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Magenta Therapeutics Reports Third Quarter 2019 Financial Results and Recent Business Highlights

On November 13, 2019 Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, reported financial results for the third quarter ended September 30, 2019 and recent business highlights (Press release, Magenta Therapeutics, NOV 13, 2019, View Source [SID1234551122]).

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"Magenta has a big vision: to bring the curative power of immune reset through stem cell transplant to patients with autoimmune diseases, genetic diseases and blood cancers. At ASH (Free ASH Whitepaper) next month, we are looking forward to sharing important new data from across our portfolio. These will include clinical data from our MGTA-145 program, which we are developing as a new first-line standard of care for stem cell mobilization. We will also present the first gene therapy data on our CD117-ADC, which we are developing as a non-genotoxic preparation regimen for gene therapy and transplant," said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. "In addition, earlier this week we presented our first data in autoimmune disease, showing that a single dose of our CD45-ADC led to successful immune reset in disease models of multiple sclerosis, systemic sclerosis and inflammatory arthritis. Immune reset through stem cell transplant has previously demonstrated durable remissions in thousands of patients with autoimmune diseases such as multiple sclerosis and systemic sclerosis, and it is recommended in multiple guidelines in US and Europe."

Recent Business Highlights:

First clinical data for MGTA-145 stem cell mobilization program and groundbreaking CD117-ADC gene therapy data to be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting: On November 6th, 2019, Magenta announced that the Company will share results from six abstracts at ASH (Free ASH Whitepaper) in December 2019, covering Magenta’s patient preparation, mobilization and cell therapy programs. The Company will present clinical data for the MGTA-145 stem cell mobilization program, building on the positive early data in the ASH (Free ASH Whitepaper) abstract. Magenta is developing MGTA-145 to be the new standard of care for first-line stem cell mobilization, with mobilization and collection taking place in a single day without the use of G-CSF, the current standard of care. The Company will also highlight important proof-of-concept data from the CD117-ADC patient preparation program. In a rhesus model of gene therapy, a single dose of CD117-ADC enabled engraftment of stem cells modified with the β-globin gene, the gene that causes sickle cell disease and β-thalassemia when mutated. These results showed for the first time that a single dose of CD117-ADC can enable successful gene therapy transplant in non-human primates without the need for chemotherapy or radiation.

First Magenta preclinical immune reset data presented at the American College of Rheumatology (ACR) annual meeting: On November 10th, 2019, Magenta presented the first data on the use of targeted antibody-drug conjugates (ADCs) to reset the immune system and halt progression of autoimmune disease. Results showed that a single dose of C45-ADC removed disease-causing cells, enabled successful reset and rebuild of the immune system and was well tolerated in models of multiple sclerosis, systemic sclerosis and inflammatory arthritis. Further, a single dose of CD45-ADC significantly delayed disease onset in a model of multiple sclerosis that has successfully provided preclinical proof of concept for many clinically validated standard of care therapies.

Exercised option with Heidelberg Pharma for ADCs using an amanitin payload and targeting CD45: On November 11th, 2019, Magenta announced that it had exercised its option with Heidelberg Pharma for exclusive worldwide development and marketing rights for ADCs using an amanitin payload and targeting CD45.

Received Regenerative Medicine Advanced Therapies (RMAT) Designation for MGTA-456 in inherited metabolic disorders: In September 2019, Magenta announced that the FDA granted RMAT designation for MGTA-456, a one-time cell therapy for the treatment of multiple inherited metabolic disorders, based on encouraging clinical data generated to date in the Company’s ongoing Phase II trial. RMAT designation is a dedicated program designed to expedite the development and approval processes for promising pipeline products, including cell therapies. Potential advantages of the RMAT designation include all of the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA that may be used to discuss potential surrogate or intermediate endpoints to support accelerated approval.

Appointed Senior Vice President, Head of Translational Sciences; Chief Scientific Officer to transition out of Company: In October 2019, Magenta announced that it had hired Jan Pinkas, Ph.D., as Senior Vice President, Head of Translational Sciences. Dr. Pinkas is a seasoned scientist with deep expertise in leading drug development programs, including ADCs. Prior to joining Magenta, he was Head of Translational Research & Development at ImmunoGen, Inc., where he led nonclinical and translational research and development-related activities for all programs in discovery through late-stage clinical development. The Company also announced that Mike Cooke, Ph.D., Chief Scientific Officer, will leave Magenta to pursue other opportunities.

Financial Results:

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2019, were $160.6 million compared to $142.6 million on December 31, 2018. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the second half of 2021.

Research and Development Expenses: Research and development expenses were $16.5 million in the third quarter of 2019, compared to $11.4 million in the third quarter of 2018. The increase was driven primarily by investments in clinical activities for MGTA-145, as well as manufacturing related to our patient preparation programs.

General and Administrative Expenses: General and administrative expenses were $6.1 million for the third quarter of 2019, compared to $5.3 million for the third quarter of 2018. The increase was primarily due to an increase in professional fees and facility costs associated with the growth of the Company.

Net Loss: Net loss was $ 21.0 million for the third quarter of 2019, compared to net loss of $16.0 million for the third quarter of 2018.

Allakos Reports Third Quarter 2019 Financial Results

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]).

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.

NantHealth Announces FDA Marketing Authorization of Omics Core?: The Nation’s First Tumor-Normal Mutation Profiling of Overall Tumor Mutational Burden from Whole Exome Sequencing in Solid Tumors

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]).

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.

Corporate Presentation

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

Morphic Announces Corporate Highlights and Third Quarter 2019 Financial Results

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]).

"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.