On August 7, 2018 ADC Therapeutics, an oncology drug discovery and development company that specializes in the development of proprietary antibody drug conjugates (ADCs), reported that the first patient has been dosed in its Phase II clinical trial intended to support the submission of Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) (Press release, ADC Therapeutics, AUG 7, 2018, View Source [SID1234596075]). The clinical trial is evaluating the efficacy and safety of ADCT-402 (loncastuximab tesirine) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

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At the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, the Company presented interim Phase I data on ADCT-402 in 138 evaluable, heavily pre-treated lymphoma patients who had failed, or were intolerant to, any established therapy known to provide clinical benefit, with a median of three prior therapies. At the time, for the 49 response-evaluable patients in Part 1 of the study (dose escalation) with DLBCL who received ADCT-402 at doses greater than or equal to 120 μg/kg, the overall response rate (ORR) was 55 percent (27/49), with 18 patients achieving a complete response (37 percent) and 9 patients achieving a partial response (18 percent).

The primary endpoint of the Phase II, multi-center, open-label, single-arm trial is the ORR in patients treated with ADCT-402, as confirmed by central review. Secondary endpoints include assessments of duration of response, complete response rate, relapse-free survival, progression-free survival and overall survival, as well as safety, pharmacokinetics and health-related quality of life. The trial will enroll approximately 140 patients with relapsed or refractory DLBCL at multiple centers in the USA and Europe.

"We are pleased to have dosed the first patient in our registrational Phase II clinical trial evaluating ADCT-402 in patients with DLBCL who have relapsed and have refractory disease after two or more multi-agent treatment regimens. Our Phase I clinical trial of ADCT-402 in non-Hodgkin lymphoma showed significant activity in patients with DLBCL and an acceptable safety profile," said Jay Feingold, MD, PhD, Chief Medical Officer and Senior Vice President of Clinical Development at ADC Therapeutics. "Unfortunately, there is no effective treatment for patients with multiple relapsed and refractory DLBCL, so we are excited about the potential to improve outcomes in these patients with ADCT-402 in a single-arm trial. We anticipate reporting results from the Phase II trial in the third quarter of 2019 and are hopeful that the data will support our submission of a BLA to the FDA."

Alex Spira, MD, PhD, FACP, Director of Virginia Cancer Specialists Research Institute and Clinical Assistant Professor of Oncology at Johns Hopkins School of Medicine, added, "Patients with DLBCL who have relapsed or are refractory after second-line chemotherapy face a very poor prognosis. There is a

significant unmet need for an effective new treatment option for this patient population, and we believe ADCT-402 has the potential to help impact patient outcomes in this disease."

For more information about the Phase II clinical trial, please visit www.clinicaltrials.gov (identifier NCT03589469).

ADC Therapeutics also plans to initiate multiple combination studies with ADCT-402 in the fourth quarter of 2018.

About Diffuse Large B-Cell Lymphoma (DLBCL)

Non-Hodgkin lymphoma (NHL) is the seventh most common type of cancer in the U.S., and accounted for an estimated 4.3 percent of new cancer cases in 2017.1 Diffuse large B-cell lymphoma (DLBCL) accounts for nearly one-third (32.5 percent) of NHL.2 The most common initial treatment for patients with DLBCL is chemo-immunotherapy. Response to initial treatment is high, but more than half of patients do not have long-term disease control.3 The current standard of care for relapsed DLBCL is additional chemotherapy, which can be followed by stem cell transplantation (SCT). The prognosis for relapsed patients is poor, especially for those with chemotherapy-refractory disease with a short interval between remission and relapse or those who relapse after high-dose therapy and SCT. There is a significant unmet need for an effective treatment for patients with relapsed or refractory DLBCL.

About ADCT-402

ADCT-402 is an antibody drug conjugate (ADC) composed of a humanized monoclonal antibody that binds to human CD19, conjugated through a linker to a pyrrolobenzodiazepine (PBD) dimer toxin. Once bound to a CD19-expressing cell, ADCT-402 is internalized into the cell where enzymes release the PBD-based warhead. CD19 is a clinically validated target for the treatment of B-cell malignancies. The PBD-based warhead has the ability to form highly cytotoxic DNA interstrand cross-links, blocking cell division and resulting in cell death. Preliminary data from a Phase I clinical trial in relapsed or refractory B-cell non-Hodgkin lymphoma demonstrate ADCT-402 has significant activity in patients with diffuse large B-cell lymphoma (DLBCL). ADCT-402 is also being evaluated in an ongoing Phase I clinical trial in patients with relapsed or refractory B-cell lineage acute lymphoblastic leukemia (B-ALL). The U.S. Food and Drug Administration has granted orphan drug designation to ADCT-402 for the treatment of DLBCL and mantle cell lymphoma.

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 Cancer Genetics, Inc. (Nasdaq: CGIX), a leader in enabling precision medicine for oncology through molecular markers and diagnostics, reported that it will release its financial results for the second quarter ended June 30, 2018 on Tuesday, August 14, 2018 during pre-market hours (Press release, Cancer Genetics, AUG 7, 2018, View Source [SID1234528566]). The Company will hold a conference call at 8:30 a.m. Eastern on Tuesday, August 14, 2018 to discuss the financial results and provide an update on its strategic direction and key organizational improvements being made by the Company.

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CONFERENCE CALL & WEBCAST
Tuesday, August 14, 2018, 8:30 a.m. Eastern Time
Domestic: 800-289-0438
International: 323-794-2423
Conference ID: 1766530
Webcast: View Source
Replay – Available through August 28, 2018
Domestic: 844-512-2921
International: 412-317-6671
Conference ID: 1766530

On August 7, 2018 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of genes reported financial results for the quarter ended June 30, 2018 and provided an update on recent accomplishments and upcoming events (Press release, Syros Pharmaceuticals, AUG 7, 2018, View Source [SID1234528484]).

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"The second half of 2018 promises to be an important time for Syros," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "Over the next several months, we plan to report initial clinical data from our Phase 2 trial evaluating the safety and efficacy of SY-1425 in combination with standard-of-care and targeted agents in genomically defined subsets of patients with acute myeloid leukemia and myelodysplastic syndrome. We plan to open expansion cohorts in the ongoing Phase 1 trial of SY-1365 in multiple patient populations with ovarian and breast cancers, as well as report data from the dose-escalation portion of the trial. Notably, we expect this to be the first-ever reported human clinical data on a selective inhibitor of CDK7, which is gaining increased recognition as an important new drug target in oncology. Together, we believe these clinical results will provide important insights into these programs and hopefully bring us closer to our vision of translating our leadership in gene control into medicines that provide profound benefit for patients."

Upcoming Milestones

Syros plans to report initial clinical data in the fourth quarter of 2018 from cohorts in its Phase 2 trial evaluating the safety and efficacy of SY-1425 in combination with azacitidine in RARA and IRF8 biomarker-positive patients with newly diagnosed acute myeloid leukemia (AML) who are not suitable candidates for standard chemotherapy, and in combination with daratumumab in biomarker-positive patients with relapsed or refractory AML and higher-risk myelodysplastic syndrome (MDS).
Syros plans to open expansion cohorts in the fall of 2018 in its Phase 1 trial of SY-1365. These expansion cohorts will evaluate SY-1365 as a single agent and in combination with standard-of-care therapies in multiple patient populations with ovarian and breast cancers.
Syros plans to report clinical data in the fourth quarter of 2018 from the dose escalation portion of its Phase 1 trial of SY-1365 in patients with advanced solid tumors.
Syros plans to select a new development candidate from its preclinical pipeline by the end of 2018.
Recent Pipeline Highlights

In July 2018, the European Medicines Agency (EMA) granted SY-1425 orphan drug designation for the treatment of AML. The EMA orphan drug designation is granted to medicines being developed for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition with a prevalence of not more than five in 10,000 people in the European Community. EMA orphan drug designation benefits include protocol assistance, access to the EU centralized authorization procedure, reduced EU regulatory filing fees and 10 years of market exclusivity.
In June 2018, Syros presented on the design of its Phase 1 clinical trial of SY-1365 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Based on preclinical data, which showed robust anti-tumor activity in ovarian and breast cancers, Syros designed the expansion phase of its trial to initially focus on these tumors. Expansion cohorts will evaluate the safety, tolerability and preliminary clinical activity of SY-1365 in:
Three ovarian cancer patient populations in earlier- and later-stages of disease, as either a single agent or in combination with carboplatin;
Hormone receptor-positive, CDK4/6 inhibitor-resistant breast cancer patients, in combination with fulvestrant;
Patients with solid tumors of any histology to evaluate pharmacodynamic endpoints and measures of biological activity in paired tumor biopsies.
Recent Corporate Highlights

In June 2018, Syros announced the appointment of Michael W. Bonney, Chief Executive Officer of Kaleido Biosciences, to its Board of Directors.
Second Quarter 2018 Financial Results

Cash, cash equivalents and marketable securities as of June 30, 2018 were $124.4 million, compared with $72 million on December 31, 2017. During the second quarter, Syros sold $16.6 million in common stock under its at-the-market sales facility.

For the second quarter 2018, Syros reported a net loss of $14 million, or $0.43 per share, compared to a net loss of $13.4 million, or $0.52 per share, for the same period in 2017.

Revenues were $0.4 million for the second quarter of 2018, which relate entirely to Syros’ collaboration with Incyte. Syros did not record revenues in the second quarter of 2017.
Research and development (R&D) expenses were $11.1 million for the second quarter of 2018, as compared to $10 million for the same period in 2017. This increase was primarily attributable to an increase in costs associated with Syros’ Phase 1 clinical trial of SY-1365 and increased headcount.
General and administrative (G&A) expenses were $3.8 million for the second quarter of 2018, as compared to $3.5 million for the same period in 2017. This increase was primarily attributable to an increase in employee-related costs.
Financial Guidance

Based on its current plans, Syros believes that its existing cash, cash equivalents and marketable securities will be sufficient to fund its planned operating expenses and capital expenditure requirements into 2020.

Conference Call and Webcast:

Syros will host a conference call today at 8:30 a.m. ET to discuss these second quarter 2018 financial results and provide a corporate update.

The live call may be accessed by dialing (866) 595-4538 for domestic callers or (636) 812-6496 for international callers and referencing conference ID number: 8675009. A live webcast of the conference call will be available online on the Investors & Media section of the Syros website at www.syros.com. An archived replay of the webcast will be available for approximately 90 days.

On August 7, 2018 Alder BioPharmaceuticals, Inc. (NASDAQ:ALDR), a biopharmaceutical company focused on developing novel therapeutic antibodies for the treatment of migraine, reported its financial results for the second quarter ended June 30, 2018 (Press release, Alder Biopharmaceuticals, AUG 7, 2018, View Source [SID1234528500]).

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"During the second quarter, the new data we presented from our Phase 3 clinical trials in episodic and chronic migraine further highlighted eptinezumab’s encouraging clinical profile for migraine prevention, including increased efficacy following additional quarterly infusions," said Robert W. Azelby, president and chief executive officer of Alder. "Alder is focused on changing the treatment paradigm for the millions of patients living with the disabling effects of migraine and we remain on track to submit a high-quality BLA to the U.S. Food and Drug Administration in the first quarter of 2019."

Recent Company Highlights

Presented new PROMISE 1 and PROMISE 2 Phase 3 clinical trial data for eptinezumab, Alder’s lead investigational product candidate for migraine prevention targeting calcitonin gene-related peptide (CGRP), at the American Academy of Neurology (AAN) and the American Headache Society Meeting (AHS) during the second quarter. Highlights include:
One-year data from the PROMISE 1 Phase 3 trial demonstrated long-term and increasing efficacy in episodic migraine following the third and fourth quarterly infusions.1
Six-month data for eptinezumab from the PROMISE 2 Phase 3 trial demonstrated improved efficacy for chronic migraine following the second quarterly infusion.1
These data from PROMISE 1 and PROMISE 2 continue to reinforce eptinezumab’s potential competitive clinical profile.

Alder’s one-year safety study of eptinezumab was completed with a safety profile consistent with previous eptinezumab studies.

All milestones remain on track for Alder’s planned BLA submission in Q1 2019.
Second Quarter 2018 Financial Results

As of June 30, 2018, Alder had $536.1 million in cash, cash equivalents, investments and restricted cash compared to $587.0 million as of March 31, 2018.
Research and development expenses for the second quarter ended June 30, 2018 totaled $52.8 million, compared to $65.3 million for the same period in 2017. The decrease in expenses was primarily due to lower eptinezumab clinical trial expense as the company nears completion of patient treatments for several clinical trials, offset by an increase in compensation as a result of an increase in internal headcount, and consulting fees to support manufacturing activities and the planned BLA submission.
General and administrative expenses for the second quarter ended June 30, 2018 totaled $12.2 million, compared to $9.5 million for the same period in 2017. The increase in spending was primarily due to an increase in stock-based compensation and expenses to support commercial readiness activities.
Net loss applicable to common stockholders for the second quarter ended June 30, 2018 totaled $70.7 million, or $1.04 per share, compared to net loss of $74.6 million, or $1.48 per share on a fully-diluted basis, for the same period in 2017.
Financial Outlook

Alder believes its available cash, cash equivalents, short-term investments and restricted cash will be sufficient to meet the company’s projected operating requirements into 2020.
1. For additional details regarding the trial results, please refer to Alder’s previous data press releases, which can be found at View Source

Conference Call and Webcast
Alder will host a conference call today at 5:00 p.m. ET to discuss these financial results and recent corporate highlights. The live call may be accessed by dialing (877) 430-4657 for domestic callers or (484) 756-4339 for international callers, and providing conference ID number 9588479. The webcast will be broadcast live and can be accessed from the Events & Presentations page in the Investors section of Alder’s website at www.alderbio.com. The accompanying slides are available now at the Events & Presentations page in the Investors section of Alder’s website at www.alderbio.com. The webcast will be available for replay following the call for at least 30 days.