloma.
SC-DARIC33 IND – Today, 2seventy bio is announcing that the FDA has accepted the IND for SC-DARIC33, an investigational, pharmacologically controlled CD33-targeted autologous T cell product, developed in collaboration with Seattle Children’s Therapeutics for the potential treatment of acute myeloid leukemia (AML). PLAT-08, the Phase 1 study of SC-DARIC33 in relapsed/refractory pediatric AML, led by Seattle Children’s Therapeutics, is now open and enrolling patients (NCT: 05105152). This study is a first-in-human investigation of the DARIC T cell platform.
ASH PRESENTATIONS – On November 4, 2021, 2seventy bio announced that it will present data from its portfolio of oncology cell therapies at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. These presentations include new pre-clinical data on SC-DARIC33, and in partnership with BMS, updated results from the ongoing Phase 1 CRB-402 study of the BCMA-targeted CAR T cell therapy bb21217 in patients with relapsed and refractory multiple myeloma (RRMM). In addition, a range of analyses will be presented from the pivotal KarMMa trial of ide-cel, in partnership with BMS.
UPCOMING ANTICIPATED MILESTONES

Acceptance of investigational new drug (IND) application for bbT369 in bNHL by the end of 2021.
Presentation of new preclinical data from the SC-DARIC33 program at the 63rd ASH (Free ASH Whitepaper) Annual Meeting in December 2021.
Presentation of clinical data from the ongoing CRB-402 study of bb21217 at the 63rd ASH (Free ASH Whitepaper) Annual Meeting in December 2021.
SELECT THIRD QUARTER AND YEAR-TO-DATE 2021 FINANCIAL RESULTS

The results for the period ended September 30, 2021, have been prepared on a carve-out basis and are derived from bluebird bio, Inc.’s consolidated financial statements and accounting records. Upon separation on November 4, 2021, bluebird bio made a contribution to 2seventy bio of approximately $441.5 million in cash and cash equivalents, which is expected to be sufficient to fund current planned operations for at least the next twelve months.
Revenues: Total revenues were $19.3 million for the three months ended September 30, 2021 compared to $18.4 million for the three months ended September 30, 2020. Total revenues were $38.5 million for the nine months ended September 30, 2021 compared to $238.2 million for the nine months ended September 30, 2020. The increase for the three-month period was primarily driven by revenue recognized under our collaboration arrangement with BMS. The decrease in the nine-month period was primarily driven by revenue recorded in connection with the May 2020 BMS contract modification in the second quarter of 2020.
ABECMA Revenue: BMS reported total U.S. revenues of $67 million for ABECMA (idecabtagene vicleucel; ide-cel) in the third quarter of 2021. 2seventy bio reported net collaboration revenue of $12.3 million for the third quarter of 2021, which includes our share of revenue and costs associated with the commercialization of ABECMA in the U.S.
R&D Expenses: Research and development expenses were $61.1 million for the three months ended September 30, 2021 compared to $72.3 million for the three months ended September 30, 2020. Research and development expenses were $202.4 million for the nine months ended September 30, 2021 compared to $227.6 million for the nine months ended September 30, 2020. The decrease for the three-month period was primarily driven by decreased R&D costs under our collaboration with BMS and decreased manufacturing expenses. The decrease for the nine-month period was primarily driven by decreased manufacturing expenses.
SG&A Expenses: Selling, general and administrative expenses were $23.0 million for the three months ended September 30, 2021 compared to $22.1 million for the three months ended September 30, 2020. Selling, general and administrative expenses were $69.0 million for the nine months ended September 30, 2021 and September 30, 2020. The increase for the three-month period was primarily driven by an increase in employee compensation, benefit, and other headcount related expenses.
Net Loss: Net loss was $60.0 million for the three months ended September 30, 2021 compared to $72.1 million for the three months ended September 30, 2020. Net loss was $231.2 million for the nine months ended September 30, 2021 compared to $43.3 million for the nine months ended September 30, 2020.

On December 1, 2021 Tempus, a leader in artificial intelligence and precision medicine, reported the expansion of its multi-year agreement with Janssen Research & Development, LLC (Janssen) (Press release, Tempus, DEC 1, 2021, View Source [SID1234596357]). The expanded scope is anchored in new AI-based work, in which Tempus will collaborate with a multidisciplinary team of data scientists at Janssen to enhance the discovery and development of therapies for oncology indications using AI/ML and real-world evidence (RWE). This agreement leverages Tempus’ platform, including its multimodal data library, genomic sequencing offerings, and TIME Trial Network of clinical sites to advance the recruitment of patients.

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Tempus’ platform is well positioned to solve many of the challenges clinical trial sponsors face, especially finding rare biomarker-driven patient populations. This work leverages Tempus’ AI capabilities to jointly create algorithms intended to further patient pre-screening efforts for specific cancer indications, including biomarker-selected cohorts. Tempus’ multidisciplinary team’s expertise spans clinical RWE, computational biology, software engineering, machine learning, and trial matching program management, and is a key factor in successfully leveraging an AI-enabled approach to trial recruitment.

"We’re excited to expand our collaboration to apply the Tempus AI platform," said Ryan Fukushima, Chief Operating Officer of Tempus. "We will explore leveraging our data to develop and co-develop novel AI applications to identify the best treatment options for patients in need, and ultimately accelerate the drug development process, bringing novel medicines to patients in a fraction of the time."

On December 1, 2021 Curie Therapeutics Inc. ("Curie"), a biotechnology company incubated by Atlas Ventures, Access Biotechnology and RA Capital Management, reported a Series A investment of $75 million by its founding syndicate to continue the development of its multimodal pipeline of precision radiopharmaceuticals to a broad range of high unmet need solid tumors (Press release, Curie Therapeutics, DEC 1, 2021, View Source [SID1234596380]).

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Commenting on the emergence of Curie from stealth, Dr. Simon Read, Curie Founder and Chief Executive Officer, said, "We envision a world where cancer care is transformed by precision radiopharmaceuticals and a future in which these drugs are increasingly integrated into mainstream treatment."

He added, "We founded Curie to maximize the potential of targeted radiopharmaceuticals for frank tumor ablation, as well as to harness the growing understanding of how radiobiology of targeted therapies can favorably modulate the tumor microenvironment as part of a rational sequence of care. Our team is unique. We have bought together seasoned leaders from pharmaceutical biotech R&D with radiobiology and radiochemistry experts drawn from both industry and academia. Backed by a syndicate of three of the foremost incubating venture firms, we have a pipeline underway and key radionuclide supply agreements for both alpha and beta radionuclide strategies to support our vision."

Dr. Dan Becker, Partner at Access Biotechnology and a member of Curie’s Board of Directors (BoD), said "Radiopharmaceuticals are emerging as potent, safe, and commercially viable options for cancer patients. While late-stage therapeutic radiopharmaceutical pipelines are predominantly focused on metastatic castration resistant prostate cancer and neuroendocrine tumors, we see a much broader opportunity to drug a broad spectrum of targets with our high therapeutic index, precision radiopharmaceuticals."

Dr. Kevin Bitterman, Partner at Atlas Ventures and a member of Curie’s BoD said, "All three syndicate members showed an early interest in the radiopharmaceutical space. We have been incubating Curie for 18 months with a focus on building a fully integrated company in which both peptidic and non-peptidic ligand R&D, radiochemistry, CMC and clinical translation can occur under one roof."

Dr. Josh Resnick, Managing Director of RA Capital Management and a member of Curie’s BoD, added, "This founding team has the ligand discovery breadth, company formation experience, and radiobiology and CMC experience required to build the leading precision radiopharmaceutical company. We are delighted to co-lead the series A round with Access Biotechnology and Atlas Venture."

Curie Management and Board of Directors

Curie’s management includes biotech leaders with broad experience across peptide discovery and development, radiochemistry, and in building top tier clinical stage peptide, radiopharmaceutical, and platform companies, including founder Simon Read, Ph.D. as CEO (former CSO of Ra Pharma, former Entrepreneur in Residence at Atlas Venture), Alonso Ricardo, Ph.D. as CSO (former Chief Technology Officer of Ra Pharma, former Entrepreneur in Residence at RA Capital Management), and Sandy Mong, M.D. as SVP of Business Development and Operations (former VP of Business Development and Operations of BridgeBio Pharma, former Entrepreneur in Residence at Atlas Venture). The broader team includes ligand discovery, radiopharmacology, radiochemistry talent from public and private peptide companies, radiopharmaceutical companies, as well as academia.

Curie’s BoD consists of Kevin Bitterman, Ph.D. (Partner, Atlas Venture), Dan Becker, M.D., Ph.D. (Partner, Access Biotechnology), Josh Resnick, M.D. (Managing Director, RA Capital Management), and Simon Read, Ph.D. (CEO, Curie Therapeutics).

On December 1, 2021 Molecular Targeting Technologies, Inc. (MTTI) and Evergreen Theragnostics, Inc., reported an agreement in which Evergreen will manufacture MTTI’s EvaThera platform of radiopharmaceuticals (Press release, Molecular Targeting Technologies, DEC 1, 2021, View Source [SID1234632260]).

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The EvaThera platform is a new generation of Evans blue-based molecules with strong affinity for albumin, which extends the blood half-life of the radiopharmaceutical, potentially leading to improved outcomes. MTTI’s leading pipeline candidates are both Lu-177 based radiotherapeutics: EBTATE, targeting neuroendocrine and other SSTR2 expressing tumors, and EBRGD, targeting integrin expressing cancers like glioblastoma multiforme.

"The Evergreen team brings deep experience in manufacturing and nationwide distribution of Lu-177 products. This agreement is key to a robust and reliable supply in North America for our upcoming clinical trials and beyond," said Chris Pak, President & CEO of MTTI.

Evergreen will manufacture MTTI clinical supplies from its newly commissioned facility in Springfield, NJ and will leverage the team’s broad experience with Lu-177 based radiotherapeutics by providing comprehensive supply chain management from isotope sourcing to drug delivery at the clinical trial site.

"We are very pleased to support MTTI by providing quality GMP manufacturing for their very promising platform of products. The technology behind the EvaThera platform has the potential to make a meaningful improvement in the lives of patients," said James Cook, President & CEO of Evergreen.

On December 1, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that the US Food and Drug Administration (FDA) is allowing the Company’s Investigational New Drug (IND) application to study WP1122 for the treatment of Glioblastoma Multiforme (GBM) to go forward (Press release, Moleculin, DEC 1, 2021, View Source [SID1234596322]). With this IND now cleared, Moleculin plans to initiate a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The Company believes WP1122 has significant potential as both an antiviral therapy and as a cancer therapy. Moleculin recently announced its plans to initiate a Phase 1a clinical trial of WP1122 in healthy volunteers in the United Kingdom to facilitate future testing in COVID-19 patients. This new US IND sets the stage for parallel development of WP1122 as a cancer therapy. Consistent with its strategy of leveraging external funding for many of its clinical trials, Moleculin intends to seek opportunities for an investigator-initiated clinical trial of WP1122 in cancer patients in 2022.

"This IND underscores our dual pronged approach to the development of WP1122 for the treatment of both certain types of cancers and viruses. In addition to the trial in the UK designed to position WP1122 as an antiviral therapy, we can now be advancing the cancer therapy path in parallel. Along with GBM, we believe WP1122 has the potential to be well suited as a treatment for other highly glycolytic cancers such as pancreatic cancer," commented Walter Klemp, Chairman and CEO of Moleculin.

GBM is the most aggressive malignant primary brain tumor and remains as an incurable tumor with a median survival of only 15 months1. It is the most common malignant primary brain tumor making up 54% of all gliomas and 16% of all primary brain tumors,2 and despite advancements, survival rates for patients with GBM have shown no notable improvement in population statistics in the last three decades.3 The average annual age-adjusted incidence rate of GBM is 3.19 per 100,000 persons in the United States.4

1 Koshy M, Villano JL, Dolecek TA, Howard A, Mahmood U, Chmura SJ, et al. Improved survival time trends of glioblastoma using the SEER 17 population-based registries. J Neuro Oncol. 2012;107(1):207–12

2 Ostrom QT, Gittleman H, Farah P, Ondracek A, Chen Y, Wolinsky Y, et al. CBTRUS statistical report: Primary brain and central nervous system tumors diagnosed in the United States in 2006–2010. Neuro Oncol. 2013;15 Suppl:2ii–56.

3 De Vleeschouwer S, editor. Brisbane (AU): Codon Publications; 2017 Sep 27.

4 Thakkar J, Dolecek TA, Horbinski C, Ostrom QT, Lightner DD, Barnholtz-Sloan JS, et al. Epidemiologic and molecular prognostic review of glioblastoma. Cancer Epidemiol. Biomarkers Rev. 2014;23(10):1985–96.