On February 28, 2020 Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, reported fourth quarter and full year 2019 financial results and recent business highlights (Press release, Sangamo Therapeutics, FEB 28, 2020, View Source [SID1234554987]).

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"This quarter marked an important milestone for Sangamo, as we transitioned to a Phase III company following the transfer of the IND for SB-525 hemophilia A gene therapy to our partner Pfizer, who plan to commence the registrational study this year. This is a significant step in our mission to bring our genomic medicines to patients," said Sandy Macrae, CEO of Sangamo. "Additionally this year, we look forward to progressing our wholly owned assets, ST-920 gene therapy for Fabry disease and TX200 CAR-Treg cell therapy, in the clinic, and will work closely with our collaborator, Kite, as they advance KITE-037, an anti-CD19 allogeneic CAR-T therapy into a Phase 1/2 clinical trial. We will also continue to advance new IND targets in highly prevalent diseases, as exemplified by the newly announced Biogen collaboration, and will continue to look for additional synergistic partnership opportunities to advance our mission to bring innovative genomic medicines to patients and to create value for our shareholders."

Recent Highlights

Yesterday, announced global collaboration agreement with Biogen to develop and commercialize gene regulation therapies for Alzheimer’s, Parkinson’s, neuromuscular and other neurodegenerative diseases. Under the terms of the collaboration, Biogen has exclusive global rights to ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, and a third undisclosed neuromuscular disease target. In addition, Biogen has exclusive rights to nominate up to nine additional undisclosed targets over a target selection period of five years. Closing of the transaction is contingent on completion of review under antitrust laws, including the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in the United States (HSR).

Completed the transfer of the investigational new drug application (IND) for SB-525 gene therapy in hemophilia A to development partner Pfizer, triggering a $25 million milestone payment. Pfizer is currently enrolling patients in a 6-month Phase 3 lead-in study, which serves as the foundation of the Phase 3 registrational study.

Presented updated follow-up of the Phase 1/2 Alta Study assessing SB-525 in adult patients with severe hemophilia A in partnership with Pfizer at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2019. The data showed that SB-525 was generally well tolerated and demonstrated sustained increased Factor VIII levels following treatment with SB-525 through to 44 weeks, the extent of follow-up for the longest treated patient in the 3e13 vg/kg dose cohort.

Received Orphan Drug Designation from the European Medicines Agency for ST-920, an investigational gene therapy candidate for Fabry disease. Six US sites are currently active and screening subjects for the Phase 1/2 STAAR study evaluating ST-920, which Sangamo is enrolling in 2020.

Presented preliminary data from the Phase 1/2 THALES Study assessing ST-400, investigational ex vivo gene-edited cell therapy in patients with transfusion-dependent beta thalassemia in partnership with Sanofi at ASH (Free ASH Whitepaper).

Achieved $7.5 million milestone from Sanofi for the first patient dosed in their Phase 1/2 PRECIZN-1 trial evaluating BIVV003, investigational ex vivo gene-edited cell therapy for the treatment of sickle cell disease.

Received clinical trial application authorization in the United Kingdom for the Phase 1/2 STEADFAST clinical study evaluating the CAR-Treg cell therapy TX200 for kidney transplantation.

Hosted R&D Day in New York where Sangamo executives and scientists provided updates across the Company’s clinical and preclinical pipeline, as well as an overview of manufacturing capabilities to support clinical and commercial supply.

Established new Scientific Advisory Board comprising industry and academic international thought leaders who will advise Sangamo on its current and future clinical programs and research and development strategy.

Fourth Quarter 2019 Financial Results

For the fourth quarter ended December 31, 2019, Sangamo reported consolidated net income attributable to Sangamo of $4.6 million, or $0.04 per share, compared to a net loss attributable to Sangamo of $18.7 million, or $0.18 per share, for the same period in 2018.

Revenues for the fourth quarter ended December 31, 2019 were $54.9 million, compared to $26.8 million for the same period in 2018. The increase in revenue was primarily attributable to a $25.0 million milestone achieved for the completion of the IND transfer for SB-525 to Pfizer for hemophilia A, and a $7.5 million milestone achieved for dosing the first subject in the BIVV003 sickle cell disease Phase 1/2 clinical trial partnered with Sanofi.

Total operating expenses were $53.4 million for the fourth quarter ended December 31, 2019, compared to $47.6 million for the same period in 2018.

Research and development expenses were $38.3 million for the fourth quarter of 2019, compared to $33.3 million for the same period in 2018. General and administrative expenses were $15.1 million for the fourth quarter of 2019, compared to $14.4 million for the same period in 2018.

Full Year 2019 Results

For the year ended December 31, 2019, the consolidated net loss attributable to Sangamo was $95.2 million, or $0.85 per share, compared to a net loss attributable to Sangamo of $68.3 million, or $0.70 per share, for the year ended December 31, 2018.

Revenues were $102.4 million in 2019, compared to $84.5 million in 2018. The increase in revenues was primarily attributable to milestones achieved with Sanofi and Pfizer as well as higher revenues related to our collaboration agreement with Kite, a Gilead Company.

On a GAAP basis, total operating expenses were $207.6 million in 2019, compared to $161.6 million in 2018. Stock-based compensation expense included in total operating expenses in 2019 was $19.3 million, compared to $14.7 million in 2018.

Non-GAAP total operating expenses excluding the above stock-based compensation expense, were $188.3 million and $146.9 million in 2019 and 2018, respectively.

The increase in total operating expenses was primarily related to the Company’s overall headcount growth and facilities expansion to support the advancement of Sangamo’s therapeutic pipeline.

Research and development expenses were $145.9 million in 2019, compared to $114.9 million in 2018. General and administrative expenses were $61.7 million in 2019, compared to $46.7 million in 2018.

As of December 31, 2019, the Company had cash, cash equivalents, marketable securities and interest receivable of $385.0 million.

Financial Guidance for 2020

On a GAAP basis, Sangamo expects total operating expenses to be in the range of $270 to $285 million in 2020, which includes non-cash stock-based compensation expense of approximately $25 million.

Non-GAAP total operating expenses, excluding expected non-cash stock-based compensation expense of approximately $26 million, are expected to be in the range of $245 to $260 million.

Conference Call

Sangamo will host a conference call today, February 28, 2020, at 8:00 a.m. Eastern Time, which will be open to the public. The call will also be webcast live and can be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

The conference call dial-in numbers are (877) 377-7553 for domestic callers and (678) 894-3968 for international callers. The conference ID number for the call is 4609858. Participants may access the live webcast via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. A conference call replay will be available for one week following the conference call. The conference call replay numbers for domestic and international callers are (855) 859-2056 and (404) 537-3406, respectively. The conference ID number for the replay is 4609858.

On February 28, 2020 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), currently developing pelareorep, an intravenously delivered immuno-oncolytic virus, reported that it will host a conference call for Analysts and Institutional Investors at 5:00 p.m. ET on Thursday, March 5, 2020 following release of its fourth quarter and year end 2019 financial results (Press release, Oncolytics Biotech, FEB 28, 2020, https://ir.oncolyticsbiotech.com/news/detail/495/oncolytics-biotech-to-host-conference-call-to-discuss-fourth-quarter-and-year-end-2019-financial-results-and-operational-highlights [SID1234554986]).

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The live call may be accessed by dialing +1-888-231-8191 for callers in North America. Overseas callers should contact investor relations for the toll-free dial information for their country. A replay of this call will be available approximately two hours after the call is ended at +1-855-859-2056, using the replay code 3119607 and will be available for one week.

A live webcast of the call will be accessible on the Investor Relations page of Oncolytics’ website at www.oncolyticsbiotech.com and will be archived for three months.

On February 28, 2020 Verastem, Inc. (NASDAQ: VSTM) (the "Company"), reported that it has agreed to sell approximately 46.5 million shares of its common stock to certain institutional investors, including RA Capital Management, Vivo Capital, Venrock Healthcare Capital Partners, Farallon Capital Management, Acuta Capital, EcoR1 Capital LLC, Avidity Partners and Logos Capital, in a private placement (Press release, Verastem, FEB 28, 2020, View Source [SID1234554985]). The Company anticipates aggregate gross proceeds from the offering will be approximately $100 million, before deducting fees to the placement agents and other estimated offering expenses payable by the Company, based on the offering price of $2.15 per share, a 12.6% premium to the February 27, 2020 closing price. The closing is anticipated to occur on March 3, 2020 subject to customary closing conditions. The Company intends to use the net proceeds from the offering to advance the defactinib and CH5126766 (VS-6766) programs, as well as for general corporate purposes.

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Jefferies acted as sole lead placement agent for the offering and H.C. Wainwright & Co. acted as a placement agent.

The securities are being sold in a private placement and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements. The Company has agreed to file a resale registration statement with the Securities and Exchange Commission (the "SEC") as soon as practicable, and in all events within 30 days of the closing of the offering, for purposes of registering the resale of the shares of common stock issued or issuable in connection with the offering.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be by means of a prospectus.

On February 28, 2020 NantHealth, Inc. (NASDAQ-GS: NH), a next-generation, evidence-based, personalized healthcare company, reported financial results for its fourth quarter and full year ended December 31, 2019 (Press release, NantHealth, FEB 28, 2020, View Source [SID1234554984]).

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"For both the 2019 fourth quarter and full year, we continued to generate strong positive sales momentum, largely due to the ongoing growth of our SaaS business," said Ron Louks, Chief Operating Officer, NantHealth. "The results speak to the dedicated efforts of the entire NantHealth team, who have rallied around our initiatives to grow our business and drive down expenses.

"In recent months, we have streamlined our operations to accelerate growth for Eviti and NaviNet SaaS solutions. In June 2019, we completed the divestiture of our home healthcare services business, and a few weeks ago, we finalized the sale of the assets related to our Connected Care business for $47.25 million. These transactions have improved our capital position and financial flexibility, and allow us to explore growth opportunities."

Software and Services Q4 Highlights:

Clinical Decision Support (Eviti):
Presented new breast cancer research findings at the San Antonio Breast Cancer Symposium (SABCS). The study results indicate that the simultaneous presentation and publication of oncology data is an effective method for relaying practice-changing clinical data, enabling oncologists to quickly adjust treatment patterns and regimen selections
Deployed significant workflow enhancements to the Eviti platform including:
Multiple Payer Access: a significant time-saving enhancement that simplifies workflow, by allowing a user with multiple payers to toggle quickly between payer dashboards
Configuration for Appeals: allows payers to self-configure the appeals function within their account
Patient Match and Attach: automatically associates provider-submitted medical records to the appropriate treatment plan, eliminating manual processes and helping speed up treatment plan reviews
Signed an agreement with Wexford Health Sources, one of the nation’s largest correctional healthcare companies, as previously announced. Implementation was completed in early Q1 2020
In January 2020, announced two expanded Eviti Connect programs:
A leading U.S. health insurance company extended a successful two-state pilot program to 13 states after seeing a significant improvement in the use of evidence-based medicine for member oncology care
One of the largest non-profit rural health plans in the country signed a three-year renewal agreement to continue providing high-quality, high-value care for members
Payer Engagement (NaviNet):
Achieved 25% increase in NaviNet’s direct to provider Q4 2019 revenues from Q4 2018
In January 2020, signed an agreement with The Health Plan, where NaviNet Open will help decrease administrative costs and improve provider network communication and collaboration for the plan in West Virginia and Ohio
Announced enhancements to the NaviNet Open platform:
Claim Appeal: a new application that allows payers to offer an electronic channel for appeals submission, enabling stronger collaboration with the payer for claim resolution, streamlining what is often a manual analog process for both the provider and payer
NaviNet Attachments, now available within our Claim Investigation workflow, provides our users the ability to submit their required documentation easily through our portal to our payer partners, eliminating the need to send supporting documentation through manual processes outside of the portal and saving valuable time when reviewing and resolving inquiries
New Open Authorization enhancements have been introduced that enable payers and providers to manage complex prior authorization requests through electronic submissions, reducing manual workflows based on fax or phone submissions
Connected Care (VCX, DCX):
As noted above, the company completed the sale of its Connected Care assets to Masimo, a global leader in noninvasive monitoring technologies, on February 3, 2020 for $47.25 million. Assets in the sale included the Company’s DCX device connectivity software product (formerly known as DeviceConX), VCX patient vitals software (formerly known as VitalsConX), HBox connectivity hub and Shuttle interface cable. As part of the transaction, the NantHealth employees associated with the Connected Care business joined Masimo
Precision Medicine – Highlights:

Announced FDA 510(k) clearance for Omics Core℠, the nation’s first tumor-normal mutation profiling of overall tumor mutational burden (TMB) from whole exome sequencing in solid tumors. TMB is an emerging biomarker predicting response to checkpoint therapy and identifies tumors that may benefit from immunotherapy
In January 2020, presented GPS Cancer platform data revealing increased opportunities for HER2 directed therapy in colorectal cancer patients at the 2020 Gastrointestinal Cancer Symposium sponsored by the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). Data showed that up to 40% more patients may be eligible for HER2 directed therapies, which have implications for drug development and clinical trials
Artificial Intelligence – Highlights

In January 2020, NantHealth and NantOmics presented an initial report on a novel artificial intelligence (AI) platform for aiding pathologists in image-based lung cancer subtyping at the Society for Imaging Science and Technology’s International Symposium on Electronic Imaging 2020. This novel machine vision software platform accurately subtypes lung cancer pathology and achieves high concordance with analysis performed by trained medical pathologists.
In February 2020, NantHealth and NantOmics announced the publication of a peer-reviewed study in Breast Cancer Research, a Springer Nature journal, on a novel AI technique in breast cancer. The study reports on a novel deep-learning system of digital pathology images and omics data used together to more precisely identify mechanisms of therapy resistance.
Business and Financial Highlights

For the 2019 fourth quarter, total net revenue was $24.2 million, compared with $22.9 million in the 2018 fourth quarter, with the increase driven primarily by new customers in SaaS business and the variability in our DCX product line. On June 7, 2019, the Company completed the divestiture of its home healthcare services business. Accordingly, financial results for the 2019 fourth quarter do not include the results of operations from this business, which in the recent past averaged approximately $1.5 million of revenue per quarter.
Gross profit increased to $15.2 million, or 63% of total net revenue, compared with $11.5 million, or 50% of total net revenue, for the prior year period. The increase was primarily driven by continued growth of the Company’s higher margin SaaS business and overall cost management.
Selling, general and administrative (SG&A) expenses declined to $13.9 million from $14.6 million in 2018 fourth quarter, mainly driven by ongoing cost management efforts and efficiencies in overall processes. Research and development (R&D) expenses decreased to $4.8 million from $5.0 million.
Net loss from continuing operations, net of tax, was $11.7 million, or $0.11 per share, compared with $49.2 million, or $0.45 per share, for the 2018 fourth quarter.
On a non-GAAP basis, net loss from continuing operations was $4.9 million, or $0.04 per share, down from $9.0 million, or $0.08 per share, for the fourth quarter of last year. The improvement reflects the company’s ongoing efforts to manage costs, growth of its SaaS business and better overall financial performance.
For the 2019 full year, total net revenue increased to $96.0 million from $89.5 million for the 2018 full year, due to strong growth in the SaaS and DCX product lines, partially offset by the divestiture of the home healthcare services business and declines in GPS sales.
Gross profit increased to $57.5 million, or 60% of total net revenue, from $45.2 million, or 51% of total net revenue, for the prior year.
SG&A expense declined to $61.0 million from $70.8 million in 2018, driven by continued efforts to reduce costs and maximize existing resources. R&D expense declined to $19.1 million from $20.9 million in 2018.
For the full year 2019, Net loss from continuing operations, net of tax, was $62.6 million, or $0.57 per share, compared with $190.4 million, or $1.74 per share, for the 2018 full year.
For the full year 2019, on a non-GAAP basis, net loss from continuing operations was $27.4 million, or $0.25 per share, down from $44.5 million, or $0.41 per share, for 2018.
At December 31, 2019, cash and cash equivalents totaled $5.2 million.
Conference Call Information and Forward-Looking Statements

Later today, the company will host a conference call at 1:30 p.m. PT (4:30 p.m. ET) to review its results of operations for the fourth quarter and full year ended December 31, 2019. The conference call will be available to interested parties by dialing 844-309-3709 from the U.S. or Canada, or 281-962-4864 from international locations, passcode 9998343. The call will be broadcast via the Internet at www.nanthealth.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary audio software. A playback of the call will be archived and accessible on the same website for at least three months.

Discussion during the conference call may include forward-looking statements regarding topics such as the company’s financial status and performance, regulatory and operational developments, and other comments the company may make about its future plans or prospects in response to questions from participants on the conference call.

On February 28, 2020 Kineta, Inc., a clinical stage biotechnology company focused on the development of novel immunotherapies in oncology, neuroscience and biodefense reported that Kineta’s management team has been invited to participate at the Opal Group’s Family Office Winter Forum in New York, New York on March 2, 2020 (Press release, Kineta, FEB 28, 2020, View Source;utm_medium=rss&utm_campaign=kineta-invited-to-participate-at-the-opal-groups-winter-forum [SID1234554983]). Craig W. Philips, Kineta President, will participate in a panel discussion regarding the Impact of Developing Disruptive Technologies. His talk will center on the development of innovative new drug treatments for cancer patients, especially in those areas of high unmet medical need. Mr. Philips will be presenting at 3:00 PM EST on Tuesday March 3 at the New York Athletic Club.

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