On November 7, 2019 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for its first quarter ended September 30, 2019 (Press release, MEI Pharma, NOV 7, 2019, View Source [SID1234550625]).

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"During this past quarter, our primary focus remained on advancing the clinical development of ME-401, as well as voruciclib, while continuing to explore additional clinical collaborations and partnering opportunities to effectively leverage the potential of our drug candidates for patients," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "As reported at the MEI Investor and Analyst event in October, the ME-401 intermittent dosing regimen continues to demonstrate high activity with a well-tolerated safety profile, supporting the Phase 2 TIDAL study in patients with relapsed or refractory follicular lymphoma. We are also focused on the expansion of the investigation of the ME-401 intermittent dosing regimen across a variety of B-cell malignancies in combination with other therapies, like Rituxan or zanubrutinib, an investigational BTK inhibitor as per our clinical collaboration agreement with BeiGene."

Recent Highlights

In October 2019, at MEI’s Investor and Analyst Event, the company reported updated data from the ongoing Phase 1b study of ME-401, an investigational selective oral inhibitor of phosphatidylinositol 3-kinase (PI3K) delta. The data demonstrate:
Overall response rates of 78% in relapsed or refractory (r/r) follicular lymphoma (FL) and 89% in r/r chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL).
Rates of Grade 3 adverse events of special interest related to ME-401 exposure were observed in <10% of patients dosed on an intermittent schedule (IS).
Median duration of response not yet reached in patients with FL or CLL/SLL on the IS regimen. Median follow-up for FL and CLL/SLL patients is 9.2 months (range 3.4-20.7 months) and 7.4 months (range 2.6-14.7 months), respectively.
In July 2019, Tamar Howson, M.S., MBA, a highly experienced business development executive with over 30 years of service in the pharmaceutical and biotechnology industry, joined the Board of Directors.
First Quarter Fiscal Year 2020 Financial Results

As of September 30, 2019, MEI had $65.9 million in cash, cash equivalents and short-term investments, with no outstanding debt.
For the quarter ended September 30, 2019, cash used in operations was $14.1 million, compared to $12.8 million for 2018.
Research and development expenses were $9.0 million for the quarter ended September 30, 2019, compared to $6.1 million for 2018. The increase was primarily related to increased development costs associated with ME-401 and increased headcount and professional services to support our development activities.
General and administrative expenses were $4.1 million for the quarter ended September 30, 2019, compared to $3.4 million for 2018. The increase primarily relates to increased headcount and increased professional services expenses to support our activities.
Revenues were $1.2 million for the quarter ended September 30, 2019, resulting from the recognition of fees allocated to research and development activities related to the Helsinn and Kyowa Kirin License Agreements. This compares with revenues of $0.5 million for the quarter ended September 30, 2018, resulting from the recognition of fees allocated to research and development activities related to the Helsinn License Agreement.
Net loss was $3.0 million, or $0.04 per share, for the quarter ended September 30, 2019, compared to net loss of $14.5 million, or $0.21 per share for 2018. The net loss decreased primarily as a result of a non-cash gain related to changes in the fair value of warrants issued in connection with the May 2018 financing. The company had 73,634,927 shares of common stock outstanding as of September 30, 2019, compared with 71,115,444 shares as of September 30, 2018.
The adjusted net loss for the quarter ended September 30, 2019, excluding a non-cash gain related to changes in the fair value of the warrants (a non-GAAP measure), was $12.3 million, compared to an adjusted net loss of $9.6 million for 2018.

On November 7, 2019 BioXcel Therapeutics, Inc. ("BTI" or the "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, reported it will host a conference call and webcast on Thursday, November 14, 2019 at 8:30 AM Eastern Time to discuss its third quarter 2019 operating and financial results (Press release, BioXcel Therapeutics, NOV 7, 2019, View Source [SID1234550662]).

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Conference Call & Webcast Details

Date/Time: Thursday, November 14, 2019, 8:30 AM Eastern Time
Domestic: 877-407-2985
International: 201-378-4915

The webcast will be accessible* under "Events" on the News & Media page of the Company’s website at www.bioxceltherapeutics.com.

Replay
Domestic: 877-660-6853
International: 201-612-7415
Conference ID: 13696573

*Replay available through November 28, 2019

On November 7, 2019 NantKwest Inc. (Nasdaq: NK), a next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and other diseases, reported the presentation of results from their Phase 2 trial investigation using aNK, NantKwest’s off-the-shelf natural killer cell-based therapeutic, in combination with N-803, ImmunityBio’s IL-15 superagonist, in patients with advanced refractory metastatic Merkel cell carcinoma (MCC) (NCT03853317) at The 34th Annual Meeting of the Society for Immunotherapy for Cancer (SITC 2019) on November 8, 2019 in National Harbor, Maryland. ImmunityBio is an affiliate company of NantKwest (Press release, NantKwest, NOV 7, 2019, View Source [SID1234550678]).

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Dr. Shailender Bhatia, Associate Professor of Medical Oncology at the University of Washington/Fred Hutchinson Cancer Research Center, will make an oral presentation entitled "Final results from a phase 2 study using off-the-shelf activated natural killer (aNK) cells in combination with N-803, an IL-15 superagonist, in patients with metastatic Merkel cell carcinoma (MCC)." That study demonstrated long term, durable responses in 2 of 7 patients with advanced refractory MCC. One of these patients achieved a radiologic complete response, and the second a durable partial response with more than 75% tumor regression and no subsequent therapy (response ongoing to date). Both patients with responses in the trial remain alive to date, 29 and 44 months after the initiation of treatment.

Results from this study provide evidence that treatment with aNK cell therapy can stimulate immune responses to checkpoint inhibitors, even in patients that have previously failed treatments using these types of therapies. One heavily pretreated subject refractory to pembrolizumab was rechallenged with this checkpoint inhibitor subsequent to receiving aNK cell therapy resulting in a durable complete response (CR) that is ongoing at this time, 44 months after receipt of first aNK cell infusion.

"We believe the results of this Phase 2 study are testament to the potential of our natural killer cell-based therapeutics to change the cancer treatment paradigm," said Dr. Patrick Soon-Shiong, Chairman and CEO of NantKwest. "Long term responses in patients with refractory metastatic disease is a remarkable achievement and is supportive of our upcoming trial, which will evaluate NantKwest’s off-the-shelf CD16-targeted haNK cells in combination with N-803 and Pfizer’s avelumab in patients with advanced MCC who are refractory to immune checkpoint inhibitors. We are particularly enthusiastic about the potential of aNK and N-803 to induce sensitization to checkpoint inhibitors, and thus, are evaluating this in our upcoming trial. We remain deeply committed to harnessing the power of immunogenic cell death across a wide range of indications to provide innovative treatments to patients with serious unmet need."

Other presentation highlights include:

Overall response rate of 29% (2 of 7 patients)
Evidence that aNK cell treatment may induce sensitivity to immune checkpoint inhibitors with a patient previously refractory to pembrolizumab demonstrating an ongoing complete response to the checkpoint inhibitor after completing aNK cell therapy
aNK cell therapy resulted in increased levels of tumor-infiltrating lymphocytes and increased immune response-related gene expression within tumor tissue
aNK cells in combination with N-803 were well-tolerated, with no treatment-related serious adverse events or grade ≥3 adverse events
Infusions all safely administered in the outpatient setting without observed immune related adverse events
Dr. Bhatia said, "Merkel cell carcinoma (MCC) is an aggressive skin cancer that is estimated to be three times more deadly than melanoma. PD-1 blockade works well for around 50 percent of patients with metastatic disease, but a significant proportion do not respond or progress later. Innate immunotherapy approach using aNK cells can potentially overcome resistance mechanisms in PD-1 refractory tumors. I am particularly enthusiastic about the preliminary results of the MCC study, with two complete responses in patients with very advanced metastatic disease."

On November 7, 2019 Thermo Fisher Scientific, the world leader in serving science, and Owlstone Medical, the global leader in Breath Biopsy for applications in early disease detection and precision medicine, reported that have entered into a collaborative partnership to advance the early diagnosis of cancer and other diseases through the discovery and validation of novel biomarkers by non-invasive breath sampling (Press release, Thermo Fisher Scientific, NOV 7, 2019, View Source [SID1234550695]).

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Through the integration of leading Orbitrap gas chromatography mass spectrometry (GC-MS) instrumentation into Owlstone Medical’s Breath Biopsy platform, the collaboration will qualify Thermo Fisher’s mass analyzers for the detection of new biomarkers via a validated discovery and routine analysis project. Developed in partnership, the new analytical methods will be used to conduct metabolomics studies of breath samples for unique biomarkers that could translate into non-invasive, routine screening solutions for improved early diagnosis of cancer and other disease.

"There is a growing need for non-invasive diagnostic solutions to support early disease detection, patient treatment and increase remission rates," said Morten Bern, director of marketing, gas chromatography, Thermo Fisher Scientific. "The combination of our Orbitrap GC-MS technology with Owlstone Medical’s Breath Biopsy platform provides a unique basis to improve patient outcomes through the discovery of novel biomarkers and their incorporation into research use and clinical tests."

Billy Boyle, co-founder and chief executive officer at Owlstone Medical, said: "The Orbitrap platform’s ability to detect a wide range of chemicals during targeted and untargeted analyses without losing selectivity or sensitivity, promises to be of substantial benefit to our Breath Biopsy platform. With a large and rapidly expanding installed base of GC Orbitrap systems, our collaboration with Thermo Fisher Scientific represents an exciting opportunity for cross-promotion of the platform and technique, by which the benefits of Breath Biopsy can be broadly realized."

On November 7, 2019 NanOlogy LLC, a clinical-stage oncology company, reported that interim data were presented last week at the 2019 ACG annual meeting from two of its clinical trials each evaluating endoscopic ultrasound guided fine needle injection (EUS-FNI) of NanoPac (submicron particle paclitaxel): one for treatment of locally advanced pancreatic cancer (LAPC) and the other for treatment of mucinous cystic neoplasms of the pancreas (MCNs) (Press release, NanOlogy, NOV 7, 2019, View Source [SID1234550711]).

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The pancreatic cancer clinical update of intratumoral NanoPac for treatment of LAPC was presented by Simon K. Lo, MD (Cedars-Sinai) as part of the Pancreatic Cancer/Esophagus plenary session.

The Phase 2a dose-rising and expansion pancreatic cancer trial is evaluating the safety and preliminary efficacy of NanoPac delivered intratumorally by EUS-FNI over 6 months in patients with nonresectable LAPC. After completion of the dose-rising phase, the trial has now enrolled 16 of 22 subjects into the dose expansion phase of the trial in which patients are receiving two intratumoral injections of NanoPac 4 weeks apart.

Highlights from Dr. Lo’s presentation on the dose expansion phase of the trial:

No drug-related local or systemic serious adverse events have been reported to date (n=25) including no reports of acute pancreatitis.
Of 7 subjects who have completed the 6-month study to date, one subject had a partial response with restaging from nonresectable to resectable (see video), 3 had stable disease, 1 had progressive disease, and 2 were withdrawn from the study.
Tumor volume decreases ranging from 7% to 76% have been seen in 7 of 11 subjects upon latest mpMRI to date at either the 3- or 6-month time point.
CA19-9 reductions of greater than 20% have been seen in 5 of 11 subjects upon latest measure to date at either the 3- or 6-month time point.
The pancreatic cyst clinical update on intracystic NanoPac for treatment of MCNs of the pancreas was presented during the poster sessions by Mohamed O. Othman, MD (Baylor College of Medicine). Dr Othman’s poster was recognized as a Presidential Poster, a distinction given to fewer than 5% of accepted abstracts each year, and was ultimately named co-winner for high quality, novel, unique and interesting research.

The Phase 2a dose rising and expansion pancreatic cyst trial is evaluating the safety and preliminary efficacy of NanoPac delivered intracystically by EUS-FNI following aspiration in patients with MCNs. The study is also enrolling in the dose expansion phase of the study and patients are receiving two intracystic injections of NanoPac 12 weeks apart.

Highlights from Dr. Othman’s poster presentation on the trial:

No drug-related local or systemic serious adverse events (SAE) have been reported to date (n=15) including no reports of acute pancreatitis. One case of gastric outlet obstruction that was possibly drug-related occurred in a subject who had a recent unrelated endoscopic procedure for hepatobiliary dysfunction. This condition was subsequently added as an exclusion criterion for the trial.
Plasma paclitaxel levels for all subjects analyzed have not exceed 1ng/mL suggesting that NanoPac particles are retained in the cyst over time.
Cyst volume decreases ranging from 8% to 89% have been seen in 9 of 11 subjects at last available imaging at either the 3- or 6-month time point.
Nanology plans to design follow-on clinical trials for both pancreatic cancer and pancreatic cysts in 2020 to further advance the programs toward regulatory submission.

In 2019, an estimated 57,000 new cases of pancreatic cancer will be diagnosed in the U.S. and 46,000 people will die from the disease. Pancreatic cancer is among the deadliest cancers with 9% survival rate at 5 years. It is also one of the few cancers for which no meaningful improvement in survival has been achieved in the last two decades. MCNs are a subset of pancreatic cysts that risk progression to pancreatic cancer. Patients with high risk MCNs may undergo surgical resection of the pancreas to remove the lesion, a complicated procedure associated with mortality and morbidity rates of 2% and 30% respectively. For both the disease itself and one of its common precursors, Nanology investigational drugs may offer a new way to help prevent or treat pancreatic cancer.

In addition to these trials, NanOlogy is advancing its therapeutic platform in preclinical and clinical programs across genitourinary, peritoneal, lung, and dermal cancers. The NanOlogy therapeutic platform is based on a proprietary submicron particle production technology that reduces the size of taxane API crystals by up to 400 times into stable submicron particles of pure drug with exponentially increased surface area and unique geometry. The characteristics of the particles have recently been granted a composition of matter patent valid in the US (9,814,685) and Australia until 2036, and pending globally.