On March 21, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the Stifel 2022 CNS Days virtual conference at 3:30 p.m. Eastern Time on Monday, March 28, 2022 (Press release, Neurocrine Biosciences, MAR 21, 2022, View Source [SID1234610471]). Eiry Roberts, Chief Medical Officer, and Kyle Gano, Chief Business Development and Strategy Officer, will present at the conference.

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The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the events and will be archived for approximately one month.

On March 21, 2022 Yemaachi Biotech, a cancer research and diagnostics company headquartered in Accra, Ghana with offices in Washington, DC, reported the close of a $3 million seed round to advance its mission of diversifying precision oncology globally (Press release, Yemaachi Biotech, MAR 21, 2022, https://www.businesswire.com/news/home/20220321005271/en/Yemaachi-Africa%E2%80%99s-Cancer-Research-Company-Raises-Seed-Round-To-Diversify-Genomic-Datasets-Advance-Precision-Oncology-Globally [SID1234610490]). V8 Capital led the round, with LifeLine Family Heritage Fund, Y Combinator, Tencent, LoftyInc Capital, VestedWorld, V Square Capital and Ethan Perlstein also participating.

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Founded by Yaw Bediako, David Hutchful, Joyce Ngoi, and Yaw Attua-Afari in 2020, Yemaachi is dedicated to accelerating precision oncology in Africa and beyond by expanding access to research and diagnostics. Yemaachi’s first of its kind Pan-African genomic and clinical knowledge base and research platform, combined with deep clinical partnerships across Africa, provide the foundation for innovative products and partnerships to advance new molecular diagnostics and therapeutic targets. The Company also offers clinical testing services including NGS-based screening and diagnostic testing services, optimized for local populations.

Africa has been largely excluded from both genomic and oncology research. Although the continent accounts for 17% of the world’s population, only 2% of genomic study participants are of African descent. Africa’s fast-growing, treatment-naive population, significant disease burden, and the greatest human genetic diversity of any region worldwide create a fertile landscape for harvesting groundbreaking insights and improving outcomes for patients.

"We’ve only begun to scratch the surface of genomic data and understanding. We know genetic outcomes are context dependent, including within the genome. Creating a dataset that has the greatest genomic diversity can enable rapid discoveries that have long-term implications for cancer research, drug development, and patient care, not just in Africa, but globally," said Yaw Bediako, PhD, co-founder and CEO of Yemaachi. "Combined with Yemaachi’s expertise in immunogenomics, bioinformatics, and deep learning, the Company’s expansive datasets can be a force multiplier for rapidly accelerating advancements in oncology."

"The breadth of expertise of Yemaachi’s highly talented founding team, the clinical partnerships they have already formed, and their focus on leveraging the vast untapped resource of African genetic diversity to discover the next generation of cancer diagnostics and therapeutics makes them a very exciting and valuable investment for us," said Tobi Oke, Managing Partner at V8 Capital Partners.

Yemaachi was also recently named a recipient of a $1 million grant as part of the Calestous Juma Science Leadership Fellowship awarded to Bediako by the Bill and Melinda Gates Foundation. The prestigious fellowship is designed to support scientists who are working towards developing innovations in urgent global health priorities.

The Company has already begun to break ground with novel diagnostics and partnerships. Late last year, Yemaachi launched the AMBER Study in collaboration with Lucence to better characterize and understand the genomics of breast cancer in women of African descent using liquid biopsy. In January, the Company launched its at-home Sheba HPV Test in Ghana to help identify women who are at high risk of cervical cancer, the second most common cancer in West African women.

The Company’s name combines three Ghanian languages in a portmanteau that means "A New Dawn for Health in Africa."

On March 21, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported business highlights and financial results for the fourth quarter and full year 2021 (Press release, Caribou Biosciences, MAR 21, 2022, View Source [SID1234610472]).

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"2021 was a year of tremendous accomplishment for Caribou as we completed our successful IPO, advanced our pipeline of allogeneic CAR-T and CAR-NK cell therapies, including the initiation of the ANTLER Phase 1 clinical trial for our lead program CB-010, and expanded our leadership team," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "These achievements put us in a great position to execute on our 2022 plans to present initial data from the ANTLER clinical trial for CB-010 at a medical meeting; submit an IND for CB-011, our second allogeneic CAR-T cell program; and share target selection for CB-020, our first genome-edited CAR-NK cell therapy. We believe our chRDNA genome-editing platform has superior specificity and has the potential to be applied across a broad number of therapeutic applications, in oncology and beyond."

Recent Business Highlights

Pipeline

•Caribou continues to enroll patients in ANTLER, a Phase 1 clinical trial of CB-010 in adults with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). CB-010 is an allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to boost the persistence of antitumor activity. More information can be found at www.clinicaltrials.gov (NCT04637763).
•Caribou is conducting IND-enabling studies to support a planned IND application submission in 2022 for CB-011 in patients with relapsed or refractory multiple myeloma (r/r MM). CB-011 is an allogeneic anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA technology to insert a BCMA-specific CAR into the TRAC gene and armor the cells with an immune cloaking strategy that includes a knockout of the endogenous B2M gene and site-specific insertion of a B2M–HLA-E fusion transgene into the B2M gene.

Expanded leadership team

•In January 2022, Caribou appointed Syed Rizvi, M.D., as chief medical officer. Dr. Rizvi has more than two decades of experience in all stages of drug development, from clinical strategy and execution through regulatory submissions to support approval and commercialization of several cancer treatments, including three approved autologous CAR-T cell therapies ABECMA, BREYANZI, and CARVYKTITM. Prior to joining Caribou, Dr. Rizvi served as chief medical officer of Chimeric Therapeutics and worked for Legend Biotech, Celgene Corporation (now Bristol Myers Squibb), Novartis, Merck, and Genta, Inc. Since the beginning of 2021, Caribou has welcomed three new members of the executive leadership team. In addition to Dr. Rizvi, Ruhi Khan joined as chief business officer, and Jason O’Byrne joined as chief financial officer.
•In January 2022, Zili An, M.D., joined Caribou as Vice President of Pharmacology. He brings over 20 years of drug development experience in multiple cancer therapeutic modalities, including CAR-T cell therapies.
•During 2021, Caribou expanded its board of directors, which currently includes Andrew Guggenhime (board chair), Scott Braunstein, M.D., Rachel Haurwitz, Ph.D., Dara Richardson-Heron, M.D., Natalie Sacks, M.D., Nancy Whiting, Pharm.D., and Ran Zheng. Caribou’s directors bring significant and broad expertise in strategy, drug development, operations, and patient need.
•In 2021, two new members joined the Caribou’s scientific advisory board, Katy Rezvani, M.D., Ph.D., and Christopher Sturgeon, Ph.D., both of whom bring significant expertise in the development and role of natural killer (NK) cells in mediating immunity against hematologic and solid tumors.

Anticipated Milestones for 2022 and Beyond

•CB-010: Caribou expects to present initial data from its ongoing ANTLER Phase 1 trial for CB-010, an anti-CD19 CAR-T cell therapy, in adults with r/r B-NHL at a medical meeting in 2022.
•CB-011: Caribou expects to submit an IND application for CB-011, an anti-BCMA CAR-T cell therapy, in r/r MM in 2022.
•CB-020: Caribou expects to announce target selection for CB-020, an iPSC-derived CAR-NK cell therapy in 2022. Additionally, Caribou expects to disclose multiple armoring strategies under development for its CAR-NK platform in 2022.
•CB-012: Caribou expects to submit an IND application for CB-012, an anti-CD371 CAR-T cell therapy for r/r AML, in 2023.

Upcoming Meetings

•American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting – April 10, 2022, presentation of preclinical data from Caribou’s CB-011 program
•Cell & Gene Meeting on the Med – April 20-22, 2022, corporate overview
•Allogeneic Cell Therapies Summit – May 9-12, 2022, Caribou scientists will provide an overview of the company’s T cell programs

Fourth Quarter and Full Year 2021 Financial Results

Cash, cash equivalents, and marketable securities: Caribou had $413.5 million in cash, cash equivalents, and marketable securities as of December 31, 2021, which included $321.0 million in aggregate net proceeds from the company’s IPO completed in July and August 2021. Additional funding during 2021 came from the Series C financing completed in March 2021 and an upfront payment from Caribou’s collaboration and license agreement with AbbVie in February 2021.

Licensing and collaboration revenue: Revenue from Caribou’s licensing and collaboration agreements was $2.6 million for the three months ended December 31, 2021 and $9.6 million for the full year 2021, compared to $1.0 million and $12.4 million, respectively, for the same periods in 2020. The decrease for the year ended December 31, 2021, was primarily due to revenue recognized in 2020 pursuant to an exclusive license agreement between Caribou and a private company, partially offset by an increase in revenue recognized in 2021 pursuant to the AbbVie agreement.

R&D expenses: Research and development expenses were $15.1 million for the three months ended December 31, 2021, and $52.3 million for the full year 2021, compared to $12.0 million and $34.4 million, respectively, for the same periods in 2020. The increase for the year ended December 31, 2021, was primarily due to costs associated with clinical trial and preclinical study activities, personnel-related expenses attributable to increased headcount, and facilities expenses, partially offset by a decrease in expenses related to licenses and sublicensing revenues.

G&A expenses: General and administrative expenses were $7.9 million for the three months ended December 31, 2021, and $24.3 million for the full year 2021, compared to $4.2 million and $14.1 million, respectively, for the same periods in 2020. The increase for the year ended December 31, 2021, was primarily due to personnel-related expenses attributable to increased headcount, legal and accounting services associated with operating as a public company, and facilities and other expenses.

Other income (expense): The company recorded other income of $2.2 million for the three months ended December 31, 2021, and $0.4 million for the full year 2021.

Net loss: For the three months and year ended December 31, 2021, net loss was $18.5 million and $66.9 million, respectively, compared to $14.7 million and $34.3 million, respectively, for the same periods in 2020.

About Caribou’s Novel Next-Generation CRISPR Platform

CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Type II and Type V CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems occasionally edit unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed chRDNAs (pronounced "chardonnays"), RNA-DNA hybrid guides that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA technology to carry out high efficiency multiple edits, including multiplex gene insertions, to develop CRISPR-edited therapies.

On March 21, 2022 Chimeron Bio, an RNA company developing self-amplifying RNA (saRNA) vaccines and therapeutics designed on its proprietary ChaESARTM RNA delivery platform, reported it has signed a Non-Clinical Evaluation Agreement (NCEA) with the National Institute of Allergy and Infectious Diseases (NIAID), a division of the National Institutes of Health (NIH), to evaluate the potential of its vaccine for COVID-19 (Press release, Chimeron Bio, MAR 21, 2022, View Source [SID1234610492]). The Company’s lead candidate, CB-106, delivers the Spike gene as a self-amplifying mRNA. Preliminary data on this vaccine obtained in collaboration with George Mason University, demonstrated positive results in pre-clinical models with nanogram quantities of RNA, a significant dose advantage over other RNA based technologies. In addition to CB-106, other ChaESAR candidates under testing by the Company include particles co-delivering multi-variant antigens for viral and non-viral vaccines for broad spectrum protection.

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Under the current agreement, Chimeron Bio will utilize the pre-clinical services program offered by the NIAID. Chimeron Bio will provide the vaccine candidate to test in the hamster model of COVID-19 which has previously been used for the assessment of other COVID-19 vaccines. "A successful outcome from this study will further validate ChaESAR technology in pre-clinical models and boost the design of a next generation single shot RNA vaccine that is durable, low dose, low cost, scalable and can render broad-spectrum protection against multiple variants or infectious agents," said Thimmaiah Chendrimada, Co-founder and CSO of Chimeron Bio.

"We are grateful to receive NIAID’s support and work with NIAID funded world-class institutions. The COVID-19 program is Chimeron’s first foray into infectious diseases, and we expect the data from the current study to offer critical validation to advance CB-106 into non-human primates and demonstrate the applicability of ChaESAR technology for vaccines at large," said Jolly Mazumdar, Co-founder and CEO of Chimeron Bio.

On March 20, 2022 GenScript Biotech, the world’s leading biotech company, reported its annual results as of December 31, 2021 (Press release, GenScript, MAR 20, 2022, View Source [SID1234610393]).

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"2021 is a pivotal year for GenScript. Our continuous investments in innovation and optimization of current business portfolio over the past few years lead to encouraging results.", said Dr. Patrick Liu, Rotating CEO of GenScript. "In the life science segment, we are pushing forward our business through automation and industrial-scale production. In the emerging gene and cell therapy CDMO segment, GenScript ProBio achieved explosive revenue growth thanks to strategic investment, advanced platforms and high quality standards. Bestzyme reached breakeven, which is made possible by business optimization and product innovation. With continued investment in synthetic biology, Bestzyme will further unfold its potential in the future. In early 2022, our subsidiary Legend Biotech, an innovative cell therapy company, together with our partner J&J, successfully commercialized CarvyktiTM, which is a monumental event for the cell therapy industry and demonstrates innovation capabilities of China’s biopharmaceutical industry. Looking ahead, GenScript Group will continue investment in innovation capabilities to address challenges in the fields of gene and cell therapy and synthetic biology. We will stay committed to our mission to "make people and nature healthier through biotechnology" by accelerating innovation in the biotech industry, and continuously create value for our shareholders."

Results Analysis of the Four Business Segments

Life-science services and products

During the Reporting Period, revenue from life-science services and products was approximately US$315.8 million, representing an increase of 26.4% as compared with approximately US$249.8 million for the year ended December 31, 2020. During the Reporting Period, the gross profit was approximately US$183.3 million, representing an increase of 10.8% as compared with approximately US$165.4 million for the year ended December 31, 2020. During the Reporting Period, the operating profit of life-science services and products was approximately US$91.6 million, representing an increase of 6.5% from approximately US$86.0 million for the same period in 2020.

The growth of revenue and gross profit was mainly attributable to the (i) expanded capacity and productivity, (ii) successful commercial operation that focused on the novel products such as oligo synthesis and cPass services and kit, (iii) successful development of key accounts, and (iv) the improvement of online commercial platform and tools to attract new customers. The decrease in gross profit margin was primarily attributable to the (i) significant decrease of exchange rate of USD against RMB as compared to 2020 which caused an increase of converted cost as the majority of production cost occurred in Mainland China, (ii) increased freight and duty costs, and (iii) change of product portfolio strategy. The increase in operating profit was primarily attributable to the improved capacity utilization and operational efficiency, and was partially offset by increased investment in research and development.

Biologics development services

During the Reporting Period, revenue from biologics development services was approximately US$81.4 million, representing an increase of 101.5% as compared with approximately US$40.4 million for the year ended December 31, 2020. During the Reporting Period, the gross profit was approximately US$25.6 million, representing an increase of 158.6% as compared with approximately US$9.9 million for the year ended December 31, 2020. Total backlog for biologics development services increased by 108.4% from US$94.7 million as at December 31, 2020 to US$197.4 million as at December 31, 2021. The gross profit margin increased from 24.5% for the same period in 2020 to 31.4% this year. During the Reporting Period, the operating loss of biologics development services was approximately US$4.5 million, whilst the operating loss was approximately US$7.6 million for the same period in 2020.

The increase in revenue was primarily attributable to the (i) accumulated biologics development track records and expanded global customer base, (ii) expanded capacity and productivity of pre-clinical and clinical development, (iii) shorter delivery time for antibody and protein drug development, and (iv) significant increase in plasmid revenue from the boosting GCT market, including mRNA related applications. The increase in gross profit was primarily attributable to the (i) increased revenue, (ii) production cost reduction and quality improvement, and (iii) improved capacity utilization. The operating loss was primarily attributable to the (i) investment in selling and distribution, and (ii) investment in research and development activities.

Industrial synthetic biology products

During the Reporting Period, revenue from industrial synthetic biology products was approximately US$38.6 million, representing an increase of 33.6% as compared with approximately US$28.9 million for the year ended December 31, 2020. During the Reporting Period, the gross profit was approximately US$11.3 million, representing an increase of 31.4% as compared with US$8.6 million for the year ended December 31, 2020. During the Reporting Period, the industrial synthetic biology products segment has achieved operating break-even this Year, whilst the operating loss was approximately US$3.0 million for the same period in 2020.

The increase in revenue and gross profit was primarily attributable to the (i) launch of innovative products, (ii) increased penetration into big industrial customers, and (iii) business development in overseas markets. The gross profit margin remained stable during the Reporting Period.

Cell therapy

During the Reporting Period, revenue from cell therapy was approximately US$89.8 million, representing an increase of 18.6% as compared with approximately US$75.7 million for the year ended December 31, 2020. During the Reporting Period, gross profit was approximately US$89.8 million, representing an increase of 18.6% as compared with approximately US$75.7 million for the year ended December 31, 2020. During the Reporting Period, the operating loss of cell therapy was approximately US$373.9 million, whilst the operating loss was approximately US$233.4 million for the same period in 2020.

The increase in both revenue and gross profit was primarily attributable to additional milestones achieved in 2020 and 2021, and thus further recognition of contract revenue from the collaboration with Janssen on developing cilta-cel. The operating loss was primarily attributable to the (i) investment in clinical trials, higher patients enrollment and more pipelines, (ii) cost for commercial preparation activities for the launch of cilta-cel, and (iii) expansion of administrative functions.

Revenue

In 2021, the Group recorded revenue of approximately US$511.1 million, representing an increase of 30.8% from approximately US$390.8 million in 2020. This was primarily attributable to (i) the continued increase of non-cell therapy products and services from major strategic customers and new competitive services and products, and (ii) the increase of contract revenue derived from Legend’s collaboration with Janssen with new milestones achieved.

Gross Profit

In 2021, the Group’s gross profit increased 18.6% to approximately US$303.5 million from approximately US$255.9 million in 2020. The increase in gross profit was primarily attributable to the (i) rapid growth of revenue, and (ii) operational efficiency improvement. The increase in gross profit was partially offset by unfavorable exchange rate fluctuation and increased shipping costs.

Selling and distribution expenses

The selling and distribution expenses increased by 56.6% to approximately US$168.0 million in 2021 from approximately US$107.3 million in 2020. This was mainly attributable to the (i) recruiting of more experienced personnel and improved incentive packages to enhance the business development capability, and (ii) increased marketing and advertising expenses, primarily attributable to the global expansion of our business, including Legend’s collaboration with Janssen.

Administrative expenses

The administrative expenses increased by 48.9% to approximately US$134.5 million in 2021 from approximately US$90.3 million in 2020. This was mainly caused by (i) reinforcing key administrative functions such as information technology, supply chain and legal to support the Group’s overall business expansion and ensure compliance with certain updated requirements, (ii) one-time consultation expenses and other costs related to the Investigation, and (iii) the CARVYKTI application and the Follow-on Public Offering of Legend.

Research and development expenses

The research and development expenses increased by 36.1% to approximately US$358.4 million in 2021 from approximately US$263.4 million in 2020. This was mainly due to the (i) increase in clinical trial expenses and preclinical study costs in the cell therapy segment, (ii) investment in new research and development projects to strengthen our competitiveness in the GCT market and related supply chain, (iii) investment in development projects that improved our production efficiency, and (iv) increase in compensation package including equity-settled share-based compensation expense for research and development personnel.