On June 22, 2020 A new program called PATRIOT, developed by the Translational Genomics Research Institute (TGen), an affiliate of City of Hope,reported that it is using organoids — laboratory cultures derived from samples of patient tumors — to provide a whole new level of accuracy in prescribing anti-cancer treatments (Press release, TGen, JUN 22, 2020, View Source [SID1234561511]).

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PATRIOT builds on other precision medicine programs devised by Ashion Analytics, a TGen clinical laboratory, which uses its GEM ExTra proprietary test to match each patient’s unique cancer to the best available cancer treatments.

PATRIOT, which stands for PAThway based RNA and DNA Integration with tumor Organoid Testing for clinical therapeutics, will be showcased in a study presentation June 22-24 at the second 2020 virtual annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper).

"Cancer tumors are complicated," said Dr. Sunil Sharma, Deputy Director of TGen Clinical Sciences and Chief of Translational Oncology and Drug Development at the HonorHealth Research Institute. "PATRIOT is a very powerful platform that will make GEM ExTra even more powerful. This will expand the use of RNA analysis in a way that has never been used before."

In this system, organoids — which can mimic the reactions of solid tumors in patients’ bodies —are grown in a laboratory and then used to test different anti-cancer therapies.

"It’s a way of conducting clinical trials on a laboratory plate," said Dr. Sharma, who also is a Professor and Director of TGen’s Applied Cancer Research and Drug Discovery Division.

The study being presented at AACR (Free AACR Whitepaper) shows how Dr. Sharma’s TGen lab, using melanoma tumor samples provided by HonorHealth, used the new PATRIOT system to identify potential therapeutic targets by focusing on molecular pathways within tumor cells, a level of analysis that goes beyond searching for mutations in DNA, and even builds on top of the intricate analysis of RNA-expression provided by Ashion’s GEM ExTra.

"These druggable targets were validated on the tumor organoids," said Dr. Sharma, who hails the system as a whole new way to provide therapeutic benefit to patients. "This allows for a holistic assessment of a patient’s tumor for improving therapy recommendations and expanding personalized therapy options."

In addition, he said, PATRIOT analysis of organoids gives investigators the ability to test immunotherapy options in the laboratory.

Ashion’s GEM ExTra platform already has expanded the therapeutic potential of genomic sequencing by using RNA sequencing to identify novel fusions and alternate transcripts, providing additional tumor profiling data in addition to that identified by DNA sequencing.

Unlike many other genomic sequencing tests, which use panels of dozens or even hundreds of known cancer-causing genomic variants, Ashion’s GEM ExTra screens cancer patients for all of the nearly 3 billion nucleotides, or letters, in human DNA, which includes more than 19,000 genes.

The next step, Dr. Sharma said, is to test the predictions from PATRIOT and GEM ExTra analysis of patient organoids in the laboratory to see if they might work in a larger clinical trial.

This study was supported by funding from Flinn Foundation grant #2193.

On June 22, 2020 Spectrum Pharmaceuticals, Inc. (NASDAQ-GS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported preclinical data evaluating the administration of ROLONTIS (eflapegrastim) on the same day as chemotherapy (Press release, Spectrum Pharmaceuticals, JUN 22, 2020, View Source [SID1234561283]). The duration of neutropenia in a rat model of chemotherapy induced neutropenia (CIN) was observed to be significantly shorter with eflapegrastim versus pegfilgrastim, regardless of the timing of administration: concomitantly or on the same day post-chemotherapy at 2, 5 or 24 hours post-chemotherapy.

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The poster titled "Chemotherapy induced neutropenia in rats following administration of eflapegrastim or pegfilgrastim on the same day at three different time points and at 24 hours post-chemotherapy" was part of today’s Special Populations, Survivorship, Outcomes, and Supportive Care session at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II. ROLONTIS is an investigational drug not approved by the U.S. Food and Drug Administration (FDA) and the BLA (Biologics License Application) is currently under active review by the agency for the treatment of chemotherapy induced neutropenia with a PDUFA date of October 24, 2020.

"These data in rats suggest that eflapegrastim administered at the time of chemotherapy, rather than waiting 24 hours, may be able to provide prophylaxis against chemotherapy induced neutropenia," said Francois Lebel, M.D., Chief Medical Officer of Spectrum Pharmaceuticals. "Based on these preclinical results, we recently initiated a Phase 1 clinical trial to compare the effect of same day dosing of eflapegrastim on the duration of neutropenia in patients with early-stage breast cancer when administered on the same day, at varying intervals following docetaxel and cyclophosphamide chemotherapy."

On June 22, 2020 Helix BioPharma Corp. (TSX: HBP) ("Helix" or the "Company"), an immuno-oncology
company developing innovative drug candidates for the prevention and treatment of cancer, reported that it will be presenting at Proactive One2One Virtual Investor Forum on June 23, 2020 (Press release, Helix BioPharma, JUN 22, 2020, View Source [SID1234561306]).

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Dr. Heman Chao, Chief Executive Officer of Helix is scheduled to present on Tuesday, June 23rd at approximately
1:20pm Eastern Time. The presentation will be webcast live on the conference event platform. You can access the
invitation and register to the Investor Forum with the following link:
View Source

About Proactive Investors
With offices in the US and in Canada, Proactive Investors enables companies and investors to connect. The group
is one of the fastest growing financial media portals in the world, providing breaking news, commentary and analysis
on hundreds of listed companies and pre-IPO businesses across the globe on a 24/7 basis. For more information:
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On June 22, 2020 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, reported financial results for the first quarter ended March 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Ayala Pharmaceuticals, JUN 22, 2020, View Source [SID1234561323]).

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"Despite the challenges presented by the COVID-19 pandemic, Ayala continues to operate from a position of strength both clinically and operationally. We remain on track to report additional data from our ongoing Phase 2 ACCURACY study in R/M ACC and to initiate our Phase 2 AL101 study of TNBC before year end," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "In addition, in May 2020, we completed our upsized IPO, providing us the financial stability to further develop both AL101 and AL102 across a wide range of genetically defined cancer indications, as well as in multiple myeloma in collaboration with Novartis."

Key Business and Clinical Highlights

Completed Upsized Initial Public Offering: In May 2020, Ayala successfully completed its initial public offering (IPO) of 3,666,667 shares of common stock and additional 274,022 shares in connection with the partial exercise of the underwriters’ option to purchase additional shares, at a public offering price of $15.00 per share. The total gross proceeds from the IPO were approximately $59.1 million. Through this offering Ayala broadened its shareholder base with a number of U.S. healthcare dedicated funds.

Received Fast Track Designation for AL101 for the Treatment of Recurrent/Metastatic Adenoid Cystic Carcinoma (R/M ACC): In March 2020, Ayala received Fast Track designation from the U.S. Food and Drug Administration (FDA) for AL101 for the treatment of R/M ACC with Notch-activating mutations. Ayala’s lead product candidate, AL101 is a potent, selective, injectable small molecule gamma secretase inhibitor (GSI) and was granted Orphan Drug Designation in May 2019 for the treatment of R/M ACC.

Commenced Dosing of Patients in 6mg Cohort of R/M ACC Study: Ayala commenced dosing of the second patient cohort in its ACCURACY study for the treatment of R/M ACC with Notch-activating mutations at the higher dose of 6mg. In the first cohort of the study, 45 patients were dosed at 4mg where clear signs of clinical activity were observed along with a favorable safety profile. The study is expected to dose 42 patients in the 6mg cohort.

Investigational New Drug (IND) Accepted by FDA for Phase 2 Study of AL101 For Treatment of TNBC: In April 2020, the FDA accepted the IND for the Phase 2 study of AL101 for treatment of triple negative breast cancer (TNBC.) The FDA approved the dosing to commence at 6mg in a monotherapy study to evaluate TNBC patients bearing Notch activating mutations who have undergone 3 prior lines of therapies or less.

Upcoming Milestones

On Track to Report Additional Phase 2 Data of AL101 in Patients with R/M ACC Data in The Second Half of 2020: Ayala plans to report additional data from its ongoing Phase 2 ACCURACY study of AL101 for the treatment of patients with R/M ACC with Notch-activating mutations, a rare malignancy of the secretory glands, at an upcoming medical meeting in the second half of 2020. Initial data demonstrating a 15% response rate and 69% disease control rate were previously reported out of the first 39 evaluable patients in the 4mg cohort.

On Track to Initiate Phase 2 Study of AL101 in Patients with Recurrent/Metastatic Triple Negative Breast Cancer (R/M TNBC) in The Second Half of 2020: Ayala plans to initiate a Phase 2 clinical trial of AL101 for the treatment of patients with R/M TNBC with Notch-activating mutations in the second half of 2020, subject to the impact of COVID-19.

First Quarter 2020 Financial Results

Cash Position: Cash and cash equivalents were $10.1 million as of March 31, 2020, as compared to $16.7 million as of December 31, 2019. Total cash, cash equivalents and marketable securities at March 31, 2020 did not include total net proceeds of approximately $55.0 million, after deducting underwriting discounts and commissions, from the Company’s IPO of 3,940,689 shares of common stock, including the partial exercise of the underwriters’ option to purchase additional shares, in May 2020. We expect the cash balance to fund operations into the second half of 2022 through potentially multiple key clinical and development milestones.

Collaboration Revenue: Collaboration revenue was $1.0 million for the first quarter of 2020, compared to $0.3 million for the same period in 2019. The increase in revenue was due to the advancement of our collaboration with Novartis on AL102 in combination with Novartis’ anti BCMA agent.

R&D Expenses: Research and development expenses were $5.1 million for the first quarter of 2020, compared to $2.8 million for the same period in 2019. The increase was primarily driven by higher costs related to the advancement of our ongoing Phase 2 study of AL101 in ACC and preparations for the initiation of the Phase 2 study in our TNBC trial.

G&A Expenses: General and administrative expenses were $1.3 million for the first quarter of 2020, compared to $0.8 million for the same period in 2019. The increase was primarily related to higher professional services and personnel costs to support the growth of the company.

Net Income/Loss: Net loss was $6.6 million, or $1.32 loss per share, for the first quarter of 2020, compared to $3.5 million, or $0.71 loss per share, for the same period in 2019, attributable mainly to the increase in our clinical operations.

On June 22, 2020 GRAIL, Inc., a healthcare company whose mission is to detect cancer early, reported that new data for its investigational multi-cancer early detection blood test will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II (Press release, Grail, JUN 22, 2020, View Source [SID1234561339]).

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Today, the majority of deadly cancers go undetected until they have progressed to late stages, when chances of survival are much lower. This is because most deadly cancers do not have guideline-recommended screening tests available.

GRAIL’s multi-cancer early detection technology can detect more than 50 cancers, with a very low false positive rate of less than one percent, through a single blood draw. When a cancer signal is detected, the test also identifies where the cancer is located in the body (the tissue of origin) with high accuracy.

"At GRAIL, we believe that in order to bend the curve on cancer mortality, we need to transition from screening only for individual cancers, to screening individuals for all types of cancers," said Joshua Ofman, MD, MSHS, Chief Medical Officer and External Affairs at GRAIL. "These new data demonstrate our continued commitment to scientific rigor, and further support the use of our multi-cancer early detection test in broad populations."

GRAIL’s multi-cancer early detection test is supported by evidence from its population-scale clinical study program, which is believed to be the largest ever conducted in genomic medicine.

The AACR (Free AACR Whitepaper) presentation details are included below. The slides and recordings will be available on www.grail.com at the conclusion of the meeting.

AACR Presentation Details

Abstract 2308/19
Joerg Bredno, et al. Classifier performance of a cfDNA-based multi-cancer detection test on uncommon cancer types
Session PO.PR02.02 — Clinical Prevention, Early Detection, and Interception 2: June 22, 2020: 9:00AM-6:00PM EDT

Abstract 721/15
Gregory Alexander, et al. Analytical validation of a multi-cancer early detection test with tissue localization using a cell-free DNA-based targeted methylation assay
Session PO.CL11.08 — Circulating Markers 1: June 22, 2020: 9:00AM-6:00PM EDT

Abstract 2114/1
Robert Calef, et al. HPV-driven cancers show distinct methylation signatures in cell-free DNA (cfDNA)
Session PO.BSB01.05 — Multi-omic and Multimodal Discovery: June 22, 2020: 9:00AM-6:00PM EDT

Abstract 139/2
Qinwen Liu, et al. cfDNA methylation profiling distinguishes lineage-specific hematologic malignancies
Session PO.MCB05.01— DNA Methylation: June 22, 2020: 9:00AM-6:00PM EDT

About GRAIL’s Multi-Cancer Early Detection Test

GRAIL’s multi-cancer early detection blood test can detect more than 50 cancers, across all stages, with a very low false positive rate of less than one percent, through a single blood draw. When a cancer signal is detected, the test identifies where in the body the cancer is located with high accuracy, an important step to guiding diagnostic next steps and care.