Neuralstem Reports Year End 2018 Fiscal Results

On March 22, 2019 Neuralstem, Inc. (Nasdaq: CUR), a biopharmaceutical company focused on the development of nervous system therapies based on its neural stem cell and small molecule technologies, reported its financial results for the year ended December 31, 2018 (Press release, Neuralstem, MAR 22, 2019, View Source [SID1234534570]).

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Financial Results for the Year Ended December 31, 2018

Cash Position and Liquidity: At December 31, 2018, cash and investments was approximately $5.8 million as compared to approximately $11.7 million for the same period of 2017. The $8.9 million decrease is due to cash used in operations of approximately $7.7 million partially offset by the proceeds from our October 2017 registered direct offering of common stock and warrants.

Operating Loss: Operating loss for the year ended December 31, 2018 was $8.3 million compared to a loss of $13.3 million for the same period of 2017. The decrease in operating loss for the year was primarily due to a $4.0 million decrease in research and development expenses coupled with a $0.9 million decrease in general and administrative expenses.

Net Loss: Net loss for the year ended December 31, 2018 was $4.9 million, or $0.32 per share (basic), compared to a loss of $15.7 million, or $1.20 per share (basic), for year ended 2017. The decrease in net loss was primarily due to a $5.0 decrease in operating loss coupled with a $3.3 million non-cash gains related to the change in the fair value of our liability classified stock purchase warrants

R&D Expenses: Research and development expense for the year ended December 31, 2018 was $4.0 million as compared to $8.1 million for the same period of 2017. The decrease of $4.1 million, or 50% compared to the same period of 2017 was primarily attributable to (i) a $1.6 million decrease in costs related to our completed NS-189 Phase 2 clinical trial, (ii) a $1.0 million decrease in our personnel, facility and other expenses due to our ongoing corporate restructuring and cost reduction efforts (iii) a $1.0 million decrease in non-cash share-based compensation expense and (iv) a $0.5 million increase in reimbursements under our research grants.

G&A Expenses: General and administrative expense for the year ended December 31, 2018 was $4.6 million as compared to $5.5 million for the same period of 2017. The decrease of $1.1 million, or 20% compared to the same period of 2017 was primarily attributable to a decrease in personnel, facility and related expenses due to our ongoing corporate restructuring and cost reduction efforts.

Liquidity: The Company expects its existing cash, cash equivalents and short-term investments to fund its operations based on our current operating plans, into the third quarter of 2019.

bluebird bio Opens State-of-the-Art Gene and Cell Therapy Manufacturing Facility in Durham, North Carolina

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.

WuXi AppTec Reports Strong 2018 Annual Results

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.

Cofactor Genomics to Present on Predictive Immune Modeling at 2019 American Association for Cancer Research Annual Meeting

On March 21, 2019 Cofactor Genomics reported that Natalie LaFranzo, the company’s Director of Scientific Projects and Market Development, reported that it will present at the 2019 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) being held at the Georgia World Congress Center in Atlanta, GA (Press release, Cofactor Genomics, MAR 21, 2019, View Source [SID1234534535]). The presentation is scheduled to take place on Sunday, March 31, 2019, at in Session PO.TB06.04 – Gene Expression in the Tumor Microenvironment. The presentation features Predictive Immune Modeling and multidimensional biomarker data generated in collaboration with TriStar Technology Group, a leading provider of archived human bio specimens and related research services offering highly-characterized and well-curated donor samples with extensive clinical follow-up data.

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Executives from the company including CEO, Jarret Glasscock, will be attending the meeting to engage with current and future collaborators, clients, and investors. Visit View Source to arrange a time to meet with executives from Cofactor. The company also continues to solicit applications for their ImmunoPrism Grant Program, closing April 15. The grant program offers researchers the first opportunity to access Cofactor’s Predictive Immune Modeling platform. More information on the grant program may be found here: View Source

Unum Therapeutics to Host Fourth Quarter and Full Year 2018 Financial Results Conference Call and Webcast on March 28, 2019 at 8:00 A.M. ET

On March 21, 2019 Unum Therapeutics Inc. (Nasdaq: UMRX), a clinical-stage biopharmaceutical company focused on the development of cellular immunotherapies to treat cancer using its novel T cell technology platforms, reported that the company will host a conference call and live audio webcast on Thursday, March 28, 2019 at 8:00 a.m. ET to discuss financial results for the fourth quarter and full year of 2018 (Press release, Unum Therapeutics, MAR 21, 2019, View Sourcenews-releases/news-release-details/unum-therapeutics-host-fourth-quarter-and-full-year-2018" target="_blank" title="View Sourcenews-releases/news-release-details/unum-therapeutics-host-fourth-quarter-and-full-year-2018" rel="nofollow">View Source [SID1234534521]). Unum management will also provide an update on the Company’s recent progress and upcoming milestones.

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Participants may access the conference call by dialing 866-300-3411 (domestic) or 636-812-6658 (international) and refer to conference ID number 8169027. To join the live webcast, please visit the investor relations section of the Unum Therapeutics website at View Source at least 10 minutes before the event begins.

A webcast replay will be available at the same location on the Unum Therapeutics website beginning approximately two hours after the event and will be archived for 90 days.