On November 14, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, reported its quarterly results for the third quarter ended September 30, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, NOV 14, 2019, View Source [SID1234551242]).

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"We are pleased with the progress made on both of our clinical development programs during the quarter," stated Vimal Mehta, Chief Executive Officer of BTAI. "BXCL501, our candidate for the acute treatment of agitation in patients with schizophrenia and bipolar disease and delivered in a sublingual thin film, is highly differentiated from the current standards of care, which can produce unwanted side effects and be difficult for caregivers to administer. BXCL501 has the potential to significantly improve care for patients, while providing healthcare providers with an important new option for treating their patients. We’re looking forward to reporting pivotal data during the first half of 2020."

Dr. Mehta added, "We are also pleased with the progress of our Phase 1b/2 double combination study of BXCL701 and Keytruda for tNEPC and anticipate additional safety data readouts from both cohorts in the fourth quarter of this year, followed by expected initial efficacy data in the first half of 2020."

Third Quarter 2019 and Recent Highlights

BXCL501-Neuroscience Program-

BXCL501 is an investigational sublingual thin film of dexmedetomidine, a selective alpha-2A adrenergic receptor agonist, designed for the treatment of acute agitation. The Company believes BXCL501 may directly target a causal agitation mechanism.

·BXCL501 met its primary endpoint and demonstrated statistically significant mean reduction in PEC (PANSS, or the Positive and Negative Syndrome Scale, Excitatory Component) in the Phase 1b trial with agitated schizophrenia patients. Pivotal studies for the acute treatment of agitation in schizophrenia and bipolar patients are expected to initiate in Q4 2019, with data readouts expected 1H 2020;

The Phase 1b/2 study of BXCL501 for acute treatment of agitation in geriatric dementia/Alzheimer’s disease is expected to begin in Q4 2019, with data expected in 1H 2020;

· Initiated BXCL501 strategic initiative to investigate the feasibility of development of digital device technology, such as the Apple Watch, that can be used in conjunction with BXCL501 to enhance the prevention and treatment of agitation, specifically in geriatric dementia patients;

·Awarded grant by CDMRP to expand clinical development of BXCL501 program for the treatment of alcohol and substance abuse disorders related to PTSD in collaboration with Yale University.

BXCL701-Immuno-Oncology Program-

BXCL701 is an orally-delivered small molecule, innate immunity activator designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune evasion by targeting Fibroblast Activation Protein (FAP). It has shown single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

· The Phase 1b/2 trial of BXCL701 and Keytruda for tNEPC is ongoing. The Company presented safety and tolerability data from the first patient cohort at the Annual Prostate Cancer Foundation Scientific Retreat and is currently enrolling a second patient cohort. BTI expects to report additional safety findings by year-end before advancing to the Phase 2 stage of the trial;

·BXCL701 received third orphan drug designation (ODD) from the FDA for the treatment of AML. Potential to expand clinical development of BXCL701 program into hematological malignancies;

· The BXCL701 phase of the triple combination study of BXCL701, bempegaldesleukin (NKTR-214, Nektar Therapeutics, Inc.) and BAVENCIO (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in pancreatic cancer is expected to be initiated following Nektar and Pfizer’s safety run-in trial of a double combination of bempegaldesleukin and avelumab and the outcome of that trial.

Strengthened Balance Sheet

·BioXcel raised gross proceeds of $19.0 million in the quarter, the net of which, together with current reserves, provides sufficient capital to fund operations through key data readouts including Phase 3 and Phase 1b/2 studies with BXCL501.

Third Quarter 2019 Financial Results

BTI reported a net loss of $9.0 million for the third quarter of 2019, compared to a net loss of $4.9 million for the same period in 2018. The third quarter 2019 results include approximately $0.8 million in non-cash stock based compensation.

Research and development expenses were $7.1 million for the third quarter of 2019, as compared to $3.8 million for the same period in 2018. The increase was primarily due to an expansion of research and development activities, including increased personnel costs, clinical trials expenses, and professional fees, associated with BTI’s two lead product candidates.

General and administrative expenses were $2.0 million for the third quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses, professional fees and costs associated with operating as a public company.

As of September 30, 2019, cash and cash equivalents totaled approximately $40.3 million which included proceeds from the Company’s follow-on-financing completed on September 30, 2019. BTI believes it is well positioned to execute on key milestones.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The replay will be available through November 28, 2019.

On November 14, 2019 Sierra Oncology, Inc. (Nasdaq: SRRA), a late-stage drug development company focused on the development and commercialization of momelotinib, a JAK1, JAK2 & ACVR1 inhibitor with a potentially differentiated therapeutic profile for the treatment of myelofibrosis, reported that Dr. Nick Glover, President and Chief Executive Officer, will present an overview of the company entitled "Building Momentum for Patients with Myelofibrosis" at the Jefferies 2019 London Healthcare Conference in London, United Kingdom (Press release, Sierra Oncology, NOV 14, 2019, View Source [SID1234551288]).

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The presentation is scheduled for 6:00 p.m. GMT on Wednesday, November 20, 2019. A live audio webcast and archive of the presentation will be accessible through www.sierraoncology.com.

On November 14, 2019 Precision Optics Corporation, Inc. (OTCQB: PEYE), a leading designer and manufacturer of advanced optical instruments for the medical and defense industries, reported operating results on an unaudited basis for its first quarter fiscal year ended September 30, 2019 (Press release, Precision Optics, NOV 14, 2019, View Source [SID1234551322]).

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First quarter fiscal 2020 highlights:

Revenue for the quarter (ended September 30, 2019) was $2.5 million compared to $1.6 million in the same quarter of the previous fiscal year, an increase of 61% driven primarily by Ross Optical operating as a division of Precision Optics.
Gross margins of 39% compared to 30% in the same quarter of the prior year, and sequentially improved for the third consecutive quarter.
Net loss of $86,000 during the quarter included $122,000 of stock-based compensation and service fees.
Precision Optics’ CEO, Joseph Forkey, commented, "The first quarter financial results highlight the positive impact of our evolving capabilities, which now include Ross Optical, resulting in a year-over-year revenue increase of 61% and positive operating cash flow. During the quarter, we continued deliveries of the three products that went into commercial level production last year and we expect our expanding pipeline of Precision Optics-enabled products to allow for ongoing growth. Our business strategy of enabling leading medical device companies around the world to meet the increasing demands of the surgical community, who are requiring more enhanced and smaller imaging systems for minimally invasive surgery, as well as the rapid proliferation of 3D endoscopes for surgical robotic systems, continues to gain traction."

Dr. Forkey continued, "As we look to the remainder of fiscal 2020, we expect growth from the production of currently commercialized products while continuing to advance our pipeline of engineering projects toward commercialization. Further, we look to build upon the improvements we achieved during the first quarter, where overall gross margins were 39% compared to 30% a year ago. We are working to improve gross margins on our base business and are benefitting from the higher margin profile of Ross Optical. Overall, we maintain a disciplined investment strategy in sales and marketing and engineering capabilities to fuel growth while generating positive cash flows."

The following table summarizes the first quarter (unaudited) results for the periods ended September 30, 2019 and 2018:

Conference Call Details
The Company has scheduled a conference call to discuss the first quarter 2020 financial results for Thursday, November 14, 2019 at 5:00 p.m. EDT.

Call-in Information: Interested parties can access the conference call by dialing (877) 317-6789 or (412) 317-6789.

Live Webcast Information: Interested parties can access the conference call via a live Internet webcast, which is available at View Source

Replay: A teleconference replay of the call will be available until November 21, 2019 at (877) 344-7529 or (412) 317-0088 confirmation #10136579. A webcast replay will be available at View Source

On November 14, 2019 Ascentage Pharma (6855.HK), a globally-focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, hepatitis B virus and age-related diseases, reported that the updated preliminary results from a Phase I clinical trial on the company’s novel investigational drug HQP1351 has been accepted for oral presentation at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (Press release, Ascentage Pharma, NOV 14, 2019, View Source [SID1234551340]). Qian Jiang, M.D., who will be presenting, and Xiaojun Huang, M.D. from Peking University People’s Hospital, are the principal investigators of this study. HQP1351 is a third-generation BCR-ABL inhibitor for the treatment of drug-resistant chronic myeloid leukemia (CML).

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Early results from this Phase I study had been accepted for oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting in 2018. Being accepted for oral presentations two years in a row is a strong indication of the interest on HQP1351’s promising efficacy and safety data and updates.

The 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition will be held on December 7–10, 2019, in Orlando, Florida, United States. The ASH (Free ASH Whitepaper) Annual Meeting is the largest gathering of the international hematology field, bringing together the latest and most cutting-edge research and development in hematology.

The oral report will present updated safety and efficacy data from the Chinese Phase I trial of HQP1351. As of May 27, 2019, a total of 101 Chinese patients were enrolled in the study. Trial data have demonstrated that HQP1351 was well tolerated with good and durable anti-tumor activity in TKI-resistant CML patients, including those with the T315I mutation. HQP1351 is Ascentage Pharma’s novel investigational drug with the potential to effectively address the acquired drug resistance commonly associated with the first- and second-generation BCR-ABL inhibitors.

At ASH (Free ASH Whitepaper) 2019, in addition to the oral presentation on HQP1351, Ascentage Pharma will also release data in a poster presentation for a new research on the BCL-2/BCL-xL dual inhibitor APG-1252, which can overcome ibrutinib-resistance conferred by the upregulation of integrin pathway and BCL-xL. APG-1252 is one of the key drug candidates in Ascentage Pharma’s pipeline that focuses on restoring cell apoptosis function and inhibiting cancer growth.

Oral Presentation

Title: An Updated Safety and Efficacy Results of Phase 1 Study of HQP1351, a Novel Third Generation BCR-ABL Tyrosine Kinase Inhibitor (TKI), in Patients with TKI-Resistant Chronic Myeloid Leukemia

Report#: 493
Session: 632
Presenter: Qian Jiang, MD
Time: Sunday, December 8, 4:30 pm (EST)
Location: W308, Orange County Convention Center (OCCC)

Poster Presentation

Title: A Novel BCL-2/BCL-xL Dual Inhibitor Overcomes Ibrutinib-Resistance Conferred By the Upregulation of Integrin Pathway and BCL-xL

Poster#: 2573
Session: 605
Time: Sunday, December 8, 6:00 PM – 8:00 PM (EST)
Location: Hall B, Orange County Convention Center (OCCC)

About HQP1351

HQP1351 is a novel kinase inhibitor developed by Ascentage Pharma. It is an oral third-generation BCR-ABL inhibitor targeting a broad spectrum of BCR-ABL mutants, including those with the T315I mutation, to treat drug-resistant CML patients. A Phase I clinical trial for patients with TKI-resistant CML has been completed and pivotal Phase II clinical trial are ongoing in China. In addition, a Phase I trial in patients with GIST has been initiated in China.

About APG-1252

APG-1252 is a novel, highly potent, small molecule drug designed to restore apoptosis through selective inhibition of the Bcl-2 and Bcl-xL proteins for the treatment of SCLC (small-cell lung cancer), lymphoma and other solid tumors. Ascentage is currently conducting two Phase I dose-escalation trials in patients with advanced cancers in the United States and Australia, and Phase I dose-escalation/expansion trial as a monotherapy in patients with SCLC in China.

On November 14, 2019 Theragnostics, which is developing molecular radiotherapy for imaging and treating a broad range of cancers, reported two strategic hires to its leadership team with the appointment of Dr Daniel Stevens as Chief Medical Officer (CMO) and Chris Stirling to the Board as Non-Executive Director (Press release, Theragnostics, NOV 14, 2019, View Source [SID1234551133]).

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Chris Sterling has extensive experience in the life sciences industry advising multinational companies such as GlaxoSmithKline and Syngenta (Novartis/AstraZeneca) in cross-border acquisitions, mergers and disposals. He was previously Chairman of KPMG’s Global Life Sciences sector where he built the practice in markets across the world including China, India and Brazil as well as the established markets in North America, Europe and Japan. He has held many leadership positions and senior client facing roles at KPMG both as an Audit and Transaction Services partner. Chris is an FCA Chartered Accountant and holds a Master’s in Engineering Science from the University of Oxford.

Recently appointed CMO Dr Daniel Stevens has a successful track record in US and EU radiopharmaceutical registrations and has led development teams through Phase 1 to Phase 3 of novel radiopharmaceutical therapies. Daniel is a UK trained physician with a broad frontline clinical experience including five years working as both an academic and a clinician, and a specialist period undertaking basic cancer research at the University of Oxford.

Commenting on the news, Nick Stevens, Executive Chairman of Theragnostics, said:
"As we continue to grow, it is important to be able to attract high calibre individuals who have the global experience to help Theragnostics on its journey. Chris’ business acumen, extensive experience and exemplary track record in striking deals with large players in the sector will be invaluable in implementing our strategy of bringing novel diagnostic agents and new targeted therapies to patients."

Greg Mullen, Chief Executive Officer of Theragnostics, added:
"I’m very pleased to have someone of Dan’s expertise and track record join our team. This appointment comes at a pivotal moment when we prepare for the next stage of growth as a company and complements the management team as we advance our programmes into clinical development."

Theragnostics’ technology platform enables the development of molecular radiotherapy based on a PARPi for imaging and treating cancer. Theragnostics modifies a PARPi drug molecule with a radioactive atom to create a radionuclide PARPi (rPARPi). This can either be used to image PARP in a cancer patient for diagnostic use or the radioactive isotope can be used to deliver a therapeutic dose of radiation into tumour cells, which offers the potential to molecularly target the radiation in order to hit and kill tumour cells whilst avoiding damage to healthy cells and associated side effects.