On November 9, 2018 OPKO Health, Inc. (NASDAQ: OPK) reports financial results and business highlights for the three months ended September 30, 2018 (Press release, Opko Health, NOV 9, 2018, View Source [SID1234531170]).

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Financial Highlights

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Net loss for the three months ended September 30, 2018 decreased by 23% to $27.7 million or $0.05 a share compared to net loss of $35.9 million or $0.06 per share for the comparable 2017 period. Total revenues improved to $249.8 million during the three months ended September 30, 2018 compared to $246.0 million for the comparable period of 2017.
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Revenues from products during the three months ended September 30, 2018 include $5.8 million from RAYALDEE and revenues from services were $202.8 million for the 2018 period compared with $200.9 million for the corresponding 2017 period.
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During the three months ended September 30, 2018, costs of revenue and selling, general and administrative expenses decreased by approximately 8%, or $19.5 million, compared to the 2017 period. Research and Development expenses were $30.2 million compared to $32.5 million for the corresponding 2017 period.
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On November 8, 2018, OPKO secured approximately $150 million of additional capital, consisting of a private placement of common stock resulting in proceeds of $92.5 million, and an unsecured credit line of $60 million.

Business Highlights

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RAYALDEE total prescriptions reported by IMS for Q3 2018 increased 222% compared with Q3 2017 and 18% compared with Q2 2018: As of November 1, 2018, approximatley 79% of patients have access to RAYALDEE under their insurance plans.
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Initiated the Phase 2 clinical trial to study the safety and efficacy of RAYALDEE as a new treatment for secondary hyperparathyroidism (SHPT) in adults with vitamin D insufficiency and stage 5 chronic kidney disease (CKD) requiring hemodialysis. The trial will be conducted at multiple dialysis centers in the U.S. in two sequential cohorts. The first cohort of approximately 44 patients will be treated for 26 weeks in a randomized, open-label fashion with

either RAYALDEE or placebo to identify the appropriate dosing to be studied in the second cohort. Data readout for this first cohort is expected in 2019. The second cohort of more than 200 patients will be treated for 26 weeks in a randomized, double-blind fashion with one of three different doses of RAYALDEE or placebo. The primary efficacy endpoint will be correction of vitamin D insufficiency and control of SHPT. Patients will then be treated with RAYALDEE for another 26 weeks in an open-label extension.
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4Kscore utilization in Q3 2018 was approximately 18,700 tests compared to 18,900 during Q3 2017. Utilization of the 4Kscore remains strong while we continue to work with our Medicare administrator, Novitas, on their proposed local coverage determination. During this process, Novitas has continued to provide coverage of the 4Kscore to Medicare beneficiaries.
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Completed the enrollment of a Global Phase 3 study of somatrogon (hGH-CTP) in Growth Hormone Deficient Children: The somatrogon Phase 3 trial is a randomized, open-label study comparing once-weekly somatrogon to once daily Genotropin. This study has enrolled 228 treatment naïve children with growth hormone deficiency (GHD) in 21 countries. The primary endpoint of the trial is height velocity at 52 weeks. Secondary endpoints are safety and pharmacodynamics.
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Enrollment in Japanese Phase 3 registration trial of somatrogon in growth hormone deficient children expected to complete by year end: The global and Japanese pediatric studies utilize the multiple dose pen device that will be launched commercially upon approval.
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Completed the enrollment of a Phase 2b clinical trial for our once-weekly oxyntomodulin dual GLP1-Glucagon agonist to treat type 2 diabetes and obesity: In a previous Phase 2 trial in 420 overweight patients with type 2 diabetes, the drug was shown to be safe and effective. The current trial is to study a new dosing schedule to achieve even greater weight loss and topline results are anticipated during 1Q 2019.
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Advanced the Phase 2b trial for our SARM (selective androgen receptor modulator) to treat benign prostatic hyperplasia (BPH): Enrollment of approximately 110-120 patients in this dose ranging study of our orally administered SARM is expected to be completed by the end of this year.
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Premarket Approval (PMA) application for Claros point-of-care PSA test under review by FDA; decision anticipated during 1H 2019: OPKO has completed a PMA submission to FDA for Sangia, our point of care PSA test utilizing the Claros 1 immunoassay analyzer. This is the first test on our proprietary diagnostic platform that can provide rapid, quantitative blood test results in the physician’s office with only a finger stick drop of whole blood. Several biomarkers and biological meaningful chemistry tests such as testosterone and Vitamin D utilizing the Claros platform are advancing toward a 510(k) submission to the FDA.

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 4:15 p.m. Eastern time today. The conference call dial-in and webcast information is as follows:

WHEN: Friday, November 9, 2018 at 4:15 p.m. Eastern time
DOMESTIC DIAL-IN: (866) 634-2258
INTERNATIONAL DIAL-IN: (330) 863-3454
PASSCODE: 3487296
WEBCAST: www.investor.opko.com/events

For those unable to participate in the live conference call or webcast, a replay will be available beginning November 9, 2018 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: 3487296. The replay can be accessed for a period of time on OPKO’s website at www.investor.opko.com/events.

On November 9, 2018 Personalis, Inc., a leader in advanced genomics for precision oncology, reported the launch of its universal cancer immunogenomics platform, ImmunoID NeXT (Press release, Personalis, NOV 9, 2018, View Source [SID1234531186]). This is the first platform to enable comprehensive analysis of both a tumor and its microenvironment from a single sample.

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Personalis CEO, John West, said, "While the success of checkpoint blockade has been hugely promising, it’s increasingly apparent that predicting response to immunotherapies and developing new ones requires a more comprehensive approach to tumor immunogenomics. With ImmunoID NeXT, it’s now possible to characterize the complex interactions between the tumor cells and immune cells of the microenvironment using a single platform. This means researchers no longer have to make the difficult choice of which biomarkers to analyze due to sample limitations, particularly when dealing with FFPE specimens."

The unique design of the ImmunoID NeXT Platform facilitates the delivery of therapeutic and diagnostic biomarker information across ~20,000 genes from DNA and RNA, including:

Neoantigen identification and characterization
Human leukocyte antigens (HLA) typing
Tumor infiltrating adaptive immune cells
T-cell receptor (TCR) repertoire (α, β, γ, and δ chains)
B-cell receptor (BCR) repertoire (heavy and light chains)
Tumor infiltrating innate immune cells
Immune response and tumor escape mechanisms
Neoantigen load and tumor mutational burden (TMB)
Microsatellite instability (MSI) status
Oncoviral detection
Germline genomic variation
Personalis is offering pharmaceutical customers the opportunity to participate in an Early Access Program beginning in January, 2019. The company also anticipates the release of a clinical diagnostic test based on this platform in 2019.

Personalis will be presenting new data at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting in Washington, D.C., November 8-11, 2018. Personalis representatives will also be available to discuss the ImmunoID NeXT Platform at the company’s exhibit (Booth #617).

On November 9, 2018 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported that it will present at the following upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, NOV 9, 2018, View Source [SID1234531203]):

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President, will present at the Credit Suisse Healthcare Conference on Wednesday, November 14, 2018 at 11:30 a.m. MT in Scottsdale, Arizona.
Shalini Sharp, the company’s Chief Financial Officer, will present at the Piper Jaffray Healthcare Conference on Tuesday, November 27, 2018 at 11:30 a.m. ET in New York.
Dr. Kakkis will present at the Evercore HealthConX on Wednesday, November 28, 2018 at 11:00 a.m. ET in Boston.
The live and archived webcast of the company presentations will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On November 8, 2018 ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), reported it will present two posters highlighting preclinical data and the Phase Ib clinical trial design for ADCT-301 (camidanlumab tesirine) in advanced solid tumors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting, which is being held November 7-11 in Washington, DC (Press release, ADC Therapeutics, NOV 8, 2018, View Source [SID1234596074]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Jay Feingold, MD, PhD, Chief Medical Officer and Senior Vice President of Clinical Development at ADC Therapeutics, said, "ADCT-301 is already being evaluated in relapsed and refractory Hodgkin lymphoma, and at the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting we will be updating our abstract data from June, at which time we had an overall response rate of 80.8 percent with a complete response rate of 50 percent in median 6th line patients. Based on the immune-oncology potential ADCT-301 demonstrated in preclinical studies, we are excited to be starting a clinical trial for ADCT-301 in solid tumors to see if we can make an impact and improve patient outcomes in multiple solid tumor cancers."

Patrick van Berkel, PhD, Senior Vice President of Research and Development at ADC Therapeutics, said, "ADCT-301 targets CD25, which is expressed on Tregs that infiltrate the local tumor environment. In preclinical models, a single dose of the CD25-targeted ADC induced strong and durable anti-tumor activity against established CD25 negative solid tumors with infiltrating Tregs. Moreover, re-challenged mice did not develop new tumors indicating the CD25-targeted ADC was able to induce tumor-specific protective immunity."

ADC Therapeutics’ posters will be located in Poster Hall E in the Walter E. Washington Convention Center. The Poster Hall will be open Friday, November 9 from 8 a.m. to 8 p.m. and Saturday, November 10 from 8 a.m. to 8:30 p.m. EST. Details of the posters are below.

Abstract Poster Number: P11
Title: A CD25 targeted pyrrolobenzodiazepine dimer-based antibody-drug conjugate shows potent anti-tumor activity in pre-clinical models of solid tumors either alone or in combination with a PD-1 inhibitor
Presentation Date and Time: Friday, November 9, 12:45-2:15 p.m. and 6:30-8 p.m. EST
Presenter: Francesca Zammarchi, PhD, ADC Therapeutics

Abstract Poster Number: P316
Title: Phase 1b dose-escalation and dose-expansion study to evaluate safety, tolerability, pharmacokinetics, and antitumor activity of ADCT-301 (camidanlumab tesirine) in patients with advanced solid tumors
Presentation Date and Time: Saturday, November 10, 12:20-1:50 p.m. and 7-8:30 p.m. EST
Presenter: Francesca Zammarchi, PhD, ADC Therapeutics

For more information about the SITC (Free SITC Whitepaper) 2018 Annual Meeting, please visit View Source

About ADCT-301

ADCT-301 (camidanlumab tesirine) is an antibody drug conjugate (ADC) composed of a monoclonal antibody that binds to CD25 (HuMax-TAC, licensed from Genmab A/S), conjugated to the pyrrolobenzodiazepine (PBD) dimer payload tesirine. Once bound to a CD25-expresing cell, ADCT-301 is internalized into the cell where enzymes release the PBD-based warhead. The intra-tumor release of its PBD warhead may cause bystander killing of neighboring tumor cells. In addition, the PBD warhead will trigger immunogenic cell death, which in turn will strengthen the immune response against tumor cells. ADCT-301 is being evaluated in a Phase Ib clinical trial in solid tumors (NCT03621982), as well as ongoing Phase Ia/Ib clinical trials in patients with relapsed or refractory Hodgkin lymphoma and non-Hodgkin lymphoma (NCT02432235).

On November 8, 2018 Histogenics Corporation (Histogenics) (Nasdaq: HSGX), a leader in the development of restorative cell therapies that may offer rapid-onset pain relief and restored function, reported its financial and operating results for the quarter ended September 30, 2018 (Press release, Histogenics, NOV 8, 2018, View Source [SID1234530923]).

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"We continue to focus on advancing NeoCart towards the potential submission of a Biologics License Application by the end of the first quarter of 2019 and have an extensive dialogue with the FDA covering all aspects of the NeoCart development program, including the clinical data and related statistics from the NeoCart Phase 3 clinical trial," said Adam Gridley, President and Chief Executive Officer of Histogenics. "We believe, based on our continued analysis of the totality of the data, that NeoCart, if approved, may offer patients a treatment alternative that provides a more rapid recovery from pain and return to daily activity than other options to treat damaged knee cartilage. We are also working closely with MEDINET, our development and commercialization partner in Japan, toward the goal of initiating the Japanese Phase 3 clinical trial of NeoCart in 2019."

Third Quarter 2018 and Recent Highlights

Meeting Held with FDA to Discuss Potential NeoCart Regulatory Submission: Histogenics recently met with the United States Food and Drug Administration (FDA) to discuss the NeoCart Phase 3 clinical trial data and the potential for the submission of a Biologics License Application (BLA) for NeoCart. The FDA and Histogenics continue to discuss the clinical data generated to date, the potential need for any additional supplemental clinical data, including longer-term data from the ongoing Phase 3 trial or additional studies, and possible alternative regulatory pathways for acceptance of the BLA. The FDA has not made a final decision regarding a potential BLA submission and based on expected feedback from the FDA, Histogenics intends to provide a further update on these negotiations by the end of November 2018.

NeoCart Top-Line Phase 3 Data Announced in Third Quarter of 2018: Histogenics reported top-line data from the NeoCart Phase 3 clinical trial in September 2018. This trial is believed to be the largest and first prospectively designed, randomized clinical trial in North America evaluating the safety and efficacy of a restorative cell therapy to treat knee cartilage damage. It is also believed to be the only trial with a dual threshold responder analysis endpoint. While the Phase 3 clinical trial did not meet its primary endpoint of a statistically significant improvement in pain and function in the dual threshold responder analysis one year after treatment as compared to microfracture, patients treated with NeoCart saw statistically significant and clinically meaningful improvements in their pain and function levels using a variety of clinically validated scales. In addition, NeoCart demonstrated superiority on the responder analysis endpoint at six months, and in different subgroups at one year such as patients with lesions greater than 2 cm or with higher body mass index. Both NeoCart and microfracture were well tolerated and exhibited strong safety profiles.

Completion of $17.0 Million Financing: In October 2018, Histogenics completed an underwritten public offering of common stock and warrants that generated $17.0 million in gross proceeds and approximately $15.4 million in net proceeds after deducting underwriting discounts and commissions, and estimated offering expenses payable by Histogenics. These amounts do not include the proceeds, if any, that Histogenics may receive in connection with any exercise of the warrants issued in the offering. The financing was led by healthcare-focused, institutional investors and supported by existing Histogenics investors. Histogenics believes the financing will be sufficient to fund its projected cash needs into the middle of 2019.

Development and Presentation of Additional NeoCart Biomechanical Data to Support Potential Commercialization: As part of their Sponsored Research Agreement, Histogenics and Cornell University continue to generate additional biomechanical data to support the potential commercialization of NeoCart, if approved. The most recent data were presented at the Biomedical Engineering Society Annual Meeting in October 2018. These data build upon earlier studies and further demonstrate the importance of the presence of extracellular matrix, or tissue, in making biomechanically competent cartilage. Histogenics believe the data may be important to both regulatory agencies and clinicians regarding the potential performance advantages of NeoCart when compared to other treatment alternatives.
Financial Results for the Third Quarter of 2018

Histogenics’ loss from operations was $(7.0) million in the third quarter of 2018, compared to $(5.7) million in the third quarter of 2017. The increase in loss from operations was due to higher operating expenses in the third quarter of 2018 relative to the third quarter of 2017.

Research and development expenses were $4.6 million in the third quarter of 2018, compared to $3.5 million in the third quarter of 2017. The increase was primarily due to increases in consulting and compensation related expenses in connection with the preparation and evaluation of the data from the NeoCart Phase 3 clinical trial and the potential submission of a BLA for NeoCart with the FDA. General and administrative expenses were $2.4 million in the third quarter of 2018, compared to $2.2 million in the third quarter of 2017. The increase was primarily due to higher salaries and consulting expenses related to increased activities to support the potential commercialization of NeoCart.

As of September 30, 2018, Histogenics had cash, cash equivalents and marketable securities of $5.2 million, compared to $8.0 million at December 31, 2017. In October 2018, Histogenics received net proceeds of approximately $15.9 million through an underwritten public offering of common stock and warrants and sales through its at-the-market offering facility. Histogenics believes its current cash position will be sufficient to fund its operations into the middle of 2019.

Conference Call and Webcast Information

Histogenics management will host a conference call on Thursday, November 8, 2018 at 8:30 a.m. ET. A question-and-answer session will follow Histogenics’ remarks. To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID "5491168" five to ten minutes before the start of the call.

To access a live audio webcast of the presentation on the "Investor Relations" page of the Histogenics website, please click here. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation.