On January 7, 2019 bluebird bio, Inc. (Nasdaq: BLUE) and Inhibrx, Inc. (Inhibrx) reported that they have entered into an exclusive license agreement to research, develop and commercialize chimeric antigen receptor (CAR) T cell therapies using Inhibrx’s proprietary single domain antibody (sdAb) platform to multiple cancer targets (Press release, bluebird bio, JAN 7, 2019, View Source [SID1234532541]). The small size of sdAbs may enable the generation of more complex CAR T cell products such as those designed to combine additional functions into a single CAR molecule or recognize multiple tumor antigens simultaneously.

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"Access to the Inhibrx sdAb binder technology will allow us to combine the advancements we’ve made with our T cell therapy platform with their sdAb binder technology to generate novel cellular therapies with the potential to help patients in their fight against cancer," said Philip Gregory, D. Phil., chief scientific officer, bluebird bio. "The technology from Inhibrx adds to our growing portfolio of tools and technologies that we can combine with our internal lentiviral vector, CAR and T cell expertise to discover potential new product candidates designed to recognize tumor-specific proteins expressed by cancer cells and kill them upon engagement."

"We are pleased to have formalized our relationship with bluebird bio, allowing us to couple our proprietary sdAb platform with a leading cell therapy platform," said Brendan Eckelman, Chief Scientific Officer and Executive Vice President of Corporate Strategy of Inhibrx. "Together with bluebird bio, we have generated compelling proof of concept preclinical data on the utility of incorporating our sdAbs into bluebird bio’s constructs for CAR-T cell generation."

Under the terms of the license agreement, Inhibrx will provide bluebird bio the exclusive worldwide rights to develop, manufacture and commercialize certain cell therapy products containing sdAbs directed to various cancer targets. bluebird bio will be responsible for the clinical development and commercialization of the cancer-targeting CAR-T products. Inhibrx received a $7.0 million upfront payment and is also entitled to receive specified developmental milestone payments as well as percentage tiered royalties on future product sales.

On January 7, 2019 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), is reported that it has entered into a clinical trial collaboration agreement, a supply agreement and a service agreement with CYTLIMIC Inc. (the "Agreements") for its lead product candidate eftilagimod alpha as part of a cancer vaccine (Press release, Immutep, JAN 7, 2019, View Source [SID1234532573]).

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The agreements enable the two parties to collaborate on clinical trials to evaluate efti as part of a therapeutic cancer vaccine (a therapy containing cancer antigens to boost a patient’s own immune cells to recognize and kill cancer cells related to the antigens) containing CYTLIMIC’s innovative cancer peptide vaccine, called CYT001.

The trials will be conducted by and are under the control of CYTLIMIC who will fully fund all development costs.

Under the collaboration agreement, Immutep will receive an upfront payment of US$500,000 and is eligible to receive up to US$4.5M in milestone payments upon the achievement of milestones by CYTLIMIC.

This therapeutic cancer vaccine with efti is the third example wherein efti is being evaluated in clinical studies in a combination.

Immutep retains complete exclusivity over its patent rights specifically covering its own clinical development programs and those it is conducting in conjunction with its other collaboration partners evaluating IMP321 in combination with either chemotherapy (AIPAC trial) or PD-1 / PD-L1 immunotherapy (INSIGHT and TACTI trials).

In addition to the collaboration agreement, Immutep has entered into a supply agreement to provide efti to CYTLIMIC for the manufacture of CYT001 for use in the clinical development and commercialisation of the vaccine. The Parties have also entered into a service agreement where Immutep will provide technical support services to CYTLIMIC during the development and commercialisation of CYT001.

Commenting on the Agreements, Immutep CEO Marc Voigt said: "We are very excited to be working alongside CYTLIMIC to help evaluate efti as part of an innovative cancer vaccine that has potential as a new therapy. Efti is generating interest globally. Following the agreements with CYTLIMIC, it is now being evaluated as part of three different combination therapy types: as part of a therapeutic cancer vaccine, as a chemo-immunotherapy and in an IO combination, showing its broad therapeutic potential."

Commenting on the collaboration, CYTLIMIC President and CEO, Shun Doi, Ph.D. said: "We are delighted with the engagement with Immutep, which will strongly help realize an innovative cancer vaccine-immunotherapy. Our own studies have shown that the combination of LAG-3Ig and Poly IC synergistically boost the efficacy of peptide vaccine, and thus I believe the combination of efti in our vaccine CYT001, which is also unique as an application of artificial intelligence, is an important step to add a new solution in cancer immunotherapy world."

On January 7, 2019 Emergent BioSolutions Inc. (NYSE: EBS) reported selected preliminary unaudited 2018 financial results and its financial forecast for 2019 (Press release, Emergent BioSolutions, JAN 7, 2019, View Source;p=RssLanding&cat=news&id=2382272 [SID1234532526]).

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Daniel J. Abdun-Nabi, chief executive officer of Emergent BioSolutions, said, "Our preliminary results for 2018 reflect another year of strong financial and operational performance as we continue to execute our strategy. As we enter 2019, we anticipate revenues topping $1 billion for the first time in our corporate history driven by solid organic growth in each of our business units together with contributions from the products that we acquired in 2018. Importantly, we also anticipate significant growth in each of our profitability metrics while simultaneously expanding our portfolio of advanced stage product candidates that address serious global public health threats. We remain steadfast in our commitment to enable governments and commercial customers worldwide to address their public health threat preparedness and response needs as we further our mission of protecting and enhancing life."

PRELIMINARY 2018 FINANCIAL RESULTS (Unaudited)

The company is providing the following preliminary, unaudited financial results for full year 2018.

See "Reconciliation of Net Income to Adjusted Net Income, EBITDA and Adjusted EBITDA" for a definition of terms and a reconciliation table.
Total Revenue
For the full year 2018, the company anticipates total revenue of $779 to $784 million, the midpoint of which represents a $221 million or 39% increase from 2017. This annual increase is due primarily to the contribution of sales of ACAM2000, (Smallpox (Vaccinia) Vaccine, Live), raxibacumab and NARCAN (naloxone HCl) Nasal Spray in 2018 as well as higher CMO revenue, offset by lower BioThrax (Anthrax Vaccine Adsorbed) revenue.

Net Income (GAAP and Adjusted)
For the full year 2018, the company anticipates net income of $60 to $64 million and adjusted net income of $117 to $121 million. The midpoint of the adjusted net income range represents a $23 million or 24% increase from 2017 and reflects the impact of higher product sales and CMO services revenue as well as the positive impact of a lower estimated effective tax rate. (See "Reconciliation of Net Income to Adjusted Net Income and EBITDA" for a definition of terms and a reconciliation table.)

Note
The preliminary 2018 financial results are subject to revision and will be finalized upon completion of the company’s external audit, which is anticipated in late February 2019. Once the external audit is completed, the company may report financial results that could differ, and the differences could be material.

2019 FINANCIAL FORECAST

See "Reconciliation of Net Income to Adjusted Net Income, EBITDA and Adjusted EBITDA" for a definition of terms and a reconciliation table.
For the full year of 2019, the company’s financial forecast includes the impact of the following items:

continued deliveries of BioThrax to the Strategic National Stockpile (SNS) under the current procurement contract with the Centers for Disease Control and Prevention (CDC), (the contract and the SNS are now managed by the Office of the Assistant Secretary for Preparedness and Response (ASPR));
initial deliveries of NuThrax (anthrax vaccine adsorbed with CPG 7909 adjuvant) to the SNS following expected Emergency Use Authorization pre-approval by the U.S. Food and Drug Administration (FDA) under the company’s current development and procurement contract with the Biomedical Advanced Research and Development Authority (BARDA);
full year sales of NARCAN Nasal Spray, Vaxchora (Cholera Vaccine, Live, Oral), and Vivotif (Typhoid Vaccine Live Oral Ty21a), all of which were acquired in the fourth quarter of 2018;
deliveries of ACAM2000 to the SNS under the anticipated follow-on procurement contract with the ASPR;
deliveries of raxibacumab to the SNS under the current procurement contract with BARDA;
domestic and international sales of the other medical countermeasures that comprise Other Product sales;
continued CDMO services revenue;
increased Contract & Grant revenue due to anticipated increased work related to development projects funded by third parties; and
continued investment in discretionary development projects funded by the company targeting opportunities in medical countermeasures for emerging infectious diseases and other public health threats.
The outlook for 2019 does not include estimates for potential new corporate development or other M&A transactions.

Q1 2019 REVENUE FORECAST
For the first quarter of 2019, the company anticipates total revenues of $185 to $205 million.

PRESENTATION WEBCAST
The company will provide an update on the current business and discuss preliminary 2018 financial results, the forecast and corporate goals for 2019, and long-term goals for 2020 during its presentation at the 37th Annual J.P. Morgan Healthcare Conference on January 8, 2019 at 11:00 AM Pacific time.

A live webcast of the presentation can be accessed through Emergent’s website. Visit www.emergentbiosolutions.com and select the "Investors" section. An on-demand replay of the webcast can also be accessed in the investors section after the presentation has concluded.

RECONCILIATION OF NET INCOME TO ADJUSTED NET INCOME, EBITDA AND ADJUSTED EBITDA

This press release contains two financial measures (Adjusted Net Income and EBITDA (Earnings Before Interest, Taxes, Depreciation and Amortization), and Adjusted EBITDA) 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 resulting from purchase accounting (which are all tax effected utilizing the statutory tax rate for the US). EBITDA reflects net income excluding the impact of depreciation, amortization, interest expense and provision for income taxes. Adjusted EBITDA also excludes specified items that can be highly variable and the non-cash impact of certain purchase accounting adjustments (which are all tax effected utilizing the statutory tax rate for the US). 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.

On January 7, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported expected milestones for 2019, which highlight the company’s progress advancing its clinical development candidates: NiCord, an investigational universal bone marrow donor source in late-stage development for patients with hematologic malignancies (blood cancers), and NAM-NK, an investigational, cell-based cancer immunotherapy in development in patients with non-Hodgkin lymphoma and multiple myeloma (Press release, Gamida Cell, JAN 7, 2019, View Source [SID1234532542]).

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Gamida Cell expects to complete patient enrollment in its Phase 3 clinical study of NiCord in the second half of this year. The international, randomized, multi-center study is designed to evaluate the safety and efficacy of NiCord compared to standard umbilical cord blood for allogeneic hematopoietic stem cell (bone marrow) transplant in approximately 120 patients with no available matched donor. The company also expects to report additional data from the ongoing Phase 1 study of NAM-NK at a medical meeting during the first half of 2019.

Today Gamida Cell also announced the appointment of Thomas Klima to the newly created role of chief commercial officer. Mr. Klima brings nearly 20 years of global experience in the pharmaceutical industry with expertise in cellular therapy, hematology, oncology and transplantation. During his career, he has played key roles in building commercial organizations and leading multiple successful product launches. Additionally, the company announced that Nurit Benjamini, an experienced financial executive, has been appointed to Gamida Cell’s board of directors and will chair the board’s audit committee.

"We are intently focused on advancing NiCord, which has potential to expand and improve bone marrow transplants, giving patients a chance for a cure. We are on track to complete enrollment by the end of this year, with topline data from the Phase 3 study anticipated in first half of 2020," stated Julian Adams, Ph.D., chief executive officer at Gamida Cell. "We are also continuing to build a pipeline based on our proprietary nicotinamide, or NAM, technology and plan to report additional data from the ongoing Phase 1 study of NAM-NK in the coming months."

"We have also augmented our team with two new experienced individuals. I’m pleased to welcome Tom Klima, who will join as chief commercial officer, and Nurit Benjamini, who will serve as an independent director on the company’s board. Their experience and contributions will be invaluable as we advance toward anticipated commercialization," Dr. Adams continued.

Mr. Klima most recently served as head of global commercial planning and operations at Atara Biotherapeutics, prior to which he played a key role as senior vice president and chief commercial officer at Navidea Biopharmaceuticals Ltd. Mr. Klima also served as head of sales and commercial operations at Algeta U.S. and led the successful commercial build-out and launch of Xofigo. Before Algeta, he held various commercial leadership positions at Dendreon. Mr. Klima began his pharmaceutical career at Eli Lilly where he held several positions of increasing responsibility and participated in the global launch of Cymbalta. Mr. Klima earned a B.A. in Business Administration and Marketing from Western State College.

Ms. Benjamini has served as chief financial officer of TabTale Ltd. since 2013. Previously, she held a number of chief financial officer positions, including at Wix.com Ltd., Sigma Designs Israel Ltd. and Compugen Ltd. She has served as a director at RedHill Biopharma Ltd., BioLine Rx and Allot Communications. Ms. Benjamini holds a BA in Economics and Business and an MBA in Finance from Bar Ilan University, Israel.

Expected 2019 Milestones

Gamida Cell expects to achieve the following milestones this year:

Present additional data from the Phase 1 study of NAM-NK in first half of 2019
Report early data from the Phase 1/2 study of NiCord in patients with severe aplastic anemia data in the first half of 2019
Complete patient enrollment in the Phase 3 study of NiCord in patients with high-risk hematologic malignancies in the second half of 2019
2019 Financial Outlook

Gamida Cell ended 2018 with cash, cash equivalents and available-for-sale securities of approximately $60 million (unaudited). The company expects that such funds will support the company’s capital needs through the data readout for the Phase 3 clinical study of NiCord, which is expected in the first half of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

Gamida Cell intends to provide additional financial guidance for 2019 when it reports its fourth quarter and full-year 2018 financial results in March 2019.

About NiCord

NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 clinical study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.1 For more information on clinical trials of NiCord, please visit www.clinicaltrials.gov.

About NAM-NK

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. NAM-NK addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.2

NiCord and NAM-NK are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On January 7, 2019 NANOBIOTIX (Euronext : NANO – ISIN : FR0011341205) and The University of Texas MD Anderson Cancer Center reported a large-scale, comprehensive clinical research collaboration to evaluate innovative strategies for treating patients with head and neck, pancreatic, thoracic, lung, gastrointestinal and genitourinary cancers (Press release, Nanobiotix, JAN 7, 2019, View Source [SID1234532558]).

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The collaboration will expand clinicaldevelopment of NBTXR3, a first-in-class agent designed to physically destroy cancer cells when activated by radiotherapy and to activate the immune system for both local control and systemic disease treatment.

MD Anderson will initially conductthe launch of nine new phase I/II clinical trials-involving around 340 patients-focused on evaluating the potential clinical benefit of NBTXR3 when activated by radiotherapy, either as a monotherapy or in combination with checkpoint inhibitors. The planned trials also will look at various disease stages, including low-risk/good prognostic patients, locally advanced disease, and metastatic disease. The trials will studyNBTXR3 across different radiation modalities such as radiation, re-irradiation, and compare different levels of intensity of radiation, to evaluate whether the addition of NBTXR3 to radiotherapy will improve progression-free survival, loco-regional control, quality of life and organ preservation.

Dr. Thomas Morris, Global Head of Development, Nanobiotix, said "It is important for Nanobiotix to collaborate with academic institutionsto develop a broad spectrum of clinical trials in an expedited fashion. This is a tremendous opportunity to strengthen our scientific and clinical understanding of the potential of NBTXR3 across the wide range of cancers treated with radiotherapy, with the goal of benefitting patients by applying novel research and bringing forward an innovative therapy. The collaboration between Nanobiotix and MD Anderson expands the number of NBTXR3 clinical trials to 16, illustrating our dedication to identify more effective treatments for cancer patients.

As part of the funding scheme for this collaboration, part of the payment will be made at the start of the collaboration. Other parts will be paid during development and the balance in case of FDA registration for a total of $12M investment.

This new collaboration between Nanobiotix and MD Anderson follows up the immunotherapeutic pre-clinical research collaboration, launched in April 2018, in lung cancer to explore NBTXR3 potential in immuno-oncology with checkpoint inhibitors, as well as its potential to control metastatic disease.

About NBTXR3 NBTXR3 is a first-in-class product designed to destroy, when activated by radiotherapy:
• tumors through physical cell death
• metastasis due to immunogenic cell death leading to activation of the immune system
NBTXR3 has a high degree of biocompatibility, requires one single administration before the whole radiotherapy treatment.

NBTXR3 is a late clinical stage product which has shown the potential for clinical benefit in advanced STS phase III randomized clinical trial.

NBTXR3 is actively being evaluated in head and neck cancer with locally advanced squamous cell carcinoma of the oral cavity or 2 oropharynx in elderly and frail patients unable to receive chemotherapy. The other ongoing studies are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally advanced or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent chemotherapy, and prostate adenocarcinoma.

Nanobiotix is also running an Immuno-Oncology development program. In the U.S., the Company received the FDA’s approval to launch a clinical study of NBTXR3 activated by radiotherapy in combination with anti-PD1 antibodies in lung, and head and neck cancer patients (head and neck squamous cell carcinoma and non-small cell lung cancer).