On May 7, 2019 Oncoceutics reported that the US Food and Drug Administration invited the company to present information regarding its lead compound ONC201 at the public advisory committee meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee on June 20, 2019 (Press release, Oncoceutics, MAY 7, 2019, View Source [SID1234558355]).

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The meeting will provide Oncoceutics with the opportunity to present its clinical data for patients treated with ONC201, including studies for both adult and pediatric patients with gliomas containing the H3 K27M mutation, a disease that has been recognized to be an area of high unmet medical need. Additionally, the meeting will enable Oncoceutics to receive feedback from the subcommittee on issues concerning diseases to be studied, patient populations to be included, and possible study designs in the development of ONC201 for pediatric use.

"We are honored and grateful for the opportunity to present to the Pediatric Oncology Subcommittee of the ODAC," said Wolfgang Oster, MD, PhD, Chief Executive Officer and Chairman of Oncoceutics. "ONC201 has shown biological activity that we hope will translate into the clinic and eventually make a difference in patients’ lives. We look forward to furthering the clinical development of ONC201 with the support and guidance of the FDA."

The Public Advisory Committee Meeting will take place in the Great Room of the FDA’s White Oak campus from 8am to 4:30pm. Oncoceutics will present at 12:30pm. Background materials will be available at least 2 business days before the meeting at View Source, and interested persons from the public may present data, information, or views, orally or in writing, on issues pending before the subcommittee.

On May 7, 2019 GeneQuantum Healthcare reported that it has closed nearly RMB 100 million Pre-B round of financing (Press release, GeneQuantum Healthcare, MAY 7, 2019, View Source [SID1234553994]). This financing was led by Huagai Capital, joined by Hofon Capital and Changjinboya. GeneQuantum Healthcare’s A-round investor Oriza Seed and TF Capital continued for additional investments. The funds raised in this round are mainly used to promote the global clinical development of next generation of ADCs, as well as the rapid development and layout of a series of differentiated innovative technologies and products.

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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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GeneQuantum Healthcare focuses on the research and development of various innovative bioconjugate drugs via its innovative LDC technology and the international first-class iLDC platform. The company’s iLDC platform utilizes an enzyme-catalyzed intelligent continuous conjugation process, which has successfully achieved pilot production for next generation of highly homogeneous ADCs with significantly improved quality and reduced costs. The platform process revolutionizes the existing chemical conjugation based production mode and can be widely applied to the automated manufacturing of various bioconjugate drugs represented by ADC drugs. Taking advantage of this platform, GeneQuantum Healthcare has established cooperation with pharmaceutical companies regarding innovative bioconjugate drug research and development.

Dr. Gang Qin, Chairman and President of GeneQuantum Healthcare, said: "GeneQuantum’s innovative ADC is about to initiate clinical trial and the pipeline is rapidly expanding. We are thrilled to be recognized and supported by the leading investors such as Huagai Capital and others at this time point. We look forward to working with investors to expedite our business growth in highly differentiated innovative products, to further expand the application of iLDC platform, and to enhance our unique advantages in precision diagnosis and treatment of tumors, so as to provide innovative treatment plan for cancer patients worldwide. With our advantages in intelligent continuous conjugation platform, we will cooperate with international and domestic pharmaceutical companies to rapidly promote more and better bioconjugate drug development and industrialization."

Mr. Zhiqiang Zeng, the managing partner of Huagai Capital, said: "GeneQuantum is a first class enzymatic-conjugation ADC company in the world. It has independently developed its unique iLDC platform and is capable of growing into an internationally competitive biopharmaceutical leader. The next generation of ADCs developed by GeneQuantum Healthcare will help more patients in the future."

Mr. Qun Zhao, partner of Oriza Seed, said: "We are very optimistic about the potential of enzymatic conjugation technology in the field of ADCs. GeneQuantum Healthcare comprises vast technical accumulation and innovative reserves. We’ll expect the release of GeneQuantum Healthcare’s products to benefit patients as soon as possible."

On May 7, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that it will release its first quarter 2019 financial results and operational highlights before open of U.S. markets on Tuesday, May 14, 2019 (Press release, Autolus, MAY 7, 2019, View Source [SID1234550818]).

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Management will host a conference call at 8:30 a.m. ET/1:30 p.m. BST the same day to discuss the company’s financial results and provide a general business update. To listen to the webcast and view the accompanying slide presentation, please go to: View Source

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 7358198. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 7358198.

On May 7, 2019 Castle Biosciences, Inc., a skin cancer diagnostics company providing personalized genomic information to improve cancer management decisions, reported two presentations at the 2019 American College of Mohs Surgery (ACMS) Annual Meeting held in Baltimore from May 2-5 (Press release, Castle Biosciences, MAY 7, 2019, View Source [SID1234537263]).

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Progress in the development of a prognostic gene expression profile test in cutaneous squamous cell carcinoma (cSCC) was highlighted in an oral presentation. The total number of deaths from cSCC, one of the most common cancers in the U.S., is approaching or surpassing that from melanoma. Many patients with cSCC will have a favorable prognosis, but 25-40% of high-risk subsets will experience metastasis or develop recurrences. Current cSCC staging systems are evolving but more accurate methods of risk prediction are needed to appropriately direct workup and treatment. The goal of this development program is to identify and validate a clinical test that improves upon existing clinicopathologic staging systems to identify patients who have a high risk for regional/distant metastasis and enable more informed clinical management decisions.

In an ongoing development study, primary cSCC specimens with associated clinical outcome data were accrued from 18 centers. The discovery phase of the program included determining genes identified for their expression in recurrent and non-recurrent tumors. Researchers successfully identified genes that exhibited significant differential expression in cSCC samples from patients who experienced a recurrence, including several specific to regional/distant metastasis. In an archival training cohort of 122 patients, gene expression data from 140 candidate genes were used to identify a preliminary gene set and predictive algorithm.

To assess the predictive value of this assay, Kaplan-Meier survival analysis was performed in an independent validation cohort of 107 patients. Results from the analysis support the predictive value of the test to identify two groups of patients with significantly different risk for regional/distant metastasis (p<0.0001). The positive predictive value of the test in this preliminary validation study was 60%, which compares favorably to the performance of current staging systems in this cohort.

Based on these preliminary validation results, an expanded validation program for the cSCC prognostic test is now underway, including additional archival studies and a prospective validation study.

"The continued progress toward validation of this prognostic test for cSCC demonstrates that improvements in risk identification based on tumor biology are feasible," said Sarah T. Arron, M.D., Ph.D., University of California San Francisco and the San Francisco VA Health System, an investigator in the study. "Clinical use of a validated prognostic test such as this could help inform clinical decisions on staging and adjuvant therapy, and improve upon currently used staging systems."

Second study presented at ACMS

A second study presented as a poster at ACMS highlighted results from a meta-analysis evaluating the performance of the DecisionDx-Melanoma gene expression profile test in four unique, independent patient cohorts.

Results from this meta-analysis with 1,479 patients from four independent cohorts confirm that the DecisionDx-Melanoma test is a significant, independent predictor of recurrence and metastasis in patients with Stage I-III melanoma. Patients identified as highest risk (Class 2B) by the DecisionDx-Melanoma test were 2.9 times more likely to experience recurrence than those identified as lowest risk (Class 1A, p<0.0001), independent of other clinicopathologic features. The meta-analysis, a statistical method considered to be among the highest level of evidence (Level 1A) for prognostic tests, supports the use of the DecisionDx-Melanoma gene expression profile test to inform patient management decisions.

About Cutaneous Squamous Cell Carcinoma

Cutaneous squamous cell carcinoma (cSCC), a non-melanoma skin cancer, is one of the most common cancers. Approximately 200,000 patients per year are diagnosed with cSCC with high-risk features. Most patients have a favorable prognosis, but a subset of patients will develop metastasis and up to 15,000 patients each year die from their disease. As current staging parameters have a low positive predictive value, many more patients are considered high risk than actually develop metastatic disease. Conversely, many patients who develop metastatic disease are misidentified as low risk. This may lead to over and undertreatment of a substantial number of cSCC patients. To address this clinical need, Castle Biosciences is developing a gene expression profile test designed to improve upon current staging systems and identify patients with cSCC at high risk for metastasis or recurrence, in order to enable more informed clinical decisions regarding adjuvant therapy and other management options.

On May 7, 2019 OPKO Health, Inc. (NASDAQ: OPK) reported business highlights and financial results for the three months ended March 31, 2019 (Press release, Opko Health, MAY 7, 2019, View Source [SID1234535948]).

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Business Highlights

RAYALDEE total prescriptions reported by IQVIA increased 121% in 1Q 2019 compared with 1Q 2018: Total prescriptions for the first three months of 2019 increased to over 10,400, compared with approximately 4,700 during the comparable period of 2018.

RAYALDEE Marketing Authorization Applications filed by Vifor Fresenius accepted for review in several European countries: These applications request approval to market RAYALDEE for the treatment of secondary hyperparathyroidism (SHPT) in adult non-dialysis patients with chronic kidney disease (CKD).

American Journal of Nephrology published article and editorial: A post hoc analysis of RAYALDEE pivotal trials was published in the March edition of the American Journal of Nephrology and provides the first prospective data demonstrating that the current clinical practice guideline target for serum total 25-hydroxyvitamin D (30 ng/ml) is too low to decrease elevated serum PTH levels in CKD patients. These data show that the correct target is at least 51 ng/mL, and possibly as high as 93 ng/mL, levels no competitive therapy can reliably attain. This analysis received a favorable review in a co-published editorial by prominent nephrology KOLs.

OPK88003 topline results reported from Phase 2 diabetes and obesity trial: In the mITT patient population, which included all patients who had a single dose of drug and one post-baseline efficacy value, HbA1c decreased by -1.3% (p-value <0.0001) and body weight decreased by -4.4 Kg (p-value <0.0001) from baseline. In the Per Protocol patient population, which included patients who were treated for at least 26 weeks and were compliant with the protocol for the duration of the trial, HbA1c decreased by -1.47% (p-value <0.0001) and body weight decreased by -5.5 Kg (p-value <0.0001) from baseline. The safety profile was similar to that expected for the incretin class of drugs, with GI side effects such as nausea, vomiting and diarrhea mostly mild and occurring during the dose-escalation phase.

4Kscore utilization remained strong during 1Q 2019 with approximately 19,400 tests performed: With all required documentation nearly complete, the company plans to make a submission shortly to the FDA for approval or clearance. In addition, OPKO has submitted a request for Medicare coverage reconsideration to its Medicare Administrative Contractor.

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.
Financial Highlights

Consistent with financial guidance issued in February 2019, the net loss for the first quarter of 2019 was $80.8 million, compared with a net loss of $43.1 million for the comparable period of 2018.

Consolidated revenues for the first quarter of 2019 were $222.5 million, compared with $254.9 million for the comparable period of 2018. During the 2019 quarter, revenue from services was $178.9 million, revenue from products was $25.3 million, including RAYALDEE net revenue of $5.8 million, and revenue from licensing and intellectual property was $18.3 million. The decrease in revenue from services principally reflect reimbursement headwinds from higher denial rates in the company’s esoteric testing lines of business.

Operating expenses for the first quarter of 2019 of $297.8 million included continued investment in the company’s pharmaceutical pipeline, with R&D expense of $36.5 million, principally for pediatric trials with the hGH-CTP long-acting human growth hormone product.

In February 2019 OPKO completed the sale of $200 million aggregate principal amount of 4.5% Convertible Senior Notes due 2025, as well as retired and repaid $28.8 million of its 2033 Convertible Notes.

Cash, cash equivalents and marketable securities were $207.3 million as of March 31, 2019.
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, May 7, 2019. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN: 866-634-2258
INTERNATIONAL DIAL-IN: 330-863-3454
PASSCODE: 1547027
WEBCAST: View Source

For those unable to participate in the live conference call or webcast, a replay will be available beginning approximately two hours after the completion of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 1547027. The replay can be accessed for a period of time on OPKO’s website at View Source.