On April 1, 2019 AstraZeneca and MSD, Inc., Kenilworth, NJ, US (MSD: known as Merck & Co., Inc. inside the US and Canada) reported that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for the MEK 1/2 inhibitor and potential new medicine selumetinib (Press release, AstraZeneca, APR 1, 2019, View Source [SID1234534817]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

This designation is for the treatment of paediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN), a rare, incurable genetic condition.

José Baselga, Executive Vice President, Research and Development, Oncology, said: "Selumetinib shows promise in the treatment of NF1-related plexiform neurofibromas, a rare and debilitating disease with no approved medications to date. The Breakthrough Therapy Designation acknowledges the significant unmet need of these patients and the potential benefit of selumetinib in this setting."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, at MSD Research Laboratories, said: "This new designation validates our ongoing development of selumetinib. As a result of this, selumetinib has the potential to receive expedited regulatory review and we hope to bring this medicine to patients as soon as possible."

The BTD is based on Phase II data from the SPRINT trial, testing selumetinib as an oral monotherapy in paediatric patients, aged three years or older with inoperable NF1-related PN. The results of the trial were presented by the National Cancer Institute (NCI) at the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

This is the ninth BTD that AstraZeneca has received from the FDA since 2014. BTD is designed to expedite the development and regulatory review of medicines that are intended to treat a serious condition and that have shown encouraging early clinical results, which may demonstrate substantial improvement on a clinically-significant endpoint over available medicines.

Selumetinib was granted Orphan Drug Designation for the treatment of NF1 by the US FDA in February 2018 and the European Medicines Agency in August 2018.

Selumetinib is a MEK 1/2 inhibitor and potential new medicine licensed by AstraZeneca from Array BioPharma Inc. in 2003. AstraZeneca and MSD entered a co-development and co-commercialisation agreement for selumetinib in 2017.

The NF1 gene provides instructions for making a protein called neurofibromin, which negatively regulates the RAS/MAPK pathway, helping to control cell growth, differentiation and survival. Mutations in the NF1 gene may result in dysregulations in RAS/RAF/MEK/ERK signalling, which can cause cells to grow, divide and copy themselves in an uncontrolled manner, and may result in tumour growth. Selumetinib inhibits the MEK enzyme in this pathway, potentially leading to inhibition of tumour growth.

Selumetinib is being assessed as a monotherapy and in combination with other treatments in ongoing trials.

About SPRINT

The SPRINT trial is a US Cancer Therapy Evaluation Program (CTEP) NCI-sponsored Phase I/II trial. The Phase I trial was designed to identify the optimal Phase II dosing regimen, and the results were published in the New England Journal of Medicine.1

About NF1

Neurofibromatosis type 1 (NF1) is an incurable genetic condition that affects one in 3,000 to 4,000 individuals.2,3 It is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas), skin pigmentation (so-called ‘cafe au lait’ spots) and, in 20-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas). These plexiform neurofibromas can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement as well as having the potential to transform into malignant peripheral nerve sheath tumours (MPNST).

People with NF1 may experience a number of complications such as learning difficulties, visual impairment, twisting and curvature of the spine, high blood pressure, and epilepsy. NF1 also increases a person’s risk of developing other cancers, including malignant brain tumours, MPNST and leukaemia. Symptoms begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.4

About the AstraZeneca and MSD Strategic Oncology Collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza (olaparib), the world’s first PARP inhibitor and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, we are committed to advance New Oncology as one of AstraZeneca’s five Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On April 1, 2019 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported that the company will present at the H.C. Wainwright Global Life Sciences Conference in London on Monday, April 8, 2019 at 4:30 a.m. ET and the Needham Healthcare Conference in New York on Tuesday, April 9, 2019 at 2:50 p.m. ET (Press release, BioCryst Pharmaceuticals, APR 1, 2019, View Source [SID1234534816]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Links to a live audio webcast and replay of these presentations may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com.

On April 1, 2019 Sutro Biopharma, Inc. (NASDAQ: STRO), a clinical-stage drug discovery, development and manufacturing company focusedon the application of precise protein engineering and rational design to create next-generation oncology therapeutics, reported its financial results for the year ended December 31, 2018 (Press release, Sutro Biopharma, APR 1, 2019, View Source [SID1234534813]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"This last year was a landmark one for Sutro. Our initial clinical trial for our first internal program, STRO-001 for the treatment of patients with multiple myeloma and non-Hodgkin’slymphoma, commenced in April 2018. Importantly, we also advanced our second clinical program, STRO-002 for the treatment of ovarian and endometrial cancers, to its clinical trial initiation in March 2019. On the business front, we entered a significant collaboration with Merck and completed our IPO in the second half of 2018," said Bill Newell, Sutro’s Chief Executive Officer. "In 2019, we look forward to the continued advancement of our internal programs, while working with our partners on progressing the collaboration product candidates."

Recent Business Highlights and Developments

STRO-001 Clinical Program

Potential first-in-class and best-in-class antibody-drug conjugate (ADC) directed against CD74, which is highly expressed in many B cell malignancies
Phase1 dose-escalation, with dose expansion, clinical trial enrolling patients with multiple myeloma and non-Hodgkin’slymphoma, with initial safety data expected to be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress in June 2019 and initial efficacy data expected by year end 2019
STRO-001 granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma
STRO-002 Clinical Program

Potential best-in-class ADC directed against folate receptor-alpha, which is highly expressed in ovarian cancer
Phase1 dose-escalation, with dose expansion, clinical trial enrolling women with advanced ovarian and endometrial cancers, with initial safety data expected by year end 2019
Corporate Highlights

Collaboration and licensing agreement with Merck signed in July 2018 to discover and develop novel immune-modulating therapies for cancer and autoimmune disorders
Initial public offering (IPO) that closed on October 1, 2018, provided Sutro with gross proceeds of $85.0 million, before deducting underwriting discounts and commissions and offering expenses. Additionally, Sutro received proceeds of $10.0 million from Merck in a private placement of common stock concurrent with the IPO
Full Year 2018 Financial Highlights

Cash, Cash Equivalents and Marketable Securities

As of December 31, 2018, Sutro had cash, cash equivalents and marketable securities of $204.5 million.

Revenue

Revenue was $38.4 million for the year ended December 31, 2018, which included collaboration revenue of $32.4 million recognized primarily from Celgene, Merck andEMD Serono, in addition to other revenue of $6.0 million. During the third and fourth quarters of 2018, Sutro began recording revenue from Merck, primarily from the $60.0 million upfront payment received by Sutro under the July 2018 collaboration and licensing agreement, which revenue will be recognized over multiple years. Future collaboration revenue from Celgene, Merck and EMD Serono, and from any future collaboration partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones and other collaboration agreement payments.

Operating Expenses

Total operating expenses for the year ended December 31, 2018, were $75.6 million compared with $71.0 million for the same period in 2017, including non-cash stock-based compensation of $2.9 million and $1.4 million, and depreciation and amortization expense of $4.5 million and $5.0 million, in the year 2018 and 2017, respectively. Total operating expenses for the year 2018 were comprised of research and development expenses of $54.3 million and general and administrative expenses of $21.4 million, with both expense types expected to increase in 2019 as Sutro’s internal product candidates advance in clinical development and additional general and administrative expenses are incurred as a public company following its IPO that closed on October 1, 2018.

Net Loss Per Share Calculation

Sutro financial statements following September 30, 2018, including share and per share amounts, give effect to common stock shares issued in the IPO and the Merck concurrent private placement, and common stock from the conversions of Sutro’s previously outstanding redeemable convertible preferred stock, as these transactions were completed on October 1, 2018.

On April 1, 2019 LabCorp (NYSE: LH) reported that it will release its first quarter of 2019 financial results before the market opens on Tuesday, April 30, 2019, followed by a conference call and webcast beginning at 9:00 a.m. EDT to discuss the results (Press release, LabCorp, APR 1, 2019, View Source;p=RssLanding&cat=news&id=2392950 [SID1234534812]). Interested parties can access the conference call by dialing 844-634-1444 within the U.S. and Canada, or 1-615-247-0253 internationally, using the passcode 7459328.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

An audio replay of the conference call will be available from 1:00 p.m. EDT on April 30, 2019, until 11:30 a.m. EDT on May 14, 2019, by dialing 855-859-2056 within the U.S. and Canada, or 1-404-537-3406 internationally, using the passcode 7459328.

The earnings release, accompanying financial information, and a real-time webcast of the conference call will be available on the LabCorp Investor Relations website. The webcast will be archived and accessible through April 24, 2020.

On April 1, 2019 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing novel cancer immunotherapies, reported that it has received Advanced Therapy Medicinal Product (ATMP) classification for the Company’s lead investigational product candidate, TAVO (DNA plasmid vector expressing IL-12 gene), as a potential treatment for refractory metastatic melanoma from the European Medicines Agency (EMA) (Press release, OncoSec Medical, APR 1, 2019, View Source [SID1234534811]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Only innovative investigational product candidates that are based on gene, tissue or cell therapy are qualified for classification as advanced therapy medicinal products. The classification of TAVO as an ATMP is a significant step toward its potential accelerated approval in Europe. The ATMP classification qualifies TAVO to take advantage of a specific EMA regulatory framework designed to facilitate the accelerated review, approval, and access to innovative products in the European market. Further, OncoSec intends to avail itself of the numerous benefits and incentives for medicinal products undergoing clinical testing and marketing approval processes in Europe. Such incentives include fee reductions and exemptions in pre- and post-marketing authorization phases; administrative, procedural and scientific advice and eligibility for funding.

"The ATMP designation confirms TAVO as an innovative gene therapy potentially offering a groundbreaking new opportunity for the treatment of refractory metastatic melanoma," said Robert Ashworth, PhD, Senior Vice President of Regulatory and Quality at OncoSec.

"Similar to TAVO’s existing FDA Fast Track Designation in the United States, the ATMP designation allows OncoSec to take advantage of a specific European regulatory framework designed to facilitate the accelerated review and approval of TAVO in the European market," said Daniel J. O’Connor, OncoSec’s President and CEO. "Obtaining this important designation delivers on one of OncoSec’s key objectives for 2019."

The Committee for Advanced Therapies (CAT) at the EMA is responsible for classifying and assessing the quality, safety and efficacy of products designated as ATMP. The CAT is a multidisciplinary committee of the best available experts in Europe. The main responsibility of the CAT is to prepare a draft opinion on each ATMP application submitted to the EMA before the Committee for Medicinal Products for Human Use (CHMP) adopts a final opinion on granting a marketing authorization for the product. During drug development, CAT also reviews and certifies the acceptability of quality and non-clinical data.

In February 2017, the U.S. Food and Drug Administration (FDA) designated the investigation of OncoSec’s TAVO in combination with KEYTRUDA (pembrolizumab) to stop or cause the regression of the tumor of patients with Stage III/IV melanoma who are progressing on either KEYTRUDA (pembrolizumab) or OPDIVO (nivolumab) treatment as a Fast Track Development Program. Fast Track designation is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. A drug that receives Fast Track designation is eligible for Accelerated Approval if relevant criteria are met.