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.

On May 7, 2019 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and, through our license to Santen Pharmaceutical Co. Ltd., wet age-related macular degeneration, reported that it will report its first quarter 2019 financial and operating results after the close of U.S. financial markets on Tuesday, May 14, 2019 (Press release, Tracon Pharmaceuticals, MAY 7, 2019, View Source [SID1234535947]). In addition, management will host a conference call to provide an update on corporate activities and discuss the quarterly financial results.

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Conference call and webcast:
Date: May 14, 2019
Time: 4:30 pm Eastern Time (1:30 pm Pacific Time)
Dial-in: (855) 779-9066 (Domestic) or (631) 485-4859 (International)
Passcode: 9290299
Via web: www.traconpharma.com; "Events and Presentations" section within the "Investors" section
A replay of the webcast will be available for 60 days on the website.

On May 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 first quarter ended March 31, 2019 (Press release, CohBar, MAY 7, 2019, View Source [SID1234535945]).

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"The clear highlight since our last quarterly update was our announcement last week that we will be resuming the clinical trial of our lead therapeutic candidate, CB4211, for NASH and obesity," said Philippe Calais, CohBar’s interim CEO. "At the same time, we are expanding our evaluation efforts with novel analogs of mitochondrial encoded peptides targeting new potential indications, notably type 2 diabetes, cancer and fibrotic diseases. We continue to identify interactions between our novel analogs and new disease models, further validating our belief that mitochondria based therapeutics have significant potential to treat a broad range of age-related diseases with unmet medical needs."

First Quarter 2019 and Recent Clinical, Research and Business Highlights:

Upcoming Resumption of CB4211 Clinical Trial. The company recently announced that it will be resuming its Phase 1a/1b clinical trial of CB4211, its lead MBT candidate under development for the treatment of nonalcoholic steatohepatitis (NASH) and obesity. The company previously announced the suspension of the study in November 2018, to address mild but persistent injection site reactions.

New potential disease targets and mechanisms of action. During the first quarter and more recently, the company continued to advance its optimization and evaluation of novel analogs of mitochondrially encoded peptides with the identification of new disease targets and potential mechanisms of action. Recent studies have provided preliminary data on the evidence of antifibrotic activity of a novel peptide analog in in vitro and in vivo models of lung fibrosis, while another study demonstrated significant progress in exploring the potential for CohBar’s novel peptide analogs to enhance cancer cell killing in the setting of immuno-oncology.

Upcoming Presentation at American Diabetes Association, 79th Scientific Sessions. CohBar will present its recent discovery of the mechanism of action of a family of novel peptides with effects on glucose tolerance in animal models of type 2 diabetes. The mechanism involves interaction with a key cell surface receptor that plays an important role in a number of age-related diseases. We expect that the abstract title and session information will be available to the public on the ADA website on May 10, 2019.

Expansion of Board of Directors. David Greenwood joined the CohBar Board in April 2019. Mr. Greenwood brings to CohBar more than 40 years of financial and operational experience in biotechnology and investment banking. Mr. Greenwood served in leadership roles and on boards at both public and private biotechnology companies including Corium Inc. (formerly NASDAQ: CORI) and Geron Corporation.

Hosted Key Opinion Leader Call, "Mitochondria, a Source for Novel Therapeutics." The company hosted a Key Opinion Leader conference call and webcast on May 2, 2019, featuring world-leading experts and CohBar founders Dr. David Sinclair and Dr. Pinchas Cohen, and CohBar CSO 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 replay of the call is available on the homepage of the CohBar website, (www.cohbar.com).

Expanded IP Portfolio. The company’s IP portfolio recently expanded with the grant of a U.S. patent for its licensed asset MOTS-c. The company also converted its PCT application that describes CB4211 and its analogs into individual patent filings in the U.S. and in multiple international jurisdictions. The applications claim CB4211 composition of matter and methods for using it for the treatment of various diseases.

Continued Investment Community Outreach. In addition to meetings and presentations earlier in the quarter at the JP Morgan Healthcare Conference and at the BIO Investor Forum, company management met with investors at the Roth Conference in March, where interim CEO Philippe Calais also participated in a panel entitled "Why Knowing NASH as a Generalist Can Make You Rich in 2019."
During the first quarter and more recently, CohBar’s founders, Dr. Pinchas Cohen and Dr. Nir Barzilai, continued to be recognized as international leaders in the study of aging, age-related diseases and mitochondrial science.

Dr. Cohen delivered a presentation entitled "Novel Aspects of Mitochondrial Biology and its Implications for Healthy Aging" at the International Conference on Frailty and Sarcopenia Research in Miami Beach, Florida, in February 2019. More recently, in April, Dr. Cohen co-authored an article published in Mitochondrion entitled "Effects of air pollution on mitochondrial function, mitochondrial DNA methylation, and mitochondrial peptide expression."

Dr. Barzilai delivered keynote lectures at Unfolding Aging, in March 2019, in Berlin, Germany, and at the National Medical Research Council, in April 2019, in Singapore. He also lectured about diabetes and aging at the National Medical Research Council, in April 2019, in Singapore, and at the Wellness Summit in March 2019, at Rockefeller University in New York; and delivered the Raining Grant Annual Lectureship at the University of Minnesota in April 2019. During the quarter, Dr. Barzilai co-authored 3 published papers including "Interrogating the Genetic Determinants of Tourette’s Syndrome and Other Tic Disorders Through Genome-Wide Association Studies," in The American Journal of Psychiatry, "APOE Alleles and Extreme Human Longevity," in the Journal of Gerontology, and "Models and Studies of Aging: Executive Summary of a Report from the U13 Conference Series," in The Journal of American Geriatrics.
First Quarter 2019 Financial Highlights

Cash and Investments. CohBar had cash and investments of $19,531,260 on March 31, 2019, compared to $22,182,768 on December 31, 2018.

R&D Expenses. Research and development expenses were $1,371,848 in the three months ended March 31, 2019, compared to $2,680,983 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, and lower stock-based compensation costs, 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 $1,456,197 for the three months ended March 31, 2019, compared to $913,088 in the prior year quarter. The increase in general and administrative expenses was primarily due to stock-based compensation costs, recruiting costs, and increased director fees.

Net Loss. For the three months ended March 31, 2019, net loss was $2,920,584, or $0.07 per basic and diluted share, compared to a net loss of $3,586,585, or $0.09 per basic and diluted share, for the three months ended March 31, 2018.
First Quarter Investor Call and Slide Presentation:

Date: May 7, 2019
Time: 5:00 p.m. EDT (2:00 p.m. PDT)

Conference Audio

Dial-in U.S. and Canada: (800) 289-0438
Dial-in International: (323) 794-2423
Conference ID No.: 1071020
Slide Presentation

Go to www.webex.com, click on the ‘Join’ button and enter meeting number 924 256 010 and Password CWBR, or
Go to www.cohbar.com and click on Q1 2019 Shareholder Presentation at top of homepage.
We kindly request that you please call into the conference audio and log into WebEx approximately 10 minutes prior to the start time so that we can begin promptly.

An audio replay of the call will be available beginning at 8:00 p.m. EDT on May 7, 2019, through 11:59 p.m. EDT on May 28, 2019. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 1071020. The audio replay along with the slide presentation will also be available on the homepage at www.cohbar.com from May 7, 2019 through May 28, 2019.

About CB4211

CohBar’s lead program is based on CB4211, a first-in-class mitochondria based therapeutic (MBT) 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 (MDP) which was discovered in 2012 by CohBar founder Dr. Pinchas Cohen and his academic collaborators and has been shown to play a significant role in the regulation of metabolism. In July 2018, CB4211 entered a Phase 1a/1b clinical trial which includes a potential activity readout relevant to NASH and obesity. In November 2018, the company announced the temporary suspension of the trial to address mild but persistent injection site reactions, and announced the anticipated resumption of the clinical trial in May 2019. NASH has been estimated to affect as many as 12% of adults in the U.S., and there is currently no approved treatment for the disease.