On March 22, 2019 bluebird bio, Inc. (Nasdaq: BLUE) reported the official opening of its first wholly owned manufacturing facility in Durham, N.C., that will produce lentiviral vector for the company’s investigational gene and cell therapies, including: bb2121 and bb21217 for the treatment of multiple myeloma and potentially LentiGlobin for the treatment of transfusion-dependent β-thalassemia (TDT) and sickle cell disease (Press release, bluebird bio, MAR 22, 2019, View Source [SID1234534554]).

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Gov. Roy Cooper, Secretary of Commerce Tony Copeland and local patient advocates will join chief bluebird Nick Leschly in a ribbon cutting ceremony at the 125,000-square-foot facility. Currently, bluebird employs approximately 50 scientists, engineers, manufacturing and operations personnel at the facility and is on track to grow to approximately 70 employees by the end of 2019.

"At bluebird bio, we view every aspect of our path to helping patients as both a privilege and a responsibility. This includes the expertise that we’ve poured into the construction and operation of our manufacturing facility, because it is a crucial step toward our mission of bringing a new generation of treatments to people living with severe genetic diseases and cancer," said Leschly. "Our teams in North Carolina and across the globe are working to deliver treatments that will make a big difference for a lot of patients and families. This is what drives our ambition to bring four gene therapies forward in the next few years."

"North Carolina is proud to bring bluebird bio’s cutting-edge work to Durham," Governor Cooper said. "bluebird is developing treatments for devastating diseases that could change the course of medicine. And, with the Triangle’s highly-skilled workforce, it will continue to be a leader in the biotech field."

bluebird bio purchased the facility in November 2017. Once completed, the company will have invested more than $80 million building a world class site equipped with multiple manufacturing suites capable of producing lentiviral vector (LVV). The facility also includes warehouse and quality control testing laboratories. The facility construction is substantially complete and equipment qualification is underway. Initially, bluebird bio expects the facility to produce clinical and commercial supply of lentiviral vector, which is a critical component of the company’s gene and cell therapies. The facility is large enough to accommodate significant future expansion, including the possibility of manufacturing commercial drug product.

The goal of gene therapy is to change or replace faulty genes with functional ones in order to prevent, treat or cure a disease. Vectors are selected parts of viruses that have been genetically modified so they can deliver new genes into cells without causing an infectious disease. Prior to gene therapy treatment, copies of functional genes are added to a vector — the delivery system — in a laboratory setting. The vector, with copies of the functional gene, is added to blood stem cells collected from the patient. The cells that now have functional copies of the gene are referred to as gene-modified cells.

In addition to the Durham facility, bluebird bio also has multi-year agreements with three manufacturing partners in the United States and Europe: Brammer Bio (Cambridge, Mass.), Novasep (Gosselies, Belgium) and MilliporeSigma, the Life Science business of Merck KGaA (Carlsbad, Calif.). Each of these partners is collaborating with bluebird bio on production of lentiviral vector across all programs. bluebird bio also partners with Lonza (Houston, Texas) and apceth Biopharma (Munich, Germany) to produce drug product for Lenti-D and LentiGlobin.

bluebird will receive an Economic Development Award from NCBiotech upon meeting job creation targets in North Carolina and will also receive life-sciences-specific employee training support through the North Carolina Community College System’s Customized Training Program.

On March 22, 2019 WuXi AppTec Co., Ltd. (stock code: 603259.SH / 02359.HK), a leading global pharmaceutical and medical device open-access capability and technology platform company with global operations, reported its audited annual results for the year ended December 31, 2018 today (Press release, WuXi AppTec, MAR 22, 2019, View Source [SID1234534573]).

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All financials disclosed in this press release are prepared based on International Financial Reporting Standards (or "IFRS").

Financial Highlights

Strong revenue growth of 23.8% year-over-year to RMB9,614 million. Applying a constant exchange rate, revenue grew 25.4% to RMB9,739 million. We continued to enhance capabilities and build capacity across all segments, while investing in the latest technologies to expand our service offering. We particularly benefited from a surging increase in business from China-based customers, and biotech customers globally.
Gross profit grew 16.6% year-over-year to RMB3,777 million. Gross profit margin was 39.3%.[1] Excluding the impact of foreign exchange loss, gross profit margin was 39.9%.
Net profit attributable to owners of the Company increased 84.2% year-over-year to RMB2,261 million, due to strong operations execution coupled with an extraordinary gain of RMB616 million from the fair value change of our investment portfolio.
Adjusted non-IFRS net profit attributable to owners of the Company grew 23.3% year-over-year to RMB1,742 million.
Diluted EPS and adjusted non-IFRS EPS increased by 70.0% and 13.9%, respectively.
Operational Highlights

We acquired 1,400+ new customers, and our active customers count reached 3,500+. – All of the top 20 global pharmaceutical companies are our customers. – We achieved 100% retention rate for our top 10 customers. – We continue to expand our biotech customer base rapidly as our integrated R&D platform enables them to efficiently and effectively discover and develop products.
For our success-based drug discovery services, in 2018 we filed 27 new-chemical-entity INDs for our customers with the NMPA (China National Medical Products Administration) and obtained 17 CTAs (clinical trial approval). As of December 31, 2018, we have cumulatively submitted 55 new-chemical-entity IND filings with NMPA for our customers and obtained 34 CTAs.
Our small molecule CDMO/CMO segment provided services to 650+ projects, including 40 in Phase III clinical trials and 16 that have been commercialized.
We became the first CMO services provider under China’s newly implemented MAH (Market Authorization Holder) policy and began commercial manufacturing services for two products, including: Ganovo, developed by Ascletis Pharma for treatment of Hepatitis C and Elunate, developed by Hutchison MediPharma for treatment of colorectal cancer.
In the highly-specialized field of cell and gene therapy, we provided CDMO services for 30 clinical stage cell and gene therapies projects; 25 in phase I and 5 in phase II/III.
Our fast growing clinical research services business helped one multinational company’s PD-1 antibody obtain market approval in China. We also helped a domestic company’s break-through drug obtain market approval for the treatment of colorectal cancer. In addition, we helped the BLA submission of the first CD20 biosimilar product in China.
In July 2018, we acquired an Austin, Texas based clinical research CRO WuXi Clinical Development, Inc. (carrying on business as ResearchPoint Global). This acquisition has allowed us to expand our global clinical trial service offering to both China based companies and US based companies for their global clinical development.
To support future growth, we are significantly expanding capacity including: chemistry laboratory expansion in our Tianjin site and new Qidong site; new cell and gene therapy GMP facilities in our Philadelphia site and Wuxi Jiangsu site; API GMP manufacturing facilities in our Changzhou site; a new medical device testing laboratory in Suzhou, and a new biology research laboratory in San Diego, California.
Management Comments

"2018 was an incredible year for the global healthcare industry and a fantastic one for our company," said Dr. Ge Li, Chairman and CEO. "The industry experienced significant progress as new innovative drugs address increasingly complex indications with remarkable results. And with the drug approval timeline greatly accelerated, the number of new drugs approved for market in the United States and in China has reached a record high. We were proud to see that 39 out of the 59 new drugs approved by the FDA came from our customers and we are committed to continually investing in our capabilities and capacity to consistently provide the highest level of service in this rapidly-changing environment."

"We are grateful to our investors who continue to believe in our vision. The capital they provide drives our growth reinforcing the confidence they have in our management team and our business model. In May and December of 2018, we successfully listed on the Shanghai Stock Exchange and the Main Board of the Hong Kong Stock Exchange, respectively."

"Our ‘Follow the Project / Follow the Molecule / Follow the Customer’ strategy continues to deliver stellar results. All along, we continued to successfully execute on our business plan achieving revenue of RMB9,614 million, representing growth of 23.8% year-over-year. Applying a constant exchange rate, our revenue grew 25.4% to RMB9,739 million. Revenue growth was broad-based across all business segments; especially our China-based laboratory services, CDMO/CMO services and clinical research and other CRO services. In addition to investments in our operations, i.e. new talent, new laboratories and facilities, our unique ecosystem that we are relentlessly building provided additional opportunities for us to effectively put our capital to use laying the foundation for future growth."

"WuXi AppTec is committed to enable innovation worldwide, catalyzing and benefiting from the continuous transformation of the healthcare ecosystem," Dr. Li continued. "Once the platform boasts enough scale and depth, the long tail effects will be truly dramatic. Looking forward, we are now entering an unprecedented golden age with the healthcare ecosystem emerging, where an ever-increasing number of participants are able to play a role at different stages of innovation. Through our integrated platform, more and more institutions, scientists, hospitals, and doctors will realize their dreams of innovation. In doing so, we will undoubtedly help bring vital medicine to patients faster."

Full-Year 2018 IFRS Results

2018 revenue increased 23.8% year-over-year to RMB9,614 million. Applying a constant exchange rate, revenue increased 25.4% year-over-year to RMB9,739 million. – Our China-based laboratory services revenue increased 24.1% year-over-year to RMB5,113 million. We fully leveraged our platform to attract more customers, especially "long-tail" customers, while expanding services to our existing customers. – We grew our CDMO/CMO services revenue 28.0% year-over-year to RMB2,699 million by diligently executing our "follow the molecule" strategy. As our customers’ projects move into late stage, we progress alongside of them by providing process validation services all the way through to commercial manufacturing services. – Our US-based laboratory services revenue increased 6.1% year-over-year to RMB1,204 million. Revenue growth was driven by our cell and gene therapies CDMO services and partially offset by a decline in medical device testing services. Cell and gene therapies CDMO services revenue grew 7.5% and 28.4% in the first half of 2018 and second half of 2018, respectively. – Our clinical research and other CRO services revenue increased 64.2% year-over-year to RMB585 million. Revenue growth was mainly driven by the substantial increase of the domestic drug clinical trial market as well as significant improvement of our services in terms of quality, scale and capabilities. – Across all segments, we benefitted from the rapid rise of pharmaceutical innovation in China experiencing 55.5% year-over-year revenue growth from China-based customers.
2018 gross profit increased 16.6% year-over-year to RMB3,777 million. Gross profit margin was 39.3%, slightly lower than 41.7% in 2017[2] primarily due to a decrease in gross profit of US-based laboratory services and RMB appreciation against USD in the first half of 2018. Applying a constant exchange rate, the gross profit margin would be 39.9%.
2018 IFRS net profit attributable to owners of the Company increased 84.2% year-over-year to RMB2,261 million. We experienced significant synergies across business segments by fully leveraging the strength of our "integrated end-to-end" R&D services platform. In addition, we experienced an extraordinary gain of RMB616 million from the fair value change of our investment portfolio.
Full-Year 2018 Non-IFRS Results

2018 non-IFRS net profit attributable to owners of the Company increased 75.5% year-over-year to RMB2,464 million. This excludes RMB46 million share-based payments, RMB22 million listing expenses for the offering of our A shares and H shares, RMB116 million foreign exchange-related losses and RMB19 million amortization of intangible assets acquired.
Full-Year 2018 Adjusted Non-IFRS Results

Excluding a further RMB750 million realized and unrealized gains from our venture investments and RMB28 million losses from our joint ventures, 2018 adjusted non-IFRS net profit attributable to owners of the Company increased 23.3% year-over-year to RMB1,742 million in 2018.
[1] If prepared under Accounting Standard for Business Enterprises of PRC, the gross profit grew 16.8% year over year to RMB 3,793 million. Gross profit margin was 39.5%.

[2] If prepared under Accounting Standard for Business Enterprises of PRC, 2018 gross profit increased 16.8% year-over-year to RMB 3,793 million. Gross profit margin was 39.5%, slightly lower than 41.8% in 2017.

On March 22, 2019 Celyad (Euronext Brussels and Paris, and Nasdaq: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, reported that the Company will report full year 2018 financial and operating results on the evening of Thursday, March 28, 2019 (Press release, Celyad, MAR 22, 2019, https://www.celyad.com/en/news/celyad-to-announce-full-year-2018-financial-results-and-host-conference-call [SID1234534556]).

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Following the press release, Celyad management will host a conference call on Friday, March 29 at 1p.m. CET / 8a.m. EDT to discuss full year 2018 results and provide an update on the Company’s recent progress and upcoming milestones.

Participants may access the conference call by dialing +44 (0) 2071 928000. The conference ID for the call is 8745558. Alternatively, participants may access the conference call by dialing the following local numbers: Belgium 024009874, France 0176700794, Netherlands 0207143545 and U.S. 16315107495.

To access the subsequent archived recording, visit the "Events & Webcasts" section of the Celyad website.

On March 22, 2019 Genmab A/S (Nasdaq Copenhagen: GEN) reported that Janssen Pharmaceutica NV (Janssen) has submitted a Type II variation application to the European Medicines Agency (EMA) (Press release, Genmab, MAR 22, 2019, View Source [SID1234534557]). The application seeks to broaden the existing marketing authorization for daratumumab (DARZALEX) to include use in combination with lenalidomide and dexamethasone (Rd) as treatment for newly diagnosed multiple myeloma patients who are not candidates for high dose chemotherapy and autologous stem cell transplant (ASCT) . The submission is based on data from the Phase III MAIA study of daratumumab in combination with Rd as treatment for patients with newly diagnosed multiple myeloma, who are not candidates for high dose chemotherapy and ASCT. Data from the study was presented at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which took place in San Diego, California in December 2018. In August 2012, Genmab granted Janssen an exclusive worldwide license to develop, manufacture and commercialize daratumumab.

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"At Genmab we’re committed to improving the lives of patients by creating and developing innovative antibody products. With this potential label expansion, we are hopeful that DARZALEX will become available to an even wider group of multiple myeloma patients in the first line setting," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

About the MAIA (MMY3008) study
The Phase III study (NCT02252172) is a randomized, open-label, multicenter study that includes 737 newly diagnosed patients with multiple myeloma who are not candidates for high dose chemotherapy and ASCT. Patients were randomized to receive either daratumumab in combination with lenalidomide (an immunomodulatory drug) and dexamethasone (a corticosteroid) or lenalidomide and dexamethasone alone. In the daratumumab treatment arm, patients received 16 milligrams per kilogram (mg/kg) weekly for first 8 weeks (Cycles 1 and 2), every other week for 16 weeks (Cycles 3 to 6) and then every 4 weeks (Cycle 7 and beyond) until progression of disease or unacceptable toxicity. Lenalidomide is administered at 25 mg orally on days 1 through 21 of each 28-day cycle, and dexamethasone was administered at 40 mg once a week for both treatment arms. Participants in both treatment arms will continue Rd until disease progression or unacceptable toxicity. The primary endpoint of the study is PFS.

About multiple myeloma
Multiple myeloma is an incurable blood cancer that starts in the bone marrow and is characterized by an excess proliferation of plasma cells.1 Multiple myeloma is the third most common blood cancer in the U.S., after leukemia and lymphoma.2 Approximately 26,000 new patients were expected to be diagnosed with multiple myeloma and approximately 13,650 people were expected to die from the disease in the U.S. in 2018.3 Globally, it was estimated that 160,000 people were diagnosed and 106,000 died from the disease in 2018.4 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed due to symptoms which can include bone problems, low blood counts, calcium elevation, kidney problems or infections.5

About DARZALEX(daratumumab)
DARZALEX (daratumumab) injection for intravenous infusion is indicated in the United States in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.6 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX is indicated in Europe in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX is approved in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for treatment of adults with relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market. For more information, visit www.DARZALEX.com.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).6,7,8,9,10

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and frontline multiple myeloma settings and in amyloidosis. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases, such as NKT-cell lymphoma, B and T-ALL. Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies.

On March 22, 2019 LegoChem Biosciences, Inc. ("LCB") (KOSDAQ:141080) reported that it has entered a research collaboration and license agreement with Takeda Pharmaceutical Company Limited ("Takeda") for the development of antibody-drug conjugates in immuno-oncology (Press release, LegoChem Biosciences, MAR 22, 2019, View Source [SID1234534538]).

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Takeda gains certain rights to LCB’s antibody-drug conjugate (ADC) technology, ConjuAll, including LCB’s proprietary linker and conjugation platform, to research, develop and commercialize targeted immuno-oncology therapeutics. Under the terms of the agreement, Takeda has rights to use the LCB technology to develop therapeutics directed to up to three undisclosed targets.

LCB will receive $7.25 million in upfront and near-term milestone payments. In addition, LCB is eligible to receive development, regulatory and commercial milestone payments of up to $404 million as well as royalties on the sales of any resulting ADC products.

"We are delighted to collaborate with Takeda given its experience and expertise in oncology research and development. We believe this partnership provides further validation of our proprietary ADC platform technology that can be used for different oncology applications including immuno-oncology therapeutics," said Yong-Zu Kim, CEO of LCB. "Our goal is to demonstrate the competitiveness of LCB in the global ADC market by applying our linker and payload platform technologies to our own and our collaborators’ pipelines."