On August 7, 2019 Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, reported second quarter 2019 financial results and recent business highlights (Press release, Sangamo Therapeutics, AUG 7, 2019, View Source [SID1234538369]).

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"We continue to progress our strategy to develop our diversified portfolio of genomic medicine product candidates using our expertise in gene therapy, cell therapy, genome editing and gene regulation," said Sandy Macrae, CEO of Sangamo. "Last month, we reported updated results for SB-525, our investigational gene therapy for hemophilia A. We are pleased with the emerging clinical profile and competitive positioning of SB-525 and are preparing for a Phase 3 registrational study with our partner Pfizer. The THALES study evaluating ST-400, a gene-edited cell therapy for beta thalassemia being developed with Sanofi, is progressing well and recently enrolled a fourth patient. In the upcoming months, two of our wholly owned programs, ST-920, an investigational gene therapy for Fabry disease, and TX-200, our first CAR-Treg product candidate, are also expected to advance into the clinic."

"We have observed significant increases in efficacy above a defined vector dose threshold with AAV6, the vector we use in our hemophilia gene therapy and our in vivo genome editing clinical programs," Dr. Macrae continued. "These recent insights into the kinetics of AAV6 suggest rational methods for improving the delivery of zinc finger nucleases, which we believe may substantially enhance the efficacy of in vivo genome editing, especially when added to the significantly increased potency that we expect to obtain with our updated gene editing reagents. We believe we can integrate these improvements rapidly and plan to introduce them as the next step forward for our in vivo genome editing clinical development programs. Based on current assumptions, including the timelines for manufacturing, we now expect that our next in vivo genome editing clinical trial will commence by year end 2020."

Recent Highlights

Clinical

In partnership with Pfizer, presented updated Phase 1/2 data for SB-525, an investigational gene therapy for the treatment of adults with hemophilia A. The data showed that SB-525 was generally well-tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels. The first two patients treated at the 3e13 vg/kg dose rapidly achieved normal levels of FVIII activity, with no reported bleeding events or exogenous FVIII usage. The response was durable for at least 24 weeks, the extent of follow-up at the time of the data cut-off. The two patients most recently treated at the 3e13 vg/kg dose level are demonstrating FVIII activity kinetics that appear consistent with the first two patients treated in this dose cohort at similar early time points.

In July, dosed the fifth patient in the 3e13 vg/kg cohort (Patient 11 in the study), completing enrollment of the Phase 1/2 study evaluating SB-525

United States Food and Drug Administration granted regenerative medicine advanced therapy (RMAT) designation for SB-525 gene therapy to treat severe hemophilia A

Preparations have begun to advance SB-525 into a Phase 3 registrational clinical trial, including engagement with regulators and initiating the transfer of the SB-525 manufacturing process to Pfizer

Enrolled a fourth patient into the Phase 1/2 clinical trial for ST-400, an ex vivo gene-edited cell therapy candidate for the treatment of beta thalassemia, which is being developed in partnership with Sanofi. Sanofi is conducting a Phase 1/2 clinical trial evaluating BIVV003, a separate but related gene-edited cell therapy candidate for sickle cell disease.

Activated the first clinical site for the STAAR study evaluating ST-920, an investigational gene therapy candidate for Fabry disease. Sangamo expects to enroll the first patient by year end 2019.

Remain on track to file CTA in 2019 for TX200, a CAR-Treg product candidate, in HLA-A2 mismatched kidney transplantation

Kite, a Gilead Company, is planning to initiate a clinical study of KITE-037, an allogeneic anti-CD19 CAR-T, in 2020.

Research

Published a manuscript detailing the activity of disease allele-selective zinc finger proteins in preclinical models of Huntington’s disease in the July 2019 issue of Nature Medicine

Published a manuscript detailing two new approaches for optimizing the specificity of genome editing with zinc finger nucleases in the August 2019 issue of Nature Biotechnology

Corporate

Hired Gary Loeb as Executive Vice President and General Counsel

Second Quarter 2019 Financial Results

For the second quarter ended June 30, 2019, Sangamo reported a consolidated net loss of $30.3 million, or $0.26 per share, compared to a net loss of $16.6 million, or $0.17 per share, for the same period in 2018. As of June 30, 2019, the Company had cash, cash equivalents, and investments of $450.3 million.

Revenues for the second quarter ended June 30, 2019 were $17.5 million, compared to $21.4 million for the same period in 2018. The decrease was primarily due to a decline of $3.7 million in revenues related to the agreement with Pfizer due to a change in estimate resulting from the expansion of the project scope of the hemophilia A collaboration.

As anticipated, operating expenses increased in the second quarter ended June 30, 2019, reflecting the Company’s growth through the acquisition of TxCell, increased U.S. headcount in support of growth of the preclinical pipeline and clinical development programs, and manufacturing-related activities. Total operating expenses for the second quarter ended June 30, 2019 were $51.1 million, compared to $40.6 million for the same period in 2018. Research and development expenses were $36.5 million for the second quarter of 2019, compared to $29.3 million for the same period in 2018. The increase was primarily due to manufacturing and clinical trial expenses related to the progress of the Company’s clinical development programs. General and administrative expenses were $14.6 million for the second quarter of 2019, compared to $11.3 million for the same period in 2018. The increase was primarily due to increased compensation costs related to headcount growth and increased facility expenses, primarily related to our new Brisbane facility.

Financial Guidance for 2019

Operating Expense: Sangamo expects operating expense of $210 to $220 million for the year ending December 31, 2019.

Cash and Investments: Sangamo projects that current cash, cash equivalents, and investments should provide funds for operations through year end 2021.

Conference Call

Sangamo will host a conference call today, August 7, 2019, at 5:00 p.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 9582057. 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 9582057.

On August 7, 2019 Stemline Therapeutics, Inc. (Nasdaq:STML), a commercial -stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing innovative oncology therapeutics, reported that it intends to offer and sell, subject to market conditions, 5,000,000 shares of its common stock in an underwritten public offering (Press release, Stemline Therapeutics, AUG 7, 2019, View Source [SID1234538368]). All of the shares to be sold in the offering will be offered by Stemline. In addition, Stemline intends to grant the underwriters a 30-day option to purchase up to an additional 750,000 shares of its common stock offered in the public offering.

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J.P. Morgan Securities LLC is acting as book-running manager for the offering.

Stemline intends to use the net proceeds from this offering for (i) commercial activities of ELZONRIS (tagraxofusp; SL-401), (ii) clinical trials for additional indications including CMML, MF, AML, and potentially other diseases such as certain lymphomas; (ii) clinical development of additional pipeline candidates (felezonexor (SL-801), SL-1001, SL-901 and SL-701); (iii) research and development and regulatory activities; (iv) potential acquisitions and in-licensing; and (v) other general corporate purposes.

Stemline has filed a preliminary prospectus supplement to its shelf registration statement on Form S-3 (File No. 333-230341) with the U.S. Securities and Exchange Commission ("SEC") for the proposed public offering of its common stock. The offering will be made only by means of a prospectus and a prospectus supplement, which will be available on the SEC’s web site at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to these securities may also be obtained, when available, by contacting J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, Telephone: (866) 803-9204.

The offering of these securities is being made under an effective shelf registration statement on file with the SEC. This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

About ELZONRIS
ELZONRIS (tagraxofusp-erzs), a CD123-directed cytotoxin, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with blastic plasmacytoid dendritic cell neoplasm (BPDCN). For full prescribing information in the U.S., visit www.ELZONRIS.com. In Europe, a marketing authorization application (MAA) is under review by the European Medicines Agency (EMA). ELZONRIS is also being evaluated in additional clinical trials in other indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and acute myeloid leukemia (AML).

About BPDCN
BPDCN is an aggressive hematologic malignancy with historically poor outcomes and an area of unmet medical need. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123
CD123 is a cell surface target expressed on a wide range of myeloid tumors including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on certain lymphoid malignancies including multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin’s lymphoma (HL), and certain Non-Hodgkin’s lymphomas (NHL). In addition, CD123 has been detected on some solid tumors as well as autoimmune disorders including cutaneous lupus and scleroderma.

On August 7, 2019 Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) reported financial results for the quarter ended June 30, 2019 (Press release, Sunesis, AUG 7, 2019, View Source [SID1234538367]). Loss from operations for the three and six months ended June 30, 2019 was $6.2 million and $11.9 million. As of June 30, 2019, cash and cash equivalents, restricted cash, and marketable securities totaled $17.7 million. Subsequent to the end of the quarter, the Company raised approximately $25.9 million in net proceeds from an underwritten offering.

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"We continue to make progress in the Phase 1b/2 trial of our non-covalent BTK inhibitor vecabrutinib in chronic lymphocytic leukemia and other B-cell malignancies. At the annual European Hematology Association (EHA) (Free EHA Whitepaper) meeting in June, we presented preliminary data demonstrating vecabrutinib’s well-tolerated safety profile and evidence of clinical activity," said Dayton Misfeldt, Interim Chief Executive Officer of Sunesis. "More recently, in July, we announced completion of the safety evaluation period for the 200mg cohort, which continues to support the favorable safety profile with no drug-related serious adverse events experienced to date. We look forward to further defining the profile of vecabrutinib in the current study as we prepare for the upcoming Phase 2. We plan on sharing the next clinical update on the study at the annual American Society of Hematology (ASH) (Free ASH Whitepaper) meeting later this year."

"In addition, we strengthened our balance sheet by completing an equity offering in July with leading biotechnology investors, as well as refinancing our previous loan with a new facility from Silicon Valley Bank in April. The proceeds from our recent offering extends our cash runway through key milestones including the initiation of the Phase 2 portion of the trial."

Recent Highlights

Advanced Phase 1b/2 Trial of Vecabrutinib into 300 mg Cohort. In July 2019, Sunesis opened enrollment in the 300 mg cohort in the Phase 1b/2 trial of its non-covalent BTK inhibitor vecabrutinib in adults with relapsed/refractory chronic lymphocytic leukemia (CLL) and other B-cell malignancies.

Presented Preliminary Data at the EHA (Free EHA Whitepaper) Annual Meeting. In June 2019, Sunesis presented preliminary data from the ongoing vecabrutinib clinical trial at the 24th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Amsterdam. The promising data builds upon vecabrutinib’s safety, activity, pharmacokinetic, and pharmacodynamic profile.

Completion of Public Offering. In July 2019, Sunesis completed underwritten public offerings of shares of its common stock and Series F convertible preferred stock with net proceeds of approximately $25.9 million. The offerings attracted participation from leading biotechnology investors and will allow Sunesis to advance vecabrutinib through important clinical milestones as the ongoing dose-escalation study explores potentially active dose levels.

Secured $5.5 Million Loan. In April 2019, the Company entered into a $5.5 million loan agreement with Silicon Valley Bank (SVB), allowing the Company to retire its existing loan. The new agreement allows the Company to defer any principal repayment on the new loan for more than 18 months, with interest-only payments through 2020 and principal repayment over 24 months beginning in 2021. The new loan also has a lower interest rate and was used to repay the Company’s prior loan.

Announced Executive Promotions. In July 2019, Sunesis promoted Judy Fox, Ph.D. to Chief Scientific Officer, Executive Vice President, Research & Development, and Parvinder (Par) S. Hyare to Senior Vice President, Commercial. We look forward to their leadership as we prepare for the next stage of the Company’s growth.

Financial Highlights

Cash and cash equivalents, restricted cash, and marketable securities totaled $17.7 million as of June 30, 2019, as compared to $13.7 million as of December 31, 2018. This capital, plus the approximately $25.9 million in net proceeds from the July 2019 public offerings, is expected to fund the Company through the initiation of the Phase 2 portion of the ongoing vecabrutinib Phase 1b/2 trial. The increase of $4.0 million was primarily due to $19.0 million net proceeds from issuance of common and preferred stock, and $5.5 million proceeds from SVB Loan Agreement, offset by $13.0 million net cash used in operating activities and $7.5 million used in principal payments to repay the Company’s prior loan.

Research and development expense was $3.7 million and $6.9 million for the three and six months ended June 30, 2019, as compared to $3.8 million and $7.7 million for the same periods in 2018. The decreases between the comparable periods were primarily due to a decrease in salary and personnel expenses due to lower headcount, decrease in professional services related to higher expenses incurred in the first half of 2018 for the start-up costs of the Phase 1b/2 trial for vecabrutinib, offset by an increase in clinical expenses related to the preparation for the Phase 2 portion of the ongoing clinical trial of vecabrutinib.

General and administrative expense was $2.5 million and $5.0 million for the three and six months ended June 30, 2019, as compared to $2.8 million and $6.2 million for the same periods in 2018. The decreases between the comparable periods were primarily due to a decrease in salary and personnel expenses due to lower headcount and stock-based compensation and a decrease in professional services expenses due to lower legal and vosaroxin patent expenses.

Interest expense was $0.1 million and $0.4 million for the three and six months ended June 30, 2019, as compared to $0.3 million and $0.6 million for the same periods in 2018. The decreases in interest expense from both periods resulted from a lower interest rate on a lower principal amount under the SVB Loan Agreement.

Cash used in operating activities was $13.0 million for the six months ended June 30, 2019, as compared to $12.4 million for the same period in 2018. Net cash used in the six months ended June 30, 2019, resulted primarily from the net loss of $12.1 million, partially offset by adjustments for non-cash items of $0.9 million and changes in operating assets and liabilities of $1.8 million. Net cash used in the six months ended June 30, 2018, resulted primarily from the net loss of $14.1 million, partially offset by adjustments for non-cash items of $1.6 million and changes in operating assets and liabilities of $0.1 million.

Sunesis reported loss from operations of $6.2 million and $11.9 million for the three and six months ended June 30, 2019, as compared to $6.6 million and $13.7 million for the same periods in 2018. Net loss was $6.2 million and $12.1 million for the three and six months ended June 30, 2019, as compared to $6.8 million and $14.1 million for the same periods in 2018.

Conference Call Information

Sunesis will host a conference today at 4:30 p.m. Eastern Time. The call can be accessed by dialing (844) 296-7720 (U.S. and Canada) or (574) 990-1148 (international) and entering passcode 2686126. To access the live audio webcast, or the subsequent archived recording, visit the "Investors and Media – Calendar of Events" section of the Sunesis website at www.sunesis.com. The webcast will be recorded and available for replay on the Company’s website for two weeks.

On August 7, 2019 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics (MBTs) to treat age-related diseases and extend healthy lifespan, reported its financial results for the second quarter ended June 30, 2019 (Press release, CohBar, AUG 7, 2019, View Source [SID1234538366]).

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"During the quarter, we were pleased to resume the dosing of subjects in the Phase 1a/1b clinical trial of our lead candidate CB4211 for NASH and obesity, while continuing to progress our evaluation of additional novel peptides targeting fibrotic diseases, cancer, and type 2 diabetes," said Steven Engle, Chief Executive Officer. "We believe that CB4211 is the first of a number of candidates that our technology platform will identify for advancement into the clinic. Our seminar in June also provided strong validation from world class researchers and CohBar scientists of the potential of mitochondrial based therapeutics, as well as CohBar’s position as a leader in this emerging field. It’s an exciting time to be at CohBar."

Business Highlights

●Resumed CB4211 clinical trial: Dosing of subjects for the company’s Phase 1a/1b clinical trial of CB4211 resumed in June, and has continued in the current quarter for its lead MBT candidate for the potential treatment of nonalcoholic steatohepatitis (NASH) and obesity. The trial design is available at www.clinicaltrials.gov.

●Advanced new potential disease targets: During the second quarter, the company advanced its evaluation and optimization of novel analogs of mitochondrial-derived peptides for the potential treatment of fibrotic diseases. Preclinical studies provided preliminary evidence of anti-fibrotic activity in cell-based and animal models of idiopathic pulmonary fibrosis. Fibrotic disease is a major unmet medical need contributing to approximately one-third of deaths worldwide. The company also advanced its evaluation of novel analogs for the potential treatment of cancer, where in vitro data demonstrated the potential for CohBar peptides to enhance the killing of cancer cells by human immune cells. The market for cancer immunotherapy is projected to grow significantly to over $100 billion by 2023.

●Presented a novel scientific discovery at the American Diabetes Association annual conference: In June, CohBar presented its recent discovery of a mechanism of action of a family of novel peptides with positive effects on glucose tolerance in animal models of type 2 diabetes. The studies showed novel peptide analogs of CB5064 are effective in diet induced obese (DIO) mice, a widely used model of type 2 diabetes. Additional studies also demonstrated that CB5064 interacts with the apelin receptor, a key cell surface receptor involved in the regulation of glucose utilization, fluid homeostasis and cardiovascular function. Click link to ADA poster.

●Appointed new CEO and expanded Board of Directors: In May, Steven Engle was appointed as the company’s Chief Executive Officer. Mr. Engle has over two decades of executive leadership experience with public biotech companies developing breakthrough products in metabolic, autoimmune, oncologic and infectious disease areas. In April, the company expanded its Board with the addition of David Greenwood, who brings more than 30 years of financial and operational experience in biotechnology and investment banking.

●Hosted Mitochondria Based Therapeutics Seminar with key opinion leaders: In May, the company hosted a seminar, entitled "Mitochondria, a Source for Novel Therapeutics," which featured CohBar’s founders, Dr. Pinchas Cohen and Dr. David Sinclair, and CohBar’s Chief Science Officer, Dr. Ken Cundy, discussing the role of mitochondria in health and aging, and mitochondrial-derived peptides (MDPs) as a novel source of potential therapeutics for a host of major age-related diseases. A recording of the webcast is available at www.cohbar.com or by clicking on this link.

●Held Annual General Meeting and review: In June, the company held its Annual General meeting and provided a review and update on the company’s progress and plans. The slides are available on the company’s website at www.cohbar.com.

During the second quarter and more recently, Dr. Pinchas Cohen and Dr. Nir Barzilai continued to be recognized as international leaders in the study of mitochondrial science, aging and age-related diseases:

●Dr. Cohen delivered a keynote presentation on "Systems Biology of the Mitochondria in Aging" at the American Aging Association Annual Meeting in San Francisco, in May 2019; and "Addressing the Challenges of an Aging Society Through Innovation" at a panel entitled "Forum on Aging," at the G20 Meeting in Japan in June 2019. In addition, Dr. Cohen co-authored "Metabolomic profile of diet-induced obesity mice in response to humanin and small humanin-like peptide 2 treatment," published in Metabolomics, and "The mitochondrial-derived peptide MOTS-c is a regulator of plasma metabolites and enhances insulin sensitivity," published in Physiological Reports. He also authored an editorial published in The Journal of Molecular Medicine entitled "MOTS-c: an equal opportunity insulin sensitizer."

●Dr. Barzilai delivered a keynote presentation, entitled "Advancements in Genomics," at the Glenn Center Harvard Symposium in May 2019, and the keynote address at the Nutrition and Obesity Research Center (NORC) at the Harvard Annual Symposium in July 2019. In addition, Dr. Barzilai presented data at the Biology of Aging, Gordon Research Conference on Molecular, Cellular and Physiological Determinants of Lifespan and Healthspan. More recently, Dr. Barzilai co-authored three papers on "Creating the Next Generation of Translational Geroscientists," "The Development of Clinical Trials to Extend Healthy Lifespan," and a highly publicized paper in Nature on exome sequencing in type 2 diabetes.

Financial Highlights

●Cash and Investments. CohBar had cash and investments of $16.8 million as of June 30, 2019, compared to $22.2 million as of December 31, 2018. The cash burn for the quarter ended June 30, 2019, was approximately $2.9 million.

●R&D Expenses. Research and development expenses were $1.4 million in the three months ended June 30, 2019, compared to $1.8 million in the prior year quarter. The decrease was primarily due to the timing of certain preclinical and clinical costs incurred in the prior year period partially offset by an increase in costs associated with our research programs focused on the continuing development of peptides.

●G&A Expenses. General and administrative expenses were $1.5 million for the three months ended June 30, 2019, compared to $1.3 million in the prior year quarter. The increase in general and administrative expenses was primarily due to legal expenses related to the protection of our intellectual property, recruiting costs, and increased director fees.

●Net Loss. For the three months ended June 30, 2019, net loss, which included $0.8 million of non-cash expenses, was $3.1 million, or $0.07 per basic and diluted share. For the three months ended June 30, 2018, net loss, which included $0.9 million of non-cash expenses, was $3.3 million, or $0.08 per basic and diluted share.

Second Quarter Investor Call and Slide Presentation:

Date: August 7, 2019

Time: 5:00 p.m. EDT (2:00 p.m. PDT)

Conference Audio

-Dial-in U.S. and Canada: (800) 239-9838
-Dial-in International: (323) 794-2551
-Conference ID No.: 8671787

Slide Presentation

-Go to www.webex.com, click on the ‘Join’ button and enter meeting number 923 119 468 and Password CWBR, or
-Go to www.cohbar.com and click on Q2 2019 Shareholder Presentation at top of homepage.

For individuals participating in the Investor Call and Slide Presentation, please call into the conference audio and log into Webex approximately 10 minutes prior to its start.

An audio recording of the call will be available beginning at 8:00 p.m. (EDT) on August 7, 2019, through August 28, 2019. To access the recording please dial 1-844-512-2921 in the U.S. and Canada, or 1-412-317-6671 internationally, and reference Conference ID No. 8671787. The audio recording along with the slide presentation will also be available at www.cohbar.com during the same period.

About CB4211

CohBar’s lead clinical program is based on CB4211, a first-in-class mitochondria based therapeutic, that has demonstrated significant therapeutic potential in preclinical models of nonalcoholic steatohepatitis (NASH) and obesity. CB4211 is a novel and improved analog of MOTS-c, a naturally occurring mitochondrial-derived peptide, which has been shown to play a significant role in the regulation of metabolism, and was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes an evaluation of biological activity relevant to NASH and obesity. NASH is a chronic and silent disease in its early stages that can progress to more serious disease stages, such as advanced fibrosis, cirrhosis, liver failure or liver cancer. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is no approved treatment for the disease.

On August 7, 2019 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended June 30, 2019 (Press release, Opko Health, AUG 7, 2019, View Source [SID1234538365]).

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Business Highlights

RAYALDEE total prescriptions reported by IQVIA increased 92% in 2Q 2019 compared with 2Q 2018: Total prescriptions for the three months ended June 30, 2019 increased to approximately 12,700, compared with approximately 6,600 (as adjusted by IQVIA) during the comparable period of 2018.

4Kscore receives a new proposed local coverage determination from Novitas Solutions and utilization remained strong during 2Q 2019 with approximately 18,800 tests performed: Novitas Solutions, Inc. has issued a new proposed local coverage determination (LCD) for the 4Kscore test, with defined coverage criteria. Under the LCD, Medicare proposes reimbursing the test for patients who meet the defined criteria. The LCD is subject to a comment period ending August 11, 2019.

OPKO Plans to Refile 4Kscore De Novo Submission as PreMarket Approval Application (PMA): The Company plans to refile its 4Kscore test submission as a PMA application based on FDA feedback to the Company’s de novo request for this test, which has now been withdrawn. The FDA did not request any additional clinical data or any additional clinical trials in connection with a PMA submission. The Company currently anticipates submitting the PMA application in August 2019.

BioReference Laboratories formed a strategic collaboration with SOMOS, New York City’s largest multi-cultural physician led network: BioReference is now SOMOS’ preferred provider of diagnostic testing and will assist with data analytics for its patients. This collaboration is designed to streamline patient care and offer communication efficiencies between BioReference and the SOMOS network. SOMOS will leverage its relationship with BioReference to improve patient outcomes among its participants while decreasing the overall cost of care.

BioReference and its GeneDx subsidiary expand access to commercially insured lives: BioReference and GeneDx have been selected for inclusion in the UnitedHealthcare Preferred Lab Network beginning July 1, 2019. This was a comprehensive process that evaluated service

standards, turnaround time, quality and accreditation. More than 300 laboratories were invited to apply; only 100 submitted applications due to the complexity of the requirements, and BioReference and GeneDx, along with five other laboratories, earned a place in the Preferred Lab Network. In addition, effective April 1, 2019, BioReference and GeneDx are now in-network providers with Humana, which provides access to 11 million additional lives.

Pharmaceutical pipeline continues to progress. Last patient, last visit for our pivotal global Phase 3 clinical trial for hGH-CTP in growth hormone deficient children is expected this month. Topline data from the trial are expected to be announced by the end of 2019. The ongoing open-label phase 2 trial for RAYALDEE in hemodialysis patients is progressing well and initial data are expected by the end of 2019.

RAYALDEE Marketing Authorization Application filed by Vifor Fresenius in Switzerland. With several Marketing Authorization Applications already accepted and under review in other European countries, Vifor expects to receive approvals in several European countries to market RAYALDEE for the treatment of secondary hyperparathyroidism in adult non-dialysis patients with chronic kidney disease during 2020.

Financial Highlights

Consolidated revenues for the second quarter of 2019 were $226.4 million, compared with $263.7 million for the comparable period of 2018. During the 2019 quarter, revenue from services was $178.5 million, revenue from products was $28.7 million, including RAYALDEE net revenue of $5.7 million, and revenue from licensing and intellectual property was $19.2 million.

Operating expenses for the second quarter of 2019 were $273.6 million. This included continued investment in the company’s pharmaceutical pipeline, with R&D expense of $28.3 million, principally for pediatric trials with the hGH-CTP long-acting human growth hormone product.

The net loss for the three months ended June 30, 2019 was $59.8 million, compared with a net loss of $6.2 million for the comparable period of 2018 principally as a result in the decrease in revenue from services as wells as the comparable period of 2018 including a $15.4 million reversal of contingent consideration expense compared to $3.8 million for the 2019 period.

Cash, cash equivalents and marketable securities were $111.1 million as of June 30, 2019, which includes the repayment of a line of credit during the second quarter of approximately $40 million.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN: 866-634-2258
INTERNATIONAL DIAL-IN: 330-863-3454
PASSCODE: 7984069
WEBCAST: OPKO 2Q19 Results Conference Call

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 7984069. The replay can be accessed for a period of time on OPKO’s website at OPKO 2Q19 Results Conference Call.