On March 16, 2021 GRAIL, Inc., a healthcare company whose mission is to detect cancer early, reported it is joining forces with AccessHope to offer a world-class service experience to people who use Galleri, GRAIL’s first-of-kind multi-cancer early detection blood test (Press release, Grail Bio, MAR 16, 2021, View Source [SID1234576742]). This collaboration is part of GRAIL’s effort to help patients and their healthcare providers achieve favorable outcomes by making the test experience, from blood draw to cancer care, as seamless as possible.

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AccessHope, a company that provides cancer expertise to employers and their healthcare partners, will offer Galleri users coordination services during the test experience, including access to a support team for guidance and information. AccessHope also will provide support to physicians as they determine next steps to evaluate a positive Galleri test result.

"Most cancers are diagnosed too late, when outcomes are poor," said Dr. Joshua Ofman, chief medical officer and head of external affairs at GRAIL. "The introduction of Galleri as a complement to recommended cancer screening tests provides an opportunity to fundamentally change the way that we detect cancer. Best-in-class expert services like those offered by AccessHope will be an essential part of GRAIL supporting providers and their patients from test to diagnosis to treatment."

GRAIL previously announced it will introduce Galleri in the second quarter of 2021. An earlier version of Galleri demonstrated the ability to detect more than 50 types of cancers — over 45 of which lack recommended screening tests today — with a low false positive rate of less than 1%. When a cancer signal is detected, Galleri can determine where in the body the cancer is located with high accuracy, all from a single blood draw. This is critical to help guide diagnostic follow-up and care.

"The rising cost of cancer care — coupled with the extraordinary rate of innovation and changes in best practices — make early detection even more vital when it comes to improving a patient’s healthcare experience, outcomes, value, and care," said Mark Stadler, CEO of AccessHope. "Our mission at AccessHope is to deploy the latest cancer care knowledge to the places and people who need it most when it is most valuable. Providing support services for Galleri offers another way we can touch more lives and extend our mission."

Galleri, which will require a prescription, will be available initially through partner health systems, medical practices, and self-insured employers.

Cancer is projected to become the world’s leading cause of death in 2021. In the U.S., more than 600,000 people died from cancer last year. This is in large part because the majority of cancers are found too late when outcomes are often fatal. Recommended screening tests in the U.S. save lives, but they only cover five cancers and screen for a single cancer at a time. In fact, cancers responsible for 71% of cancer deaths have no recommended early detection screening at all.

On March 16, 2021 Be Biopharma ("Be Bio"), whose mission is to pioneer the emerging new category of engineered B cells as medicines, reported that Joanne Smith-Farrell, Ph.D., has been appointed Chief Executive Officer and Director (Press release, Be Biopharma, MAR 16, 2021, View Source [SID1234576741]). Dr. Smith-Farrell will be joined by Chief Scientific Officer, Richard Morgan, Ph.D., a leading expert in cell and gene therapies.

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Be Bio’s rapidly growing team of scientists, drug developers, manufacturing experts, and business builders is leading the creation of a new category of cellular therapies, engineered B cell medicines. B cells are exquisitely designed by nature to embody a unique mix of functionalities, including prolific protein production, tissue targeting, and durable engraftment in cellular niches. Be Bio was founded by Longwood Fund in October 2020 with a $52 million Series A investment led by Atlas Ventures and RA Capital, and joined by Alta Partners and Takeda Ventures to unlock this rich biology by precisely engineering B cells as therapies to develop a broad pipeline of potent and potentially curative cellular medicines.

Prior to joining Be Bio as Chief Executive Officer, Dr. Smith-Farrell was Chief Operating Officer and Business Unit Head, Oncology, at bluebird bio, where she led the growth of bluebird Oncology from an early single-candidate effort into a leading oncology cell therapy business. Prior to this, she held executive leadership roles as Chief Business Officer of bluebird bio, Vice President of Transactions at Merck, and Vice President of Business Development at Pfizer, as well as executive positions in public and private biotechs. Prior to entering the biopharmaceutical industry, she worked in the healthcare practice at The Boston Consulting Group. Dr. Smith-Farrell did her postdoctoral research in Biomedical Engineering in Bob Langer’s lab at the Harvard-MIT Division for Health Science and Technology and holds a Ph.D. in Physics from The Catholic University of America and a B.S. in Physics and Mathematics from Vanderbilt University.

"Be Bio’s mission – to unleash the power of B cells, nature’s protein factories, on many of humanity’s most challenging diseases – is an inspiring and humbling journey to be joining," said Dr. Smith-Farrell. "It has been a great privilege to participate in the birth of the first generation of cell therapies to come to market, and to witness, first-hand, cell therapy’s power to transform the lives of patients with devastating diseases. Be Bio’s aspiration, fueled by the broad utility of engineered B cell medicines to offer previously impossible solutions across a wide array of therapeutic areas, takes the potential of cell therapy to an entirely new level."

Rick Morgan, Ph.D., joins Be Bio as Chief Scientific Officer, and brings decades of experience as one of the pioneers of cell and gene therapy. Most recently, Dr. Morgan was Senior Vice President of Immunogenetics at Editas Medicine, where he focused on genome engineering to produce off-the-shelf cell medicines for cancer. Prior to that, he was Vice President of Immunotherapy at bluebird bio in 2013, where he led pre-clinical activities for bluebird’s first oncology medicine, the anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy idecabtagene vicleucel (ide-cel)—the first CAR-T for the treatment of multiple myeloma filed in the U.S. and Europe. He started his career at the National Institutes of Health, where he conducted groundbreaking research in the development of gene therapy for genetic diseases such as hemophilia, HIV/AIDS, and cancer immunotherapy. He was a member of the team that published the first approved human gene transfer experiment in 1990, and was also the first to report the successful use of T-cell receptor gene therapy for the treatment of cancer in 2006.

"By exploiting the intrinsic drug-like properties of B cells, we can make redosable medicines with superior pharmacokinetic profiles that can be administered without toxic conditioning regimens," said Dr. Morgan. "Be Bio’s ability to engineer B cells is a true paradigm shift in gene therapy that creates major opportunities to treat diseases such as cancer, autoimmune conditions, infectious disease, and protein deficiencies. As CSO, I am excited to have the rare opportunity to shape the development of a new class of medicine from the very start."

"Extraordinary science attracts extraordinary leaders," said David Steinberg, Longwood General Partner, Director and and outgoing Chief Executive Officer, Be Bio. "For over 25 years, Dr. Smith-Farrell has been leading teams that are committed to conquering cancer and rare diseases, most recently at bluebird bio where she built a 400 person oncology cell therapy business unit. We are very fortunate to have her joining alongside Dr. Morgan, an internationally recognized trailblazer in cell and gene therapy and a member of an elite group of scientists who have successfully developed these groundbreaking medicines. Together, Joanne and Rick will be invaluable to Be Bio as they lead our efforts to unlock the potential of B cell medicines, bringing transformational therapies to patients in need."

On March 16, 2021 aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, reported that it will report fourth quarter and full year 2020 financial results and provide a corporate update after the market close on Tuesday, March 23, 2021 (Press release, aTyr Pharma, MAR 16, 2021, https://investors.atyrpharma.com/news-releases/news-release-details/atyr-pharma-webcast-conference-call-reporting-fourth-quarter-and [SID1234576740]). Management will host a conference call and webcast to review the results and provide an operational update.

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On March 16, 2021 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that the U.S. Food and Drug Administration (FDA) has scheduled the pre-approval inspection at the ROLONTIS (eflapegrastim) manufacturing site in May 2021 (Press release, Spectrum Pharmaceuticals, MAR 16, 2021, View Source [SID1234576739]). In October 2020, the company received notification from the agency that it would defer its decision on the BLA because an inspection of the Hanmi Bioplant in South Korea could not be conducted during the review cycle due to restrictions on travel related to the COVID-19 pandemic.

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"I am thrilled that the FDA informed us that they will be conducting a pre-approval inspection of the ROLONTIS manufacturing facility in May," said Joe Turgeon, President and CEO of Spectrum Pharmaceuticals. "We believe the pre-approval inspection marks the final step in the ROLONTIS review process."

About ROLONTIS

ROLONTIS is a novel, long-acting granulocyte colony-stimulating factor (G-CSF) seeking an indication for the treatment of neutropenia in patients receiving myelosuppressive anti-cancer drugs. The BLA for ROLONTIS is supported by data from two identically designed Phase 3 clinical trials, ADVANCE and RECOVER, which evaluated the safety and efficacy of ROLONTIS in 643 early-stage breast cancer patients for the treatment of neutropenia due to myelosuppressive chemotherapy. In both studies, ROLONTIS demonstrated the pre-specified hypothesis of non-inferiority (NI) in duration of severe neutropenia (DSN) and a similar safety profile to pegfilgrastim. ROLONTIS also demonstrated non-inferiority to pegfilgrastim in the DSN across all 4 cycles (all NI p<0.0001) in both trials.

On March 11, 2021 Prelude Therapeutics Inc. ("Prelude", "the Company", "we", "our") (Nasdaq: PRLD), a clinical-stage precision oncology company, reported its financial results for the fourth quarter and full year ended December 31, 2020 and provided an update on recent developments (Press release, Prelude Therapeutics, MAR 16, 2021, View Source [SID1234576737]).

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"2020 marked a highly transformational year for Prelude with the successful completion of an initial public offering and progress across our three clinical-stage programs," said Kris Vaddi, PhD, Chief Executive Officer of Prelude Therapeutics. "We are firmly committed to bringing novel therapies to patients in areas of high unmet need, and we have entered 2021 with both the momentum and resources that we believe will carry us through several key milestones. Notably, we are pleased to announce the completion of dose escalation activities in our ongoing Phase 1 trial of PRT543, our lead PRMT5 inhibitor, in patients with advanced solid tumors and hematologic malignancies, and that we will soon begin initiating additional expansion cohorts. We look forward to sharing clinical data for this program, as well as for our second PRMT5 inhibitor, PRT811, in the second half of this year."

Dr. Vaddi added, "The ongoing Phase 1 trial of our third clinical candidate, PRT1419, an MCL1 inhibitor, also continues to progress, with the addition of dose expansion cohorts expected in the second half of the year. Finally, the continued advancement of our preclinical programs remains a high priority for us in 2021, with the anticipated submission of an IND application for PRT2527, our CDK9 inhibitor, in the second half of this year."

Recent Highlights and Upcoming Milestones

PRT543

Phase 1 Dose Escalation Complete; Additional Expansion Cohorts to Open in the Second Quarter. The Company reported that the dose escalation portion of the Phase 1 trial of its lead product candidate, PRT543, is now complete, and that a recommended expansion dose with adequate safety, pharmacokinetics, and target engagement profile has been established. PRT543 is designed to be a potent, selective, and oral inhibitor of PRMT5. The dose expansion portion of the Phase 1 trial is currently open for the patient cohort with adenoid cystic carcinoma, and will be open for additional patient cohorts with solid tumor and hematologic malignancies early in the second quarter. As previously announced, preliminary data from the dose escalation portion of the trial demonstrated early signs of clinical activity and tolerability. The Company anticipates presenting initial clinical data from the Phase 1 trial at medical meetings in the second half of 2021.

Preclinical Data to be Featured at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. Three preclinical presentations on PRT543 will be featured at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting being held April 10-15, 2021 in a virtual setting. A copy of the posters, titled "PRMT5 inhibition downregulates MYB and NOTCH1 signaling, key molecular drivers of adenoid cystic carcinoma" (abstract 1138), "PRMT5 inhibition epigenetically regulates DNA damage response pathways in cancer cells and sensitizes to chemotherapy and PARP inhibition" (abstract 1185), and "PRMT5 inhibition regulates alternative splicing and DNA damage repair pathways in SF3B1 R625C expressing uveal melanoma cells" (abstract 1137), will be available in the Publications section of the Prelude Therapeutics website following the conclusion of the meeting.
PRT811

Phase 1 Dose Expansion Cohorts Expected to Commence in Mid-2021. The dose escalation portion of the Company’s Phase 1 trial of its second clinical product candidate, PRT811, which is designed to be a potent, selective, and brain penetrant PRMT5 inhibitor, in patients with advanced solid tumors, including glioblastoma multiforme (GBM), remains ongoing. As previously reported, the trial has demonstrated early signs of clinical activity and tolerability. Prelude remains on track to establish the recommended expansion dose and commence the dose expansion portion of the trial in mid-2021 in patients with central nervous system cancers including GBM, with initial clinical data expected by the end of 2021.
PRT1419

Oral Formulation: Dose Expansion and Combination Cohorts Expected to be Added to Ongoing Phase 1 Trial in the Second Half of 2021. The dose escalation portion of the Company’s first-in-human Phase 1 open-label, multicenter, dose-escalation study of oral PRT1419 in patients with relapsed/refractory hematologic malignancies, including acute myeloid leukemia and high-risk myelodysplastic syndromes, remains ongoing. PRT1419, which is the Company’s third clinical candidate, is designed to be an orally available, potent, and selective MCL1 inhibitor. The Company expects to add dose expansion and combination cohorts to the Phase 1 clinical trial in the second half of 2021.

IV Formulation: IND Application Cleared. Prelude reported the recent U.S. Food and Drug Administration (FDA) clearance of the Company’s Investigational New Drug (IND) application for an intravenous (IV) formulation of PRT1419. A Phase 1 trial of the IV formulation, which leverages the optimized physicochemical properties of PRT1419, is expected to commence in the first half of 2021 in patients with solid tumors.

Data on Preclinical Characterization to be Featured at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. Data on the preclinical characterization of PRT1419 will be featured during a poster session at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. A copy of the poster, titled "Preclinical characterization of PRT1419, a potent, selective and orally available inhibitor of MCL1" (abstract 983), will be available in the Publication section of the Prelude Therapeutics website following the conclusion of the meeting.
Discovery Programs

Advancement of Earlier-Stage Candidates Expected in 2021. Prelude remains on track to submit an IND application for PRT2527, which is designed to be a potent and selective CDK9 inhibitor, in 2021. The Company also continues to expect to initiate IND-enabling studies for PRT-SCA2, which is designed to be a SMARCA2 protein degrader, in 2021.

Preclinical Data on SMARCA2 Protein Degradation to be Featured at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. A poster presentation on Prelude’s SMARCA2 protein degradation program, titled "Potent SMARCA2 targeted degraders induce genetic synthetic lethality in SMARCA4 deleted cancer" (abstract 1139), will be presented at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. A copy of the poster will be available in the Publications section of the Prelude Therapeutics website following the conclusion of the meeting.
Corporate

Completed Successful Upsized Public Offering of $172.5 Million. In January 2021, the Company announced the closing of its upsized public offering of 2,583,334 shares of its voting common stock and 291,666 shares of its non-voting common stock, each at a public offering price of $60.00 per share, which includes the exercise in full of the underwriters’ option to purchase an additional 375,000 shares of its voting common stock. The aggregate gross proceeds from this offering were $172.5 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Prelude.
Fourth Quarter and Full Year 2020 Financial Results

Cash and Cash Equivalents: Cash and cash equivalents as of December 31, 2020 were $218.3 million.

Research and Development (R&D) Expenses: For the fourth quarter of 2020, R&D expense increased to $14.6 million from $8.8 million for the prior year period, and for the full year increased to $48.2 million compared to $24.3 million for 2019. The fourth quarter and full year increases were primarily due to increased clinical research costs for the PRT543 and PRT811 clinical trials and increased costs associated with the initiation of the clinical trial for PRT1419, which began in the third quarter of 2020. The Company also incurred an increase in chemistry, manufacturing, and other costs for those trials.

General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2020 increased to $4.9 million from $1.2 million for the prior year period, and for the full year increased to $10.6 million compared to $3.8 million for 2019. The fourth quarter and full year increases were primarily due to an increase in personnel related expense due to increased employee headcount and an increase in professional fees as the Company expanded its operations to support R&D efforts and incurred additional costs associated with operating as a public company.

Net Loss: For the fourth quarter of 2020, net loss was $19.3 million, or $0.45 per share, compared with a net loss of $10.0 million, or $5.56 per share, for the same period in 2019. Net loss for the year ended December 31, 2020 was $56.9 million, or $4.56 per share, compared with a net loss of $27.6 million, or $16.52 per share, for the year ended December 31, 2019.

Financial Guidance: The Company believes that its current cash and cash equivalents will be sufficient to fund operating expenses and capital expenditure requirements into 2023.