On October 21, 2021 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, reported financial results for the third quarter ended September 30, 2021, and provided an overview of recent business highlights (Press release, Cytori Therapeutics, OCT 21, 2021, View Source [SID1234591764]).

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"In the third quarter, we made meaningful progress executing our strategy to develop novel radiotherapeutics for rare and central nervous system cancers," said Marc H. Hedrick M.D., President and Chief Executive Officer of Plus Therapeutics. "We increased the ReSPECT-GBM trial dose by 40%, were accepted to present an update of the ReSPECT-GBM trial data at the 2021 Society for Neuro-Oncology Annual Meeting in November, received clearance from the U.S. Food and Drug Administration for our Investigational New Drug application for leptomeningeal metastases, entered into an additional agreement for the production of our radiopharmaceutical products, and strengthened our leadership team with the appointment of a highly experienced Chief Medical Officer with decades of radiopharmaceutical development experience."

RECENT HIGHLIGHTS

Rhenium-186 NanoLiposome ( 186 RNL), a novel radiotherapy in development for several rare cancer targets

Recurrent Glioblastoma (GBM)

The ongoing U.S. multi-center ReSPECT-GBM Phase 1 dose-finding clinical trial is designed to safely and effectively deliver high doses of radiation directly to brain tumors. Thus far, 22 patients with recurrent GBM have been treated in the ReSPECT-GBM trial across eight cohorts. A 40% increase in absorbed radiation doses per tumor in Cohort 8 over Cohort 7 have been achieved without dose-limiting toxicities. Patient recruitment continues at UT Health San Antonio, UT Southwestern in Dallas, and MD Anderson in Houston. Learn more at ClinicalTrials.gov.
The Company will present interim data from its ReSPECT-GBM clinical trial at the 2021 American Society for Radiation Oncology (ASTRO) Annual Meeting scheduled October 24-27, 2021 and at the 2021 Society for Neuro-Oncology (SNO) Annual Meeting and Education Day scheduled November 18-21, 2021.
The Company will host a roundtable discussion of the ReSPECT-GBM clinical trial on November 18, 2021 at 4:00 p.m. EST, which will be webcast, with Toral Patel, M.D., Associate Professor, Department of Neurosurgery, UT Southwestern Medical Center, and Andrew J. Brenner, M.D., Ph.D., Professor of Medicine, Neurology, and Neurosurgery at The University of Texas.
Leptomeningeal Metastases (LM)

The Company received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for 186RNL for the treatment of LM.
The Company expects to initiate patient accrual in a Phase 1 dose escalation trial of 186RNL (ReSPECT-LM) in the fourth quarter of 2021.
ReSPECT-LM will be a multi-center, sequential cohort, open-label, single dose, dose escalation Phase 1 study of the safety, tolerability, biodistribution, dosimetry, and anti-tumor activity of 186RNL given intraventricularly via an Ommaya reservoir to subjects over 18 years old with LM. The primary endpoints of the study are the incidence and severity of adverse events and serious adverse events and the incidence of dose limiting toxicities. The secondary endpoints are the overall response rate, duration or response, progression free survival, and overall survival. Learn more at ClinicalTrials.gov.
Pediatric Brain Cancer (PBC)

The Company intends to submit an IND application to the FDA for 186RNL for the treatment of PBC. The Company expects to initiate patient accrual in a Phase 1 dose escalation trial of 186RNL (ReSPECT-PBC) in 2022.
Drug Manufacturing and Supply Chain

During the third quarter of 2021, the Company entered into an agreement with RadioMedix, Inc. for the commercial production of the Company’s investigational radiopharmaceuticals. Thus far in 2021, the Company has entered into five collaboration agreements to support its process development and analytical chemistry activities, as well as to strengthen its supply chain in compliance with current good manufacturing practices (GMP) for the manufacture of 186RNL. The Company remains on track to deliver GMP 186RNL by mid-2022.
Corporate

During the third quarter of 2021, the Company announced the appointment of Norman LaFrance, M.D. to the position of Chief Medical Officer and Senior Vice President. Dr. LaFrance joins Plus Therapeutics with nearly 40 years of experience as a nuclear medicine physician and as an executive in the pharmaceutical and healthcare industries. Dr. LaFrance area of specialization is radiotherapeutic drug research and development as well as regulatory approval and commercialization of molecular imaging, diagnostic and therapeutic products.
UPCOMING EVENTS AND MILESTONES

Over the next 12 months, the Company intends to accomplish the following key business objectives:

Complete enrollment of the ongoing cohort in the ReSPECT-GBM Phase 1 clinical trial.
Fully analyze the ReSPECT-GBM Phase 1 clinical trial data and refine its Phase 2/3 clinical trial plans with the FDA.
Initiate patient accrual in the Phase 1 ReSPECT-LM clinical trial.
Submit an IND application to the FDA for 186RNL for the treatment of patients with PBC.
Complete CMC activities for GMP 186RNL and complete CMC related meeting with the FDA.
THIRD QUARTER 2021 FINANCIAL RESULTS

As of September 30, 2021, the Company’s cash balance was $21.3 million, compared to $8.3 million as of December 31, 2020.
Total operating expenses for the third quarter of 2021 were $3.5 million, compared to total operating expenses of $1.4 million for the same quarter in 2020. This increase is primarily due to increased research and development expenses in 2021.
Net loss for the third quarter of 2021 was $3.7 million, or $(0.28) per share, compared to a net loss of $1.7 million, or $(0.39) per share, for the same quarter in 2020. The increase in net loss is primarily due to the aforementioned increase in research and development expenses.
Third Quarter 2021 Results Conference Call

The Company will hold a conference call and live audio webcast at 5:00 p.m. Eastern Time today to discuss its financial results and provide a general business update.

Event: Plus Therapeutics Third Quarter 2021 Results Conference Call
Date: Thursday, October 21, 2021
Time: 5:00 p.m. Eastern Time
Live Call: 877-876-9173 (toll free); 785-424-1667 (Intl.); Conference ID: PSTVQ321
The webcast can be accessed live via the investor section of the Plus Therapeutics website at ir.plustherapeutics.com/events and will be available for replay beginning two hours after the conclusion of the conference call.

On October 21, 2021 Athenex (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, reported that the Company will provide a corporate and financial update for the third quarter 2021, on Friday, November 5, 2021, before the market opens (Press release, Athenex, OCT 21, 2021, View Source [SID1234591681]). Athenex’s management team will host a conference call and live audio webcast at 8:00am Eastern Time.

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To participate in the call, dial either the domestic or international number fifteen minutes before the conference call begins:

The live conference call and replay can also be accessed via audio webcast here and on the Investor Relations section of the Company’s website under "Events and Presentations," located at View Source

On October 21, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported that it will host a conference call and audio webcast on Thursday, October 28, 2021 at 2:30 pm CET (8:30 am ET) to discuss its third quarter 2021 financial results and provide a business update (Press release, argenx, OCT 21, 2021, View Source [SID1234591700]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website for approximately one year following the call.

On October 21, 2021 Flamingo Therapeutics reported that was born out of a collaboration between three partners in Flanders – VIB, UGent and KU Leuven – and the University of Michigan in the US (Press release, Flamingo Therapeutics, OCT 21, 2021, View Source;utm_medium=rss&utm_campaign=the-dark-matter-of-the-genome-flamingo-therapeutics-taps-potential-of-mysterious-rna-for-cancer-therapies [SID1234591687]). "The foundations of the company were laid in 2016, when the research groups of prof. Pieter Mestdagh at UGent and prof. Jean-Christophe Marine at VIB and KU Leuven published a paper revealing a connection between certain long non-coding RNAs and malignant melanoma, the most aggressive form of skin cancer," explains Floor Stam, COO at Flamingo. These insights were later linked to research carried out at the University of Michigan.

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Thanks to this unique expertise, the young biotech company is currently one of the only players in the world that is able to explore the potential of these long-coding RNAs (lncRNAs, pronounced as ‘link RNA’). According to Flamingo, lncRNAs account for 27% of the non-coding RNA genes, which make up around two-thirds of all genes. Non-coding RNA genes are not translated into proteins and the function of most of them is currently very unclear (hence the ‘dark matter’ moniker).

Cancer targets from out of the dark
"For a long time, the non-coding portion of the genome was considered to be useless ‘junk DNA’. But it actually has an enormous potential for new therapies against many different diseases," says Stam. Flamingo is specifically looking into the possibilities for cancer therapies, as lncRNAs play a role in the energy provision of cancer cells when they divide uncontrollably. If you can manipulate that process, you should be able to produce a very targeted treatment that attacks the cancer cells and leaves healthy cells alone.

Flamingo’s approach could possibly limit the side effects of innovative cancer therapies. Stam also believes this new solution could be produced more rapidly than more traditional therapies that target proteins. "Because we have access to the unparalleled screening and hit identification capabilities of our collaborators at Ionis, a clinical candidate molecule can be developed in 12 to 14 months," specifies Stam.

"For a long time, the non-coding portion of the genome was considered to be useless ‘junk DNA’. But it actually has an enormous potential for new therapies against many different diseases." – Floor Stam, Flamingo Therapeutics

To identify targets for cancer therapies in this predominantly uncharted region of the genome, the company has developed the unique discovery engine FLAME (Flamingo LncRNA Antisense Mining Engine). Based on the findings in the groundbreaking 2016 paper, the initial focus has predominantly been on finding targets for treating the aggressive skin cancer melanoma, where the Flamingo team has been focusing on a lncRNA molecule named SAMMSON.

High profile partnership
Thanks to a recently-signed collaboration agreement with the American company Ionis Pharmaceuticals – a global leader in RNA-targeted therapies – Flamingo is now expanding its activities dramatically. The company is taking over three clinical programs for RNA-targeted cancer therapeutics from Ionis, as well as one preclinical program concentrating on lncRNAs.

"Ionis has been at the forefront of RNA-targeted therapy for thirty years… We are also a very interesting partner for them, because we concentrate on RNA-targeting therapies in oncology and have unique expertise in the field of lncRNAs." – Mike Garrett, Flamingo Therapeutics

"From the start, Ionis has been a crucial shareholder of ours, helping us to go in the right direction," says Mike Garrett, CEO of Flamingo. "Ionis has been at the forefront of RNA-targeted therapy for thirty years and offers invaluable knowhow and experience in clinical programs. We are also a very interesting partner for them, because we concentrate on RNA-targeting therapies in oncology and have unique expertise in the field of lncRNAs." While Flamingo’s headquarters are located at the biotech incubator in Leuven, much of its activities also take place in facilities at San Diego close to Ionis’ home base.

Potential solutions for a range of cancer types
Flamingo’s most advanced drug candidate in development is called danvatirsen and targets the STAT-3 gene. "It is showing great potential in the battle against head and neck cancers and lymphoma," says Garrett. Other drug candidates, targeting the androgen receptor and IRF4 gene, are being developed to fight prostate cancer and multiple myeloma (a type of bone marrow cancer).

Read this BioVox article to learn about Precirix, another start-up tackling cancer with an innovative approach.

The Flemish company has also taken over a preclinical-stage program focusing on a well-characterized lncRNA target named MALAT1. "This pioneering research could lead to solutions for different cancers, but for the moment we especially foresee therapeutic benefits for patients with triple-negative breast cancer," says COO Floor Stam. The preclinical stage of this program should be finished by the end of 2022. In ideal circumstances, the clinical programs could lead to new therapies on the market already in 2026.

To support the company’s rapid expansion, Flamingo is now preparing a new investment round. For the moment, its main investors are the well-known biotechnology investors PMV and Kurma Partners, Belgian and French respectively.

On October 21, 2021 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO), a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported the first patient has been dosed in the Phase 1/2 ASPEN-05 study evaluating the combination of evorpacept, a next-generation CD47 blocker, with venetoclax and azacitidine for the treatment of patients with acute myeloid leukemia ("AML") (Press release, ALX Oncology, OCT 21, 2021, View Source [SID1234591867]).

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The Phase 1 portion of the ASPEN-05 study will characterize the safety and confirm the dose of evorpacept in combination with venetoclax and azacitidine in patients with relapsed/refractory AML and previously untreated AML who are not candidates for intensive induction therapy. The Phase 2 portion of the study will evaluate the efficacy of the combination in patients with previously untreated AML who are not candidates for intensive induction therapy.

ASPEN-05 is based on promising preclinical data with evorpacept in combination with venetoclax and azacitidine in non-clinical models of leukemia, as well as clinical data from an ongoing phase 1 study (NCT03013218) evaluating evorpacept in combination with multiple anti-cancer agents in both solid and hematologic malignancies.

"Patients with either relapsed/refractory AML or previously untreated AML who are not considered eligible for intensive induction regimens remain in urgent need of tolerable and effective new therapies," said Harry Erba, M.D., Ph.D., Director of the Leukemia Program at the Duke Cancer Institute. "We are looking forward to evaluating the addition of evorpacept to venetoclax and azacitidine. ALX148 was specifically designed for use in combination to maximize phagocytosis of tumor cells while minimizing the toxicities commonly associated with other CD47-targeted approaches."

"ASPEN-05 builds upon compelling evorpacept combination activity observed in non-clinical models of leukemia. With demonstrated tolerability of evorpacept in multi-agent chemotherapy regimens, we are excited to characterize evorpacept with this standard backbone of AML therapy," said Sophia Randolph, M.D., Ph.D., Chief Medical Officer of ALX Oncology. "Through blockade of the CD47 myeloid checkpoint, evorpacept in combination with venetoclax and azacitidine may potentially transform treatment options for patients with AML."

The ASPEN-05 trial is registered under NCT04755244. ALX Oncology owns worldwide commercial rights to evorpacept.

About Acute Myeloid Leukemia

AML is an aggressive blood cell cancer that can rapidly progress and lead to death if not treated promptly. AML is the most common form of acute leukemia in adults, with an estimated 19,940 new cases and 11,180 deaths from AML in the United States. Due to advanced age and comorbidities at the time of diagnosis, a significant number of patients are not considered eligible for intensive and potentially curative therapies. Despite advances in available care, the estimated 5-year survival for patients in the United States with AML remains only 29%.