On August 4, 2020 GT Biopharma, Inc. (OTCQB:GTBP) (GTBP.PA) an immuno-oncology company focused on innovative therapies based on the Company’s proprietary NK cell engager (TriKE) technology reported it had completed treatment of patients enrolled at Dose Levels 1 and 2, and begun enrolling patients for treatment at Dose Level 3, in its GTB-3550 TriKE Phase I/II clinical trial (Press release, GT Biopharma, AUG 4, 2020, View Source [SID1234562762]).

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Following initial treatment, the first patient treated with GTB-3550 achieved stable disease with respect to the number of acute myeloid leukemia (AML) blasts observed in their bone marrow before and after treatment. Additionally, we observed an increase in the patient’s total NK cell population attributable to the IL-15 component of the TriKE molecule with no appreciable increase of any hyper-active T-cell population. All patients treated to date with GTB-3550 have experienced no adverse reactions including no constitutional symptoms such as fever, tachycardia, or chills. Patients now being enrolled will be treated at a dose of 25µg/kg/day.

The clinical trial is being conducted at the Masonic Cancer Center, University of Minnesota under the direction of Dr. Erica Warlick. Additional clinical trial sites are being engaged in States that have modified their COVID-19 restrictions to allow for the restarting of clinical trials.

The open-label, dose-escalation Phase I portion of the trial will evaluate GTB-3550 in patients with CD33-expressing, high risk myelodysplastic syndromes, refractory/relapsed AML or advanced systemic mastocytosis, and will determine safety and tolerability as well as the pharmacologically active dose and maximum tolerated dose of GTB-3550. The Phase II portion of the trial is planned to further evaluate the efficacy of GTB-3550 in this patient population.

Mr. Anthony Cataldo, the Chairman and Chief Executive Officer of GT Biopharma commented "we are pleased to have advanced treatment to the next higher dose of GTB-3550." Mr. Cataldo further stated "we hope to continue to see additional signals of efficacy at the new higher dose of GTB-3550."

About GTB-3550 Trispecific NK cell Engager (TriKE)
GTB-3550 is the Company’s first TriKE product candidate being initially developed for the treatment of AML. GTB-3550 is a single-chain, tri-specific scFv recombinant fusion protein conjugate composed of the variable regions of the heavy and light chains of anti-CD16 and anti-CD33 antibodies and a modified form of IL-15. The natural killer (NK) cell stimulating cytokine human IL-15 portion of the molecule provides a self-sustaining signal that activates NK cells and enhances their ability to kill. We intend to study GTB-3550 in CD33 positive leukemias such as AML, myelodysplastic syndrome, and other CD33+ hematopoietic malignancies.

On August 4, 2020 Kaleido Biosciences, Inc. (Nasdaq: KLDO), a clinical-stage healthcare company with a chemistry-driven approach to targeting the microbiome to treat disease and improve human health, reported financial results for the second quarter 2020 and provided a corporate update (Press release, Kaleido Biosciences, AUG 4, 2020, View Source [SID1234562761]).

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"We have made several important advancements across critical areas of the business, including initiating two clinical studies in our COVID-19 program and strengthening our financial foundation by extending our cash runway into the second half of 2021," said Michael Bonney, Executive Chair of Kaleido. "We anticipate top-line data from the 350-patient, multi-center COVID-19 study in the fourth quarter. We also continue to make progress on our preclinical programs and look forward to reporting data in the fourth quarter from our immuno-oncology, cardiometabolic and liver disease programs."

Company Highlights

Initiated two non-IND clinical studies that are part of the ongoing COVID-19 clinical development program evaluating KB109 when added to Supportive Self-Care in outpatients with mild-to-moderate COVID-19 disease: clinical study K031 is a multi-center study of approximately 350 patients and clinical study K032, in collaboration with Massachusetts General Hospital, aims to include approximately 50 patients.

Completed a $35.6 million offering of common stock.
Anticipated Milestones

Clinical and ex vivo data from KB174, being developed for the treatment of hepatic encephalopathy (HE), to be featured in poster presentations during The Digital International Liver Congress (EASL 2020) August 27-29, 2020.

Topline data from the multi-center (K031) clinical study of KB109 in patients with mild-to-moderate COVID-19 disease expected in Q4 2020 and top-line data from the study with Massachusetts General Hospital (K032) are expected in the first quarter of 2021. A patient may be enrolled in the study if an initial positive COVID-19 test result is received and the patient remains symptomatic.

Initiating a clinical study in Q3 2020 evaluating KB295 in approximately 30 patients with mild-to-moderate ulcerative colitis (UC). Topline results are expected in mid-2021.

Preclinical data from the immuno-oncology, cardiometabolic and liver diseases programs expected in Q4 2020.

Topline data from Phase 2 clinical trial (UNLOCKED) of KB195 in patients with UCD inadequately controlled on standard of care in second half 2021.
Second Quarter 2020 Financial Results

For the second quarter 2020, Kaleido reported a net loss of $18.9 million, or $0.59 per common share, compared to $24.6 million, or $0.83 per common share, for the second quarter 2019. The 2020 second quarter net loss includes non-cash stock-based compensation expenses of $2.8 million, as compared to $2.4 million in the second quarter of 2019.

Research and development (R&D) expenses were $12.8 million for the second quarter 2020, compared to $18.8 million for the second quarter 2019. The second quarter decrease in R&D, as compared to the second quarter of 2019, was primarily driven by decreased external development, manufacturing, and research costs.

General and administrative (G&A) expenses were $5.6 million for the second quarter 2020, compared to $6.2 million for the second quarter of 2019. The second quarter 2020 decrease in G&A, as compared to the second quarter of 2019, was primarily due to decreased personnel-related costs, partially offset by increased facility-related expenses.

As of June 30, 2020, the Company reported cash and cash equivalents of $71.0 million and has cash runway into the second half of 2021.

About Microbiome Metabolic Therapies (MMT)

Kaleido’s Microbiome Metabolic Therapies, or MMTs, are designed to drive the function and distribution of the microbiome’s existing microbes in order to decrease or increase the production of metabolites, or to advantage or disadvantage certain bacteria in the microbiome community. The Company’s initial MMT candidates are targeted, synthetic glycans that are orally administered, have limited systemic exposure, and are selectively metabolized by enzymes in the microbiome. Kaleido utilizes its discovery and development platform to study MMTs in microbiome samples to rapidly advance MMT candidates rapidly into clinical studies in healthy subjects and patients. These human clinical studies are conducted under regulations supporting research with food, evaluating safety, tolerability and potential markers of effect. For MMT candidates that are further developed as therapeutics, the Company conducts clinical trials under an Investigational New Drug (IND) or regulatory equivalent outside the U.S., and in Phase 2 or later development.

On August 4, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported its participation at the 2020 Wedbush PacGrow Healthcare Virtual Conference. Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present an overview of the company on Tuesday, August 11, 2020 at 2:20 p.m. ET / 11:20 a.m. PT. The virtual conference will be held from August 11-12, 2020.

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A live audio webcast of the presentation will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay available for 30 days following the event.

On August 4, 2020 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported that it will issue financial results for its fiscal quarter ended June 30, 2020 on Thursday, August 6th, 2020 (Press release, Anavex Life Sciences, AUG 4, 2020, View Source [SID1234562759]).

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Management will host a conference call on Thursday, August 6th, 2020 at 11:00 am Eastern Time to review financial results and provide an update on its clinical programs and corporate highlights. Following management’s formal remarks, there will be a question and answer session with equity analysts.

Conference Call / Webcast Information:

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 901-2585 and international callers should dial 1 (404) 835-7099. Please use confirmation number 49865428, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.

On August 4, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biopharmaceutical company creating a new class of drugs based on targeted protein degradation, reported financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, Arvinas, AUG 4, 2020, View Source [SID1234562758]).

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"We were pleased to report updated dose escalation data from our Phase 1/2 trial of ARV-110 in men with metastatic castration-resistant prostate cancer. These data demonstrated safety and showed an early efficacy signal in a heavily pretreated patient population, highlighting the potential benefit of our PROTAC platform. These data are a first for a targeted PROTAC protein degrader, and we are excited to report more mature data from our ARV-110 program, and to announce interim data from our ARV-471 Phase 1/2 trial, in the fourth quarter of 2020," said John Houston, Ph.D., President and Chief Executive Officer at Arvinas.

"As we look to the balance of 2020, we are in a stronger position than ever to lead the creation of an entirely new class of therapies that targets and degrades disease-causing proteins," added Dr. Houston.

Business Highlights and Recent Developments

The company presented early efficacy and updated safety data for ARV-110 at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data demonstrated that Arvinas’ PROTAC protein degrader ARV-110 had an acceptable safety profile (as of the data cut-off) and are the first to show early signs of clinical efficacy for ARV-110 in a heavily pretreated patient population.
The company enhanced its Board of Directors with the appointments of Linda Bain and Wendy Dixon, Ph.D.
Anticipated Milestones and Expectations

For the ARV-110 program, Arvinas expects to provide an update from its Phase 1/2 trial in the fourth quarter of 2020.
For the ARV-471 program, Arvinas expects to share Phase 1 dose escalation clinical data in the fourth quarter of 2020.
Arvinas expects to provide information about the advancement of additional programs in its robust preclinical pipeline in the second half of 2020.
Financial Guidance

Based on its current operating plan, Arvinas expects its cash, cash equivalents, and marketable securities will be sufficient to fund its planned operating expenses and capital expenditures into 2022.

Financial Highlights

Cash, Cash Equivalents, and Marketable Securities Position: As of June 30, 2020, cash, cash equivalents, and marketable securities were $242.7 million as compared with $280.9 million as of December 31, 2019. The decrease in cash, cash equivalents and marketable securities of $38.2 million for the first six months of 2020 was primarily related to cash used for operations of $43.2 million and the purchase of lab equipment and lease hold improvements of $3.2 million, partially offset by $4.0 million received from two collaborators, cash provided from the exercise of stock options of $2.6 million and changes in unrealized gain on marketable securities of $1.6 million.

Research and Development Expenses: Research and development expenses were $23.4 million for the quarter ended June 30, 2020, as compared with $16.0 million for the quarter ended June 30, 2019. The increase in research and development expenses of $7.4 million for the quarter was primarily related to increases in clinical trial and CMC expenses associated with our AR program of $2.3 million and our ER program of $1.0 million, in addition to increases in preclinical expenses of $4.1 million associated with exploratory programs and investments in platform research.

General and Administrative Expenses: General and administrative expenses were $8.8 million for the quarter ended June 30, 2020, as compared to $6.4 million for the quarter ended June 30, 2019. The increase of $2.4 million was primarily related to an increase in personnel and facility related costs of $2.2 million.

Revenues: Revenue was $5.7 million for the quarter ended June 30, 2020, as compared with $4.0 million for the quarter ended June 30, 2019. The increase of $1.7 million was primarily related to the collaboration and license agreement with Bayer that was initiated in July 2019. Revenues are generated from the license and rights to technology fees and research and development activities related to the collaboration and license agreement with Bayer that was initiated in July 2019, the collaboration and license agreement with Pfizer that was initiated in January 2018, and the amended and restated option, license and collaboration agreement with Genentech that was initiated in November 2017.

Net Loss: Net loss was $25.2 million for the quarter ended June 30, 2020, as compared with $17.2 million for the quarter ended June 30, 2019. The increase in net loss for the quarter was primarily due to increased research and development expenses and increased general and administrative expenses.

About ARV-110

ARV-110 is an orally bioavailable PROTAC protein degrader designed to selectively target and degrade the androgen receptor (AR). ARV-110 is being developed as a potential treatment for men with metastatic castration-resistant prostate cancer.

ARV-110 has demonstrated activity in preclinical models of AR mutation or overexpression, both common mechanisms of resistance to currently available AR-targeted therapies.

About ARV-471

ARV-471 is an orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer.

In preclinical studies, ARV-471 demonstrated near-complete ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed superior anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor.