On January 9, 2022 Emergent BioSolutions Inc. (NYSE:EBS) reported its financial guidance for 2022 and selected preliminary unaudited financial results for 2021 (Press release, Emergent BioSolutions, JAN 9, 2022, View Source [SID1234598439]).

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"This past year we celebrated wins across the business and overcame our share of challenges, which have made our organization stronger," said Robert G. Kramer, president and CEO of Emergent BioSolutions. "We remain focused on our growth goals and dedicated to our vision of protecting and enhancing the lives of 1 billion people by 2030. We are confident in our core business and our growth potential, driven by quality manufacturing, a broad R&D portfolio, diverse M&A opportunities, and most importantly, our talented workforce."

"Our 2021 financial performance reflects steady growth in our core products combined with strong CDMO services revenues stemming from our participation in the global COVID-19 response," said Richard S. Lindahl, executive vice president and CFO. "In 2022 we anticipate continued solid contributions from our Government/Medical Countermeasure and Commercial products businesses, more normalized performance from our CDMO services business, and achievement of important milestones in our R&D portfolio."

PRELIMINARY 2021 FINANCIAL RESULTS (Unaudited)

The Company is providing the following preliminary, unaudited financial results for full year 2021.

Revenue Metrics
Total revenues for 2021 are expected to be in the range of $1,770 million to $1,790 million, an increase at the midpoint of $225 million or 14% as compared to 2020. This growth primarily reflects increased sales of contract development and manufacturing (CDMO) services to pharmaceutical and biotechnology innovators and government/non-government organization (NGO) customers, and to a lesser extent higher product sales, primarily from NARCAN (naloxone HCl) Nasal Spray.

Profitability Metrics
The Company anticipates Adjusted EBITDA of $500 million to $525 million, a decrease at the midpoint of $118 million or 19% as compared to 2020. The Company anticipates Adjusted Net Income of $315 million to $335 million, a decrease at the midpoint of $99 million or 23% as compared to 2020. This decrease primarily reflects the impact of the incremental costs at the Company’s Bayview facility. (See "Reconciliation of Non-GAAP Measures" for a definition of the terms and reconciliation tables.)

Note:
The preliminary 2021 financial results are unaudited, subject to revision, and anticipated to be finalized by late February 2022. The Company is in the process of finalizing its goodwill and long-lived asset impairment assessments for 2021. Any potential impairment has not been incorporated in these preliminary 2021 financial results. The Company’s final audited financial results could differ materially from these selected preliminary results.

2022 FINANCIAL GUIDANCE

The Company is providing the following guidance of selected financial metrics for full year 2022.

* Includes revenues from the Company’s branded NARCAN Nasal Spray and revenues related to the authorized generic of NARCAN Nasal Spray, a product licensed to Sandoz AG and launched in late 2021.

Total Revenues
The 2022 guidance for total revenue indicates a re-baseline of the Company’s operational performance and primarily reflects the impact to the CDMO services business following the conclusion of the Center for Innovation in Advanced Development and Manufacturing (CIADM) task order with the Biomedical Advanced Research and Development Authority (BARDA) and to the commercial products business following the formation of a generic market for NARCAN Nasal Spray.

Adjusted EBITDA and Adjusted Net Income (1)
The 2022 guidance reflects an anticipated mix of product and services gross margin, continued investment in research and development, and scale efficiencies in selling, general & administration expenses.

2022 Product/Service Level Revenues – Select Assumptions

Anthrax vaccines revenues are expected to continue at similar levels to 2021 under the terms of the Company’s existing contract with BARDA.
ACAM2000 (Smallpox (Vaccinia) Vaccine, Live) vaccine deliveries are expected to continue under the terms of the Company’s existing contract with the U.S. Department of Health and Human Services (HHS) at unit volume levels consistent with 2021 deliveries.
Nasal naloxone product revenues reflect the formation of a generic market and comprise revenues from NARCAN (naloxone HCl) Nasal Spray and revenues related to the authorized generic of NARCAN Nasal Spray, a product licensed to Sandoz AG and launched in late 2021.
CDMO revenues include, among others, continued production of COVID-19 drug substance for Johnson & Johnson.
Other 2022 Assumptions

Gross margin is expected to be approximately 47%-51% on a GAAP basis, influenced by the mix of product and services revenues.
Pipeline progress is expected across the R&D portfolio with anticipated advancements of a number of early-stage programs, the ongoing progress of the CHIKV VLP Phase 3 clinical trial, and completion of the Biologics License Application filing for AV7909, the Company’s next generation anthrax vaccine candidate.
Capital expenditures, net of reimbursement, are expected to be approximately 10% of total revenues at the midpoint, reflecting ongoing investments in capacity and capability expansions related to the CDMO business and the Company’s R&D programs.
FOOTNOTES
(1) See "Reconciliation of Non-GAAP Measures" for a definition of terms and applicable reconciliation tables.

PRESENTATION WEBCAST
The Company will provide an update on the current business and discuss preliminary 2021 unaudited financial results, the financial guidance for 2022, and long-term goals during its presentation at the 40th Annual J.P. Morgan Healthcare Conference on January 10, 2022 at 8:15 AM Eastern time.

A live webcast of the presentation can be accessed through Emergent’s website. An on-demand replay of the webcast can also be accessed in the investors section after the presentation has concluded.

RECONCILIATION OF NON-GAAP MEASURES (unaudited)
This press release contains financial measures (Adjusted Net Income, Adjusted EBITDA (Earnings Before Interest, Taxes, Depreciation and Amortization and Adjusted EBITDA Margin)) that are considered "non-GAAP" financial measures under applicable Securities and Exchange Commission rules and regulations. These non-GAAP financial measures should be considered supplemental to and not a substitute for financial information prepared in accordance with generally accepted accounting principles. The Company’s definition of these non-GAAP measures may differ from similarly titled measures used by others. Adjusted net income adjusts for specified items that can be highly variable or difficult to predict, or reflect the non-cash impact of charges. All adjustments are tax effected utilizing the federal statutory tax rate for the US, except for changes in the fair value of contingent consideration as the vast majority is non-deductible for tax purposes. Adjusted EBITDA reflects net income excluding the impact of depreciation, amortization, interest expense and income taxes, excluding specified items that can be highly variable and the non-cash impact of certain accounting adjustments. Adjusted EBITDA Margin is defined as Adjusted EBITDA divided by total revenues. The Company views these non-GAAP financial measures as a means to facilitate management’s financial and operational decision-making, including evaluation of the Company’s historical operating results and comparison to competitors’ operating results. These non-GAAP financial measures reflect an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results and the reconciliations to the corresponding GAAP financial measure may provide a more complete understanding of factors and trends affecting the Company’s business.

The determination of the amounts that are excluded from these non-GAAP financial measures are a matter of management judgment and depend upon, among other factors, the nature of the underlying expense or income amounts. Because non-GAAP financial measures exclude the effect of items that will increase or decrease the Company’s reported results of operations, management strongly encourages investors to review the Company’s consolidated financial statements and publicly filed reports in their entirety.

This press release references changes in Revenues, Adjusted EBITDA, and Adjusted Net Income from the Company’s full year 2020 performance to the mid-point of the estimated full year 2021 performance. The Company believes these metrics are an important part of assessing performance on a year over year basis. These changes are expressed in dollars as well as percentages. A reconciliation of the calculation of these changes is included below.

Reconciliation of Net Income to Adjusted Net Income (Unaudited)

Reconciliation of the 2021 Estimated Midpoint of Revenues, Adjusted EBITDA and Adjusted Net Income and the Dollar and Percentage Changes as compared to 2020 Actual (Unaudited)

On July 20, 2022 BioRay Pharmaceutical Co., Ltd. (hereinafter referred to as "BioRay") reported that the first patient with advanced malignant tumors had been dosed in the Phase I Clinical trial of self-developed Category 1 innovative drug BR105 (Press release, Zhejiang Hisun Pharmaceutical, JUL 20, 2022, View Source;a=index&classid=43&id=1 [SID1234634616]). This is an open-label, dose-escalation, and dose-expansion trial to explore the safety, tolerability, and antitumor activity of BR105. The phase Ia part of the trial aims to evaluate the safety and tolerability of BR105 monotherapy (single and multiple doses) in subjects with advanced malignancies and to explore the maximum tolerated dose (MTD). The leading entity of the clinical trial is Beijing Cancer Hospital, and the principal investigators are Prof. Jun Zhu and Prof. Lin Shen.

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BR105 is a SIRPα-targeting humanized monoclonal antibody that was independently developed by BioRay, which can recognize the common variants of signal-regulated protein α (SIRPα), blocks the binding of SIRPα to CD47, disengage the "don’t eat me" signal and activate macrophages to promote phagocytosis of tumor cells.

SIRPα is a transmembrane protein expressed on the surface of myeloid cells such as macrophages, monocytes, dendritic cells, and granulocytes. The binding of SIRPα to CD47 activates phagocytosis-inhibitory signals in multiple cancer types, and tumor cells are able to evade phagocytosis by macrophages. Blocking CD47/SIRPα signaling potentiates phagocytosis of tumor cells by macrophages, and suppresses tumor growth. CD47/SIRPα can be a viable immune target for antitumor therapy.

Multiple tumor models have demonstrated the effectiveness of targeting CD47/SIRPα. Clinical studies suggest that blockage of CD47–SIRPα signaling pathway may generate antitumor activity in broad-spectrumof malignancies, including various hematologic and solid tumors. CD47/SIRPα-targeted therapy has shown positive efficacy results in acute myeloid leukemia, lymphoma, head, and neck squamous cell carcinoma, gastric cancer, and other tumors.

Unlike CD47, SIRPα has a limited tissue expression pattern. BR105 blocks CD47/SIRPα signaling pathway by targeting SIRPα, which is superior in safety. In addition, CD47 can interact with other proteins such as TSP-1 and SIRPγ, which are involved in more complex signaling pathways, and the corresponding targeted therapeutic regimens have more risk considerations to balance; therefore, developing SIRPα antibodies to block the CD47/SIRPα signaling pathway may be a more effective strategy for oncology drug development.

"CD47/SIRPα is considered one of the most prospective targets for tumor immunotherapy after PD-1/PD-L1," said Dr. Wei Zhu, Chief Medical Officer of BioRay, "BR105 is an important candidate of our tumor immunization pipeline. We are pleased that the first patient’s dosing of BR105 has been completed, and this marks important progress in the innovation and R&D of BioRay. We will next push forward with our research and development projects in the field of popular targets for tumor immunity and autoimmunity to benefit patients."

On January 7, 2022 ImaginAb, Inc., a market leading global biotechnology company focused on developing next generation ImmunoPET imaging agents and therapeutic radiopharmaceuticals and with a history of taking agents into clinical trials, reported an expansion of its development pipeline with six active programs focused on Radiopharmaceutical Therapies (RPT), targeting a number of cancer indications (Press release, ImaginAb, JAN 7, 2022, View Source [SID1234621841]).

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These programs are currently moving through lead selection and IND-enabling studies with a view to the most promising candidates entering clinical trials during 2023.

The RPT pipeline uses ImaginAb’s core biological technology platform which utilizes antibody fragment technologies to deliver high tumor specificity and uptake, whilst providing faster blood clearance to reduce off-target toxicities and irradiation.

This technology allows agents to be tuned "just right" to provide the optimal radiopharmaceutical characteristics, a term which ImaginAb refers to as the "Goldilocks Criteria".

These advantages bring the potential to treat more cancer patients with RPT thereby increasing the potential for radiopharmaceuticals to provide greater impact in treating a range of cancer types.

Ian Wilson, CEO stated
"RPT is gaining significant clinical and commercial interest. However, many of the agents have, to date, focused on small molecules, peptides, or intact antibodies. We believe antibody fragments based on ImaginAb IP protected platform offer greater potential in providing next generation RPT agents, by increasing delivery to tumors and reducing off target toxicity.

Our plan is to focus our R&D team on our RPT programs, expanding our pipeline, and taking candidate RPT agents through IND-enabling studies during 2022 with a view to commencing clinical studies during 2023".

To make this happen, ImaginAb has established dedicated and experienced in-house Discovery, Clinical, and Operations Teams, enabling the business to undertake lead discovery including efficacy screening, radiochemistry optimization, and multicentre clinical trials with therapeutic radiopharmaceuticals.

Wilson continued "Our developing RPT pipeline will extend our product portfolio, and add significant value to our business as we continue to move forward to achieve our vision of helping patients live better and healthier lives"

On January 7, 2022 Absci Corporation reported that inked a research pact with Merck (Press release, Biosortia Pharmaceuticals, JAN 7, 2022, View Source [SID1234607739]). Under the deal, Absci will leverage its Bionic Protein non-standard amino acid technology and its artificial intelligence-powered integrated Drug Creation Platform to create enzymes to meet Merck’s biomanufacturing applications .

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Under the terms of the agreement, Absci is eligible for up to $610 million in upfront fees and milestone payments for three targets. In addition, they are up for research funding and tiered royalties on any sales that come out of the partnership.

On January 7, 2022 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas ("NHL") and chronic lymphocytic leukemia ("CLL"), have been selected for a poster presentation at the 2022 Tandem Meetings I Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy ("ASTCT") and Center for International Blood & Marrow Transplant Research ("CIBMTR"), taking place February 2 – 6, 2022 in Salt Lake City, Utah ("2022 Tandem Meetings") (Press release, Mustang Bio, JAN 7, 2022, View Source [SID1234598444]). MB-106 is being developed in a collaboration between Mustang and Fred Hutchinson Cancer Research Center ("Fred Hutch").

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Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "The compelling clinical activity and favorable safety profile that MB-106 continues to demonstrate in the ongoing Phase 1/2 trial at Fred Hutch highlight its potential as an outpatient therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia. We look forward to the updated data that will be presented by Fred Hutch at the 2022 Tandem Meetings, in particular for patients with diffuse large B cell lymphoma and Waldenstrom’s macroglobulinemia. We also look forward to advancing our MB-106 CD20-targeted CAR T cell therapy program towards a multicenter trial under Mustang’s IND in the current quarter."

Details of the presentation are as follows:

Title: High Efficacy and Low Toxicity of MB-106, a Third Generation CD20 Targeted CAR-T for Treatment of Relapsed/Refractory B-NHL and CLL
Poster Number: 225
Dates and Times: Thursday, February 3, 6:45 pm to 8:15 pm and Saturday, February 5, 6:15 pm to 7:45 pm
Presenter: Mazyar Shadman, M.D., M.P.H., Associate Professor, Clinical Research Division, Fred Hutch, Seattle, WA; Physician at Seattle Cancer Care Alliance; Associate Professor, Division of Medical Oncology, University of Washington School of Medicine

For more information, please visit the 2022 Tandem Meetings website at View Source

Note: Scientists at Fred Hutch played a role in developing these discoveries, and Fred Hutch and certain of its scientists may benefit financially from this work in the future.

About MB-106 (CD20-targeted CAR T Cell Therapy)
CD20 is a membrane-embedded surface molecule which plays a role in the differentiation of B-cells into plasma cells. The CAR T was developed by Mustang’s research collaborator, Fred Hutch, in the laboratories of the late Oliver Press, M.D., Ph.D., and Brian Till, M.D., Associate Professor in the Clinical Research Division at Fred Hutch, and exclusively licensed to Mustang in 2017. The lentiviral vector drug substance used to transduce patients’ cells to create the MB-106 drug product produced at Fred Hutch has been optimized as a third-generation CAR derived from a fully human antibody, and MB-106 is currently in a Phase 1/2 open-label, dose-escalation trial at Fred Hutch in patients with B-NHLs and CLL. The same lentiviral vector drug substance produced at Fred Hutch will be used to transduce patients’ cells to create the MB-106 drug product produced at Mustang Bio’s Worcester, MA, cell processing facility for administration in the planned multicenter phase 1/2 clinical trial to be initiated shortly under Mustang Bio’s IND. It should be noted that Mustang Bio has introduced minor improvements to its cell processing to facilitate eventual commercial launch of the product. In addition, prior to commercial launch, Mustang Bio will replace the Fred Hutch lentiviral vector drug substance with vector produced at a commercial manufacturer. Additional information on the trial can be found at View Source using the identifier NCT03277729.