On November 22, 2019 Celularity, Inc. ("Celularity" ), a clinical-stage cell therapeutics company focused on the development of innovative allogeneic cellular therapies from human placentas, reported pre-clinical data supporting the advancement of CYNK-001, a placental-derived, cryopreserved natural killer (NK) cell therapy, as a clinical candidate for patients with glioblastoma multiforme (GBM) in a poster presentation at the 2019 Society for Neuro-Oncology (SNO) Annual Meeting in Phoenix, A.Z (Press release, Celularity, NOV 22, 2019, View Source [SID1234551621]).

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Results from pre-clinical studies demonstrated in vivo anti-GBM activity of CYNK-001. "The positive results from these pre-clinical studies highlight the versatility and activity of our investigational, off-the-shelf, placental-derived NK cell therapy program," said Robert Hariri, M.D., Ph.D., Founder, Chairman and CEO at Celularity. "There is a clear need for novel therapeutic approaches to the treatment of glioblastoma multiforme. We look forward to advancing our investigational programs and are optimistic about the possibility of bringing another therapeutic option to patients in need."

About CYNK-001
CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. When derived from the placenta, these cells offer intrinsic safety and versatility, allowing potential use across a range of organs and tissues. CYNK-001 is currently being investigated as a treatment for acute myeloid leukemia (AML), multiple myeloma (MM), and as a potential treatment option for various solid tumors.

On November 22, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) and Spark Therapeutics, Inc. (NASDAQ: ONCE) ("Spark") reported that Roche has extended the offering period of its previously announced tender offer to purchase all of the outstanding shares of common stock (the "Shares") of Spark for USD 114.50 per Share, net to the seller thereof in cash, without interest and subject to any withholding taxes required by applicable law and upon the terms and subject to the conditions set forth in the Offer to Purchase dated March 7, 2019 (as it may be amended and supplemented from time to time, the "Offer") (Press release, Hoffmann-La Roche, NOV 22, 2019, View Source [SID1234551605]).

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Pursuant to the Agreement and Plan of Merger, dated as of February 22, 2019, by and among Roche Holdings, Inc., 022019 Merger Subsidiary, Inc., and Spark (as amended, the "Merger Agreement"), the Offer, which was previously scheduled to expire at 5:00 p.m., New York City time, on Monday, November 25, 2019, has been extended until 5:00 p.m., New York City time, on Tuesday, December 10, 2019, unless it is extended further under the circumstances set forth in the Merger Agreement. All terms and conditions of the Offer shall remain unchanged during the extended period.

The Offer was extended to provide additional time for the U.S. Federal Trade Commission (the "FTC") and the UK Competition and Markets Authority (the "CMA") to complete their previously disclosed reviews of Roche’s pending acquisition of Spark. The parties remain committed to the transaction and are working cooperatively and expeditiously with the FTC and the CMA.

Citibank, N.A., the depository for the Offer, has advised Roche that, as of 5:00 p.m., New York City time, on November 21, 2019, approximately 5,717,437 Shares of Spark (none of which were tendered by notice of guaranteed delivery) had been validly tendered and received, and not validly withdrawn, pursuant to the Offer, representing approximately 14.8% of Spark’s outstanding Shares. Stockholders who have already tendered their Shares of Spark do not have to re-tender their Shares or take any other action as a result of the extension of the expiration date of the Offer.

Closing of the tender offer is conditioned upon customary closing conditions, including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, and there being validly tendered and received, and not validly withdrawn, a majority of the outstanding Spark Shares.

MacKenzie Partners, Inc. is acting as information agent for the Offer. Requests for documents and questions regarding the Offer may be directed to MacKenzie Partners, Inc. by telephone, toll-free at (800) 322-2885 (please call (212) 929-5500 (collect) if you are located outside the US or Canada) or via email at [email protected].

On November 22, 2019 Aurora Bio, Inc. reported that data for the company’s novel PET radiotracer, AUR01, in development for systemic amyloidosis, has been accepted for presentation at the upcoming 61st American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, to be held December 7 – 10, 2019, in Orlando, FL (Press release, Aurora Biosciences, NOV 22, 2019, View Source [SID1234551622]). Abstract highlights and presentation details are outlined below.

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Spencer Guthrie, Chief Executive Officer, stated, "We are looking forward to an exciting ASH (Free ASH Whitepaper) conference as we present our initial data highlighting the unique ability of AUR01 to detect and potentially monitor whole body amyloid burden in systemic amyloid diseases. The unmet need for early and accurate diagnosis for systemic amyloidosis is significant as many patients are getting diagnosed too late to obtain optimal benefit from treatment. There is currently no approved diagnostic, no agent that can detect whole body amyloid burden and no method for directly measuring change in amyloid pathology in treated patients. We believe this compound has the potential to change the way systemic amyloidosis is diagnosed and monitored and holds great potential to provide for efficient drug development for new targeted therapeutics, including Aurora Bio’s own therapeutic programs."

Abstract Highlights: UPDATED RESULTS TO BE PRESENTED AT CONFERENCE
Poster Presentation: View Source

AUR01, a synthetic peptide radiotracer (124I-p5+14), has been shown in preclinical assays and in SPECT and PET imaging of murine systemic amyloidosis, to bind many forms of amyloid, including AL, ATTR, and ALECT2
No radiotracers approved in the US for non-invasive quantitative measurement of systemic amyloid disease
Ph 1 study Included AL, ATTR and ALECT2 patients with biopsy proven amyloidosis and adequate renal function
The primary endpoint includes safety and dosimetry estimation with a secondary endpoint of efficacy
10 patients have been dosed and evaluated. No serious adverse events of any grade were noted
Retention in the heart, kidneys, liver, spleen, pancreas, bone marrow, lung, and adrenal gland of AL patients
Cardiac uptake of the radiotracer was observed in 80% of AL patients and 100% of ATTR patients
Kidney, spleen and liver retention was observed in 60%, 40%, and 20% of AL patients
Retention of the radiotracer observed in the nerves, spleen and kidney in patients with ATTR and ALECT2
Conclusion: Initial PET/CT image data indicate that 124I-p5+14 can provide quantitative detection of systemic amyloidosis in multiple organ systems and may have general utility in detecting and monitoring amyloid burden in many forms of amyloidosis
Presentation Details

3034 Preliminary Phase 1 Data on the Safety and Efficacy of a Novel PET Radiotracer, 124I-p5+14, for Imaging Systemic Amyloidosis

Program: Oral and Poster Abstracts
Session: 641. CLL: Biology and Pathophysiology, excluding Therapy: Poster II
Sunday, December 8, 2019, 6:00 PM-8:00 PM
Hall B, Level 2 (Orange County Convention Center)
Presenter Jonathan S. Wall, PhD, Head of Cancer and Amyloidosis Theranostics Program, University of Tennessee

On November 22, 2019 Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on harnessing the body’s immune system in the fight against cancer, reported a collaboration with Urology Centers of Alabama (UCA) in the Cchek early prostate cancer detection study (Press release, Anixa Biosciences, NOV 22, 2019, View Source [SID1234551607]). UCA will provide patient samples in Anixa’s ongoing study, including support of clinical validation of Anixa’s Cchek Prostate Cancer Confirmation test.

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UCA is one of several sites across the U.S. participating in this study. Dr. Charles Edward Bugg, Jr. will serve as the Principal Investigator at UCA.

Amit Kumar, Ph.D., Chief Executive Officer of Anixa, stated, "We are pleased to have another highly regarded urology practice join our team of collaboration partners. We look forward to UCA’s participation as we continue the clinical validation phase of the Cchek Prostate Cancer Confirmation test in preparation for commercial launch later this year."

About Cchek
Cchek is an early cancer detection technology, that measures a patient’s immunological response to a malignancy by analyzing immune system cells in peripheral blood. The goal is to utilize the technology to determine a patient’s cancer status from a simple blood draw, eliminating the need for a biopsy, which can be an expensive, painful and invasive procedure. Further, conventional methods using current cancer screening tests often lack accuracy and reliability. Anixa’s orthogonal approach using flow cytometry coupled with artificial intelligence provides an alternative method that offers improved affordability, efficacy and efficiency. To date, Anixa has successfully used Cchek to detect the presence of 20 different cancers including lung, colon, breast and prostate. The robust cancer detection performance of Cchek makes it a platform from which multiple cancer diagnostic tests may be developed. The first such test, a prostate cancer confirmation test, is slated for commercial launch by the end of 2019.

Urology Centers of Alabama
For over 60 years, Urology Centers of Alabama has been committed to providing the highest quality medical services in a compassionate and caring environment. With 24 skilled urologists, two urogynecologists, two radiation oncologist, one medical oncologist, one pathologist, a team of physicians’ assistants and nurse practitioners and over 160 employees, the practice has earned a well deserved reputation for excellence.

On November 22, 2019 City of Hope reported that persons who inherit Li-Fraumeni syndrome (LFS) have an extremely increased risk of developing cancer (Press release, City of Hope, NOV 22, 2019, View Source [SID1234551623]). For instance, females with the syndrome have a 50% chance of developing cancer by the age of 30 and a 90% chance over their lifetime; males with the syndrome have a 70% chance of cancer over their lifetime.

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LFS was first described 50 years ago, and 30 years ago the syndrome was linked to inheriting a mutation in the TP53 gene. City of Hope scientists and others worldwide have made advances in identifying and treating people with the syndrome, but more work remains to be done.

A new $8.5 million, five-year grant from the National Institutes of Health seeks to advance research and treatment for this serious syndrome, which can cause multiple cancers, including sarcomas, brain and breast tumors, and adrenocortical cancers.

"This is a devastating syndrome to have – we’ve cared for families with babies who had brain tumors at 18 months," said Jeffrey Weitzel, M.D., director of City of Hope’s Division of Clinical Cancer Genomics, the Dr. Norman & Melinda Payson Professor in Medical Oncology and the grant’s co-principal investigator. "City of Hope and our partner institutions are determined to develop a better understanding of the cancer risks associated with different TP53 mutations so we can better tailor screening, prevention and treatment for these patients. We are seeing the light at the end of the tunnel but more research is needed."

The project, termed LiFTUP (Li Fraumeni and TP53 Understanding and Progress), which is co-led by Judy E. Garber, M.D., M.P.H., of Dana-Farber Cancer Institute and Christopher I. Amos, Ph.D., of Baylor College of Medicine, will provide the largest and most comprehensive examination of TP53-associated cancer risk.

Traditionally, clinical TP53 testing was limited to individuals and families who met specific criteria (typically multiple cancers at very young ages). With the introduction of next-generation-based multigene panel testing, which sequences a person’s genome faster and is less costly than previous genetic tests, TP53 testing is now performed on large numbers of people who do not meet the syndrome’s criteria. However, the testing has also raised concerns about false positives, or a test result that incorrectly indicates the syndrome is present.

That’s because blood samples are often used for genetic testing and people may develop TP53 gene mutations in rapidly growing blood cells as they age. Researchers will also examine this group of people to find out why some people in this group develop blood cancers while others do not.

The inherited syndrome is rare. Only about 1,000 children and adults nationwide are included in the Li-Fraumeni Exploration Consortium, and researchers working on this project will also recruit TP53 carriers identified through broader, more agnostic approaches to testing, including commercial genetic testing laboratories, the Geisinger MyCode project, the PROMPT study of individuals with germline mutations and the ORIEN tumor/germline sequencing project.

"Carriers of true germline TP53 mutations may bear the psychological, medical and financial costs of striking personal and family cancer risks, the burden of intensive surveillance, the high risks of cancer deaths at disproportionately young ages and the weight of possibly passing TP53 variants to offspring," according to the team.

Consequently, a crucial question researchers want to answer is why some people with a TP53 mutation develop cancer while others do not.

"We’re looking at thousands of markers across the whole genome to find out if there are patterns that influence why two TP53 carriers with the same mutation have different outcomes," Weitzel added. "We will do a clinical and molecular interrogation of the factors that influence the development of cancer."

"Our hope is that by focusing more research on children and adults with the TP53 mutation, we will be able to take better care of these individuals," Weitzel said.