On November 12, 2019 Allakos Inc. (the "Company") (Nasdaq: ALLK), a biotechnology company developing antolimab (AK002) for the treatment of eosinophil and mast cell related diseases, reported financial results for the third quarter ended September 30, 2019 (Press release, Allakos, NOV 12, 2019, View Source [SID1234553324]).

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Third Quarter 2019 Financial Results

Research and development expenses were $16.1 million in the third quarter of 2019 as compared to $8.7 million in the same period in 2018. The increase in research and development expenses was primarily related to an increase in contract research and development activities in support of the advancement of antolimab (AK002), the Company’s lead antibody, as well as an increase in consulting and personnel-related costs.

General and administrative expenses were $7.5 million in the third quarter of 2019 as compared to $3.3 million in the same period in 2018. The increase in general and administrative expenses was primarily attributable to an increase in personnel-related costs as a result of the Company’s increase in employee headcount. Other period-over-period changes included increases to legal costs, accounting and audit service fees, and public company directors and officers liability insurance premiums.

Allakos reported a net loss of $21.7 million in the third quarter of 2019 as compared to $11.1 million in the same period in 2018, an increase of $10.6 million. Net loss per basic and diluted share was $0.47 for the third quarter of 2019 compared to $0.34 in the same period in 2018.

Allakos ended the third quarter of 2019 with $517.0 million in cash, cash equivalents and marketable securities.

On November 12, 2019 NantHealth, Inc. (NASDAQ: NH), a next-generation, evidence-based, personalized healthcare company, reported FDA authorization of Omics Core℠, the first whole exome tumor-normal in vitro diagnostic (IVD) that measures overall tumor mutational burden (TMB) in cancer tissue (Press release, NantHealth, NOV 12, 2019, View Source [SID1234553286]).

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The Omics Core assay is the nation’s first FDA authorized custom-targeted whole exome sequencing platform to report both overall tumor mutation burden in tumor specimens from 19,396 protein-coding genes (whole exome) and somatic alterations (point mutations, small insertions and deletions) in 468 cancer-relevant genes.

TMB is reported via two metrics:

Total number of somatic non-synonymous exonic variants within the 19,396 genes (whole exome) surveyed.
An estimate of mutation rate by counting all somatic, synonymous and non-synonymous variants detected in gene coding regions and dividing by the approximate size of the whole exome.
"Tumor mutation burden (TMB) is now recognized as a key biomarker across multiple tumor types. Studies have shown that immunotherapy treated patients with high TMB had better outcomes compared to those with low TMB. Since the potential for TMB as a precision medicine tool is so high, it is imperative that the most accurate and comprehensive method of analysis be applied to enable physicians to determine which tumors could benefit from checkpoint inhibitors and immunotherapy," said Patrick Soon-Shiong, MD, Chairman & CEO NantHealth. "I am so proud of the scientific, regulatory and bioinformatics teams who have spent almost a decade to perfect this important test that measures the absolute number of mutations occurring in 19,396 protein-coding genes (whole exome) targeting 39 million base pairs (39 Mb) of the human genome from both a tumor and patient-matched normal control sample (tumor-normal). We believe that this comprehensive diagnostic will provide greater accuracy than the widely-used formulaic extrapolation of TMB from a limited gene panel sequence ," said Soon-Shiong.

"Omics Core is the first whole exome test for TMB authorized by the FDA, and as such, marks a watershed moment in oncology. Clinicians can now directly measure the mutations in a patient’s tumor specimen accurately via tumor-normal sequencing and have confidence that the results they receive are fully validated to help support better therapeutic decisions. Also, the breadth of a whole exome means that many more neoepitopes and novel targets may be identified to support vaccine development, novel drug development, and therapies for previously undruggable targets," said Sandeep Bobby Reddy, MD – Chief Medical Officer, NantHealth

"Multiple groups, including our own presentation at ASCO (Free ASCO Whitepaper) in 2018, have shown the importance of performing whole exome sequencing to measure the comprehensive TMB. We calculated a three-fold overestimation of TMB when extrapolating from panel-based methods, potentially leading physicians to over-prescribe checkpoint inhibitors for patients that are unlikely to respond. Given the high cost and the possibility of adverse events with these therapies, it is critical we identify the most appropriate population as accurately as possible," said Steve Benz, PhD – President, Genomics, ImmunityBio.

"Determining the tumor mutational burden from whole exome sequencing is the first step in defining neoepitopes. These unique tumor mutations are recognized by T cells and elicit an immunological anti-tumor response. Patients with high TMB typically have more neoepitopes that attract cancer killing T cells to the tumor microenvironment. Identifying neoepitopes from whole exome TMB enables the development of neoepitope-targeted vaccines for the >95% of protein-encoding genes not covered by limited gene panel tests and the >99% of genes not directly targeted by drugs today. The clearance of Omics Core based upon its analytical performance and validity in reporting TMB establishes a new chapter in the era of precision cancer immunotherapy," said Shahrooz Rabizadeh, PhD – Chief Scientific Officer, ImmunityBio.

About Omics Core

The Omics Core assay is a qualitative in vitro diagnostic test that uses targeted next generation sequencing of formalin-fixed paraffin-embedded tumor tissue matched with normal specimens from patients with solid malignant neoplasms to detect tumor gene alterations in a broad multi gene panel. The test is intended to provide information on somatic mutations (point mutations and small insertions and deletions) and tumor mutational burden (TMB) for use by qualified health care professionals in accordance with professional guidelines, and is not conclusive or prescriptive for labeled use of any specific therapeutic product. Omics Core is a single-site assay performed at NantHealth, Inc. The Omics Core assay is protected by US Patents 9,652,587; 9,646,134; 9,824,181; 10,249,384; 9,721,062; 10,242,155; 10,268,800.

On November 12, 2019 Intrexon Presented the corporate Presentation (Filing, 8-K, Intrexon, NOV 12, 2019, View Source [SID1234552292]).

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On November 12, 2019 Morphic Therapeutic (NASDAQ: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and financial results for the third quarter of 2019 (Press release, Morphic Therapeutic, NOV 12, 2019, View Source [SID1234551238]).

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"Morphic began the third quarter of 2019 with a successful IPO, providing significant financial resources to fund the discovery and development of our portfolio of small-molecule integrin therapeutics," commented Praveen Tipirneni, M.D., president and chief executive officer of Morphic Therapeutic. "As we look forward, we now believe that we will submit the IND for MORF-720, our oral αvβ6 inhibitor for patients with fibrotic disease being developed in collaboration with AbbVie, in the second half of 2020, based on the request for an additional preclinical study by the FDA. In Morphic’s wholly-owned portfolio, we remain on track to file an IND for our oral inhibitor of α4β7 for patients with inflammatory bowel disease in mid-2020. Further, Morphic continues to refine and apply the MInT platform, a suite of technologies designed to streamline rapid and effective discovery of drug candidates across the entire integrin target class, where we have deep expertise."

Third Quarter Highlights and Program Updates

Oral Integrin Development Program Updates

Oral αvβ6 Inhibition Program for Fibrotic Diseases

Morphic now believes that the IND submission for MORF-720, a selective oral αvβ6-specific integrin inhibitor will take place in the second half of 2020. Morphic has made this adjustment based on the request for an additional preclinical toxicology study received during pre-IND interactions with the FDA. Morphic’s αvβ6 inhibitors remain subject to an exclusive license option pursuant to Morphic’s collaboration agreement with AbbVie.

Oral α4β7 Inhibition Program for Irritable Bowel Disease (IBD)

Morphic remains on track to submit the IND for its wholly owned oral inhibitor of α4β7 for the treatment of IBD in mid-2020 and begin clinical studies in the second half of 2020.

Morphic’s development portfolio is targeted to integrin receptors that are strongly implicated in diseases with validated clinical need. The company’s lead programs are inhibitors of the integrins αvβ6 and α4β7, which Morphic’s data suggests could be the basis of transformational treatments for patients with fibrotic diseases and inflammatory bowel disease (IBD), respectively. Fibrosis is a common feature of many chronic diseases involving tissue injury and can lead to the progressive failure of multiple organs, such as the lungs, liver, skin, and kidney. IBD is a group of chronic autoimmune and inflammatory conditions of the gastrointestinal tract that include ulcerative colitis and Crohn’s disease, among others.

Financial Highlights

Morphic’s initial public offering (IPO) of common stock on The Nasdaq Global Market, including full exercise of the underwriters’ option to purchase additional shares of common stock, closed on July 1, 2019. The IPO generated gross proceeds of approximately $103.5 million, before deducting underwriting discounts and commissions and other estimated offering expenses.

Financial Results for Third Quarter 2019:

Net loss for the quarter ended September 30, 2019 was $8.9 million or $0.30 per share compared to a net loss of $7.1 million or $7.00 per unit (units were converted to shares as a result of converting from a limited liability company to a corporation prior to the IPO), for the same quarter last year).

Revenue was $5.7 million for the quarter ended September 30, 2019 compared to $0 for the same quarter last year. The increase was due to collaboration agreements signed by AbbVie in October 2018 and Janssen in February 2019

Research and development expenses were $12.6 million for the quarter ended September 30, 2019, compared to $5.8 million in the same quarter last year. The $6.8 million increase year-over-year reflects higher development and manufacturing costs associated with our product candidate, MORF-720; research costs associated with preclinical studies; as well as increased personnel-related costs to support continued progress with the company’s pipeline.

General and administrative expenses were $2.9 million for the quarter ended September 30, 2019, compared to $1.4 million in the same quarter last year. The $1.5 million increase year-over-year was primarily attributable to increased headcount and higher professional and consulting fees associated with ongoing business activities and Morphic’s operating as a public company.

As of September 30, 2019, Morphic had cash, cash equivalents, and marketable securities of $251.7 million, compared to $185.9 million as of December 31, 2018. Morphic believes its cash, cash equivalents, and marketable securities as of September 30, 2019, will be sufficient to fund operating expenses and capital expenditure requirements at least through the end of 2022.

On November 16, 2019 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported that it will be presenting at the Stifel 2019 Healthcare Conference on Tuesday, November 19, 2019 at 3:00 PM ET / 12:00 PM PT at the Lotte New York Palace Hotel (Press release, Savara, NOV 12, 2019, View Source [SID1234551162]). Rob Neville, Savara’s Chief Executive Officer, will represent the Company at the conference.

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Interested parties can access a live audio webcast on the Investors page of the Savara website at www.savarapharma.com/investors/events-presentations/. Archived presentations will be available on Savara’s website for 90 days following the events.