On August 7, 2018 OPKO Health, Inc.(NASDAQ:OPK) reported its financial results and business highlights for the three months ended June 30, 2018 (Press release, Opko Health, AUG 7, 2018, View Source [SID1234528499]).

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Financial Highlights

Net loss for the quarter decreased by 63% to $6.2 million or $0.01 a share on revenues of $263.7 million during the three months ended June 30, 2018, compared to net loss of $16.9 million or $0.03 per share on revenues of $292.6 million for the comparable period of 2017.

Revenues from products during the three months ended June 30, 2018 include $4.8 million from RAYALDEE and revenues from services were $216.1 million for the 2018 period compared with $233.9 million for the corresponding 2017 period.

Revenue and net loss for the three months ended June 30, 2017 benefited from a non-recurring $10 million milestone payment for VARUBI while net loss for the three months ended June 30, 2018 benefited from the reduction of contingent consideration expense of $15.4 million attributable to changes in assumptions regarding the timing of milestone payments.

During the three months ended June 30, 2018, costs of revenue and selling, general and administrative expenses decreased by approximately 9%, or $26.0 million, compared to the 2017 period. Research and Development expenses were $29.2 million compared to $33.1 million for the corresponding 2017 period.
Business Highlights

RAYALDEE total prescriptions reported by IMS for Q2 2018 increased 467% compared with Q2 2017 and 36% compared with Q1 2018: As of August 1, 2018, more than 83% of patients have access to RAYALDEE under their insurance plans. Fresenius Medical Care Renal Pharma (Vifor Fresenius) received approval from Health Canada to market RAYALDEE in Canada for the treatment of secondary hyperparathyroidism (SHPT) in adults with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

4Kscore utilization in Q2 2018 increased 10% compared with Q2 2017. Utilization of the 4Kscore continues to improve with nearly 20,500 4Kscore tests performed during the three months ended June 30, 2018 compared to 18,600 tests performed during the second quarter of 2017.

Geoff Monk Appointed as General Manager, BioReference Laboratories: Mr. Monk has more than 20 years of management experience in the diagnostic laboratory business and was previously Managing Director of the New York and New Jersey unit of Quest Diagnostics.

Advanced the Phase 2b trial for our SARM (selective androgen receptor modulator) to treat benign prostatic hyperplasia (BPH): Enrollment is ongoing in this dose ranging study for our orally administered SARM. This medicine is expected to improve symptoms of BPH by reducing prostate size and, on the basis of data from a previous trial in 350 men, increase muscle mass and bone strength and decrease body fat. BPH affects approximately 50 million men in the U.S.

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, active-controlled study taking place in over 30 countries. This study enrolled approximately 225 treatment naïve children with growth hormone deficiency (GHD) who were randomized 1:1 into two arms: once-weekly somatrogon vs once-daily Genotropin. The primary endpoint of the trial is height velocity at 52 weeks. Secondary endpoints are safety and pharmacodynamic endpoints.

Japanese Phase 3 registration trial of somatrogon in growth hormone deficient children continues to enroll patients: The global and Japanese pediatric studies utilize the multiple dose pen device that will be launched commercially upon approval.

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.
Premarket Approval (PMA) application for Claros point-of-care PSA test under review by FDA: OPKO has submitted a PMA for a PSA test utilizing the Claros 1 immunoassay analyzer, a novel diagnostic instrument that can provide rapid, quantitative blood test results in 10 minutes in the physician’s office with only a finger stick drop of whole blood. A second product utilizing the Claros platform to measure testosterone is advancing toward a 510(k) submission to the FDA.
Initiation of two additional Phase 2 clinical trials anticipated in 2018:

RAYALDEE in dialysis patients with SHPT: Together with our partners, Vifor Fresenius and Japan Tobacco, OPKO is developing RAYALDEE for Stage 5 CKD patients with SHPT undergoing dialysis and anticipates initiating a global Phase 2 trial during Q3 of this year.

OPKO’s NK-1 antagonist to treat pruritus (itching) in Stage 5 CKD patients undergoing dialysis: An Investigational New Drug application was submitted to the FDA and a single-dose Phase 2a trial is commencing. Pruritus is often a problem for renal dialysis patients.
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:

WHEN:
DOMESTIC DIAL-IN:
INTERNATIONAL DIAL-IN:
PASSCODE:
WEBCAST:
Tuesday, August 7, 2018 at 4:30 p.m. Eastern time.
(866) 634-2258
(330) 863-3454
9243717
investor.opko.com/events
For those unable to participate in the live conference call or webcast, a replay will be available beginning August 7, 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: 9243717. The replay can be accessed for a period of time on OPKO’s website at View Source

On August 7, 2018 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results and recent highlights for the second quarter ended June 30 (Press release, Halozyme, AUG 7, 2018, View Source [SID1234528498]).

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"At the beginning of 2018 we projected the potential for approximately $1 billion in ENHANZE royalty revenue in 2027 resulting from continued growth of our 3 currently marketed products and the successful development, approval and launch of 7 additional products," said Dr. Helen Torley, president and chief executive officer. "I am delighted to report strong progress in both the marketed products and the development products, with all 7 new products expected to be in the clinic by the end of this year.

"In tandem, we continue to execute well in our HALO-301 study, with enrollment tracking to expectations and continued enthusiasm and support from key opinion leaders and investigators. We also look forward to advancing our pan-tumor plan by sharing data from our collaboration study with Eisai in breast cancer patients at the European Society for Medical Oncology congress in October."

Second Quarter 2018 and Recent Highlights include:

U.S. Food and Drug Administration (FDA) accepting Roche/Genentech’s Biologics License Application (BLA) for a subcutaneous formulation of Herceptin in combination with Halozyme’s ENHANZE technology in its FDA-approved breast cancer indications. Roche reported total 2017 sales of Herceptin in the United States of 2.7 billion CHF.
Roche initiating a Phase 3 study of a fixed-dose combination of subcutaneous pertuzumab (Perjeta ) and subcutaneous trastuzumab (Herceptin) using Halozyme’s ENHANZE technology in combination with chemotherapy in patients with HER2-positive early breast cancer. This follows supportive Phase 1 study results for the same combination presented at the 2017 San Antonio Breast Cancer Symposium.
Collaboration partner Bristol-Myers Squibb progressing toward three Phase 1 studies with ENHANZE. Studies include evaluation of an investigational anti-CD-73 antibody, an investigational product against an undisclosed target and the PD-1 targeted asset, Opdivo (nivolumab), all planned for initiation in Q3.
Janssen continuing in multiple ongoing trials of a subcutaneous formulation of DARZALEX (daratumumab) in support of plans for commercialization. Halozyme’s ENHANZE technology has the potential to enable a 15-ml injection to be delivered in five minutes or less. Ongoing trials in patients with Multiple Myeloma, Amyloidosis and Smoldering Myeloma include four Phase 3 studies and two earlier stage studies.
Alexion continuing to progress toward initiating a Phase 1 study of ALXN1210 with ENHANZE, planned for later this year.
Acceptance of data from the Phase 1b study of PEGPH20 and HALAVEN (eribulin) in patients with HER2-negative, high-hyaluronan metastatic breast cancer for presentation at the 2018 European Society for Medical Oncology Congress.
U.S. Patent and Trademark Office granting Halozyme a patent for the combination of PEGPH20, ABRAXANE (nab-paclitaxel) and gemcitabine for the potential treatment of metastatic pancreas cancer, with an expiration date of March 2033. The same application is pending or has been issued in multiple countries outside of the United States.
Continued progress screening and enrolling patients in the HALO-301 study of PEGPH20 in combination with ABRAXANE (nab-paclitaxel) and gemcitabine in first-line metastatic pancreas cancer patients with high levels of tumor hyaluronan (HA-High). An interim analysis will be conducted for the first primary endpoint of Progression Free Survival (PFS) when the target number of events has been reached, which the company projects will occur between December 2018 and February 2019.
Second Quarter 2018 Financial Highlights

Revenue for the second quarter was $35.2 million compared to $33.8 million for the second quarter of 2017. The year-over-year increase was driven by $10 million in milestone revenue and 36 percent growth in royalties on a reported basis from partner sales of Herceptin (trastuzumab) SC, MabThera (rituximab) SC, RITUXAN HYCELA and HYQVIA (Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase), offset by the expected decrease in bulk rHuPH20 sales to partners and research and development reimbursements. Revenue for the second quarter included $20 million in royalties and $3.8 million in HYLENEX recombinant (hyaluronidase human injection) product sales.
Research and development expenses for the second quarter were $40.1 million, compared to $38.3 million for the second quarter of 2017.
Selling, general and administrative expenses for the second quarter were $14.4 million, compared to $13.1 million for the second quarter of 2017.
Net loss for the second quarter was $22.9 million, or $0.16 per share, compared to net loss in the second quarter of 2017 of $30.8 million, or $0.23 per share.
Cash, cash equivalents and marketable securities were $398.9 million at June 30, 2018, compared to $469.2 million at December 31, 2017.
Financial Outlook for 2018

For the full year 2018, the company updated its prior guidance ranges for net revenue and year-end cash, now expecting:

Net revenue increasing from the prior range of $115 million to $125 million to $125 million to $135 million, driven by milestones from ENHANZE Phase 1 study initiations;
Operating expenses to continue to be in the range of $230 million to $240 million;
Operating cash burn to continue to be in the range of $75 million to $85 million; and
Year-end cash balance increasing from the prior range of $305 million to $315 million to $310 million to $320 million, driven by ENHANZE milestones partially offset by a modest build in rHuPH20 inventory in anticipation of future partner demand.
Webcast and Conference Call

Halozyme will webcast its Quarterly Update Conference Call for the second quarter of 2018 today, Tuesday, August 7 at 4:30 p.m. ET/1:30 p.m. PT. Dr. Torley will lead the call, which will be webcast live through the "Investors" section of Halozyme’s corporate website and a recording made available following the close of the call. To access the webcast and additional documents related to the call, please visit halozyme.com approximately fifteen minutes prior to the call to register, download and install any necessary audio software. The call may also be accessed by dialing (877) 410-5657 (domestic callers) or (334) 323-7224 (international callers) using passcode 769890. A telephone replay will be available after the call by dialing (877) 919-4059 (domestic callers) or (334) 323-0140 (international callers) using replay ID number 40189200.

On August 7, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the second quarter ended June 30, 2018, and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, AUG 7, 2018, View Source [SID1234528497]).

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"We made substantial progress during the second quarter, with key data readouts expected later in 2018," said Bellicum’s President & CEO Rick Fair. "Registration studies with our polyclonal allogeneic cell therapy, BPX-501, are on track and we expect to file MAAs in the European Union in 2019. In anticipation of these filings, we have begun to staff a European organization and initiate pre-launch activities. In addition, based on the encouraging results to date in pediatric leukemias, we are advancing plans to expand the BPX-501 opportunity with a late-stage trial in adult AML patients."

Continued Mr. Fair: "The Phase 1 study of our lead GoCAR-T program, BPX-601, continues to enroll and we plan to report initial results before the end of 2018. We are also hard at work preparing IND/IMPD regulatory submissions for two new dual-switch GoCAR-T programs we expect to enter the clinic in 2019."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

Registration Studies on Track in Europe with BPX-501
Prospective enrollment was completed in the BP-004 and C-004 pediatric trials in patients with leukemias, lymphomas, and inherited blood disorders. These trials will serve as the basis for the planned 2019 European MAA regulatory filings. During the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June, Bellicum reported compelling interim data from BP-004:

Additional interim results from both the BP-004 and the C-004 clinical trials will be presented at a medical conference later this year, with final results expected in early 2019. Bellicum also continues to advance its plans to initiate a late-stage trial of BPX-501 in adult AML patients by the end of the year.

Commercial Planning Activities Underway in Europe for BPX-501
Bellicum named Thierry Darcis, M.D., M.B.A., as General Manager of Europe to lead commercialization plans for BPX-501. Dr. Darcis has extensive experience launching orphan products in Europe and has led commercial teams that supported successful product introductions for ViroPharma and NPS Pharmaceuticals. He also held leadership roles with Zogenix, Novartis Vaccines and GlaxoSmithKline.

BPX-601 and BPX-701 Trials Advancing
BPX-601 is Bellicum’s first GoCAR-T product candidate and is the first controllable CAR-T cell to enter clinical trials. The Company expects to report preliminary findings from the Phase 1 dose-escalation safety study of BPX-601 in adults with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA) later this year, and is currently implementing an amendment to expand the study to gastric and prostate cancer patients. A Phase 1 study of BPX-701 continues to screen patients, and additional clinical sites are being added to the trial to accelerate enrollment.

Second Quarter 2018 Financial Results

Bellicum reported a net loss of $24.2 million for the second quarter of 2018 and $47.0 million for the six months ended June 30, 2018, respectively, compared to a net loss of $24.5 million and $46.4 million for the comparable periods of 2017. The results included non-cash, share-based compensation charges of $3.6 million and $7.2 million for the second quarter and six months ended June 30, 2018, and $3.2 million and $6.6 million for the comparable periods in 2017.

As of June 30, 2018, cash, restricted cash and investments totaled $135.3 million. Based on current operating plans, Bellicum continues to expect that current cash resources will be sufficient to meet operating requirements through 2019.

Research and development expenses were $18.4 million and $34.9 million, for the three and six months ended June 30, 2018, respectively, compared to $18.0 million and $33.3 million during the comparable periods in 2017.

General and administrative expenses were $5.4 million and $11.1 million for the three and six months ended June 30, 2018, respectively, compared to $5.5 million and $11.4 million during the comparable periods in 2017.

At June 30, 2018, Bellicum had 43,346,220 shares of common stock outstanding.

Conference Call and Webcast
Bellicum management will host a webcast and conference call at 5:00 p.m. Eastern today to discuss the financial results and provide a corporate update. To access the call, participants should dial 877-407-3103 (domestic) and 201-493-6791 (international) at least 10 minutes prior to the start of the call. The event will be webcast live and can also be accessed in the Investors & Media section of bellicum.com. An archived version of the webcast will also be available for replay in the Investors & Media section of the Bellicum website for at least two weeks following the call.

About BPX-501
BPX-501 is an adjunct T cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD or other T-cell mediated transplant complications occur. This may enable physicians to more safely perform stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections, and enhance graft-versus-leukemic activity while minimizing GvHD side effects.

About BPX-601
BPX-601 is a GoCAR-T product candidate containing Bellicum’s proprietary inducible MyD88/CD40, or iMC, activation switch, designed to treat solid tumors expressing prostate stem cell antigen, or PSCA. Preclinical data show enhanced T cell proliferation, persistence and in vivo anti-tumor activity compared to traditional CAR-T therapies. In addition to pancreatic cancer, PSCA is expressed in several other solid tumor indications, including gastric and prostate cancers.

About BPX-701
BPX-701 is a high affinity T cell receptor product candidate designed with the CaspaCIDe safety switch. In preclinical studies, PRAME-specific clones showed high reactivity against a panel of PRAME positive tumor cell lines, metastatic melanoma, sarcomas and neuroblastoma tissues. In vitro study data showed that BPX-701 demonstrated strong affinity to panels of cancer cells presenting PRAME peptides and low affinity to non-tumor cells, as well as elimination of BPX-701 cells in response to rimiducid.

On August 7, 2018 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2018 third quarter ended June 30, 2018. The company is hosting a conference call at 4:30 p.m. EDT to discuss results (Press release, Arrowhead Pharmaceuticals, AUG 7, 2018, View Source [SID1234528496]).

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 8452059.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 8452059.

Selected Fiscal 2018 Third Quarter and Recent Events

Made presentations at the EASL International Liver Congress, including:
Preclinical data on ARO-AAT, the second generation candidate for the treatment of alpha-1 antitrypsin deficiency liver disease
Preclinical data on ARO-HBV, the third generation clinical candidate for the treatment of chronic hepatitis B virus infection
Clinical data on ARC-520, a prior generation compound for HBV
Presented preclinical data at various medical meetings on the growing pipeline, including data on two cardiometabolic candidates ARO-APOC3 and ARO-ANG3 and the first candidate targeting the lung, ARO-ENaC
Completed enrollment and dosing of the single ascending dose portion of the ongoing Phase 1/2 study of ARO-HBV and began dosing HBV patients in the multiple ascending dose portion of the study
Completed enrollment of the Phase 1 study of ARO-AAT
Received a positive EMA opinion on orphan designation for ARO-AAT, this follows orphan drug designation that was previously granted by the US FDA
Presented initial clinical data on ARO-AAT at the Alpha-1 National Education Conference, representing this was the first clinical data presented on the proprietary Targeted RNAi Molecule (TRiMTM) platform
Announced that Amgen had administered the first dose of AMG 890, formerly ARO-LPA, in a Phase 1 clinical study, which earned Arrowhead a $10 million milestone payment

On August 7, 2018 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that Scott Wolchko, President and Chief Executive Officer, will present at the 2018 Wedbush PacGrow Healthcare Conference in New York on Tuesday, August 14, 2018 at 2:30 p.m. ET (Press release, Fate Therapeutics, AUG 7, 2018, View Source [SID1234528495]).

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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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A live webcast of the presentation will be available through the investor relations section of the Company’s website at www.fatetherapeutics.com. Following the live webcast, an archived replay will be available on the Company’s website.