On October 21, 2021 Ultimovacs ASA ("Ultimovacs") (OSE ULTI), a clinical-stage leader in immune stimulatory vaccines for cancer, reported its universal cancer vaccine, UV1, in combination with checkpoint inhibitors has received Fast Track designation from the U.S. FDA in the treatment of unresectable or metastatic melanoma – either as add-on therapy to pembrolizumab or as add-on therapy to ipilimumab (Press release, Ultimovacs, OCT 21, 2021, View Source [SID1234591774]). Ultimovacs is currently evaluating UV1 as add-on therapy to ipilimumab and nivolumab as first-line treatment for unresectable or metastatic melanoma in a Phase II study named INITIUM.

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Ultimovacs senior management will host a webcast on the Fast Track designation and the supporting clinical data at 13.00 CET on Thursday October 21.

The FDA Fast Track process is designed to facilitate the development and expedite the review of drugs that meet urgent needs in serious medical conditions. Fast Track designation enables early and frequent communication with the FDA to support the drug’s development, as well as entitlement to a Rolling Review of the Biologic License Application. Drugs with Fast Track designation may also be considered for Accelerated Approval and Priority Review provided certain criteria are met.

"We are delighted UV1 has received the Fast Track designation and look forward to working more closely with the FDA to bring UV1 to melanoma patients as soon as possible," said Carlos de Sousa, Chief Executive Officer of Ultimovacs. "The FDA’s decision recognizes the potential synergy of UV1 and checkpoint inhibitors and will greatly encourage physicians and patients involved in our Phase II clinical trial INITIUM. We remain committed to progressing UV1 in our four ongoing Phase II clinical studies and assessing development of UV1 with pembrolizumab in advanced melanoma."

The Fast Track designation is based on data from two separate Phase I trials of UV1 in combination with checkpoint inhibitors, either with pembrolizumab (anti-PD-1) or with ipilimumab (anti-CTLA-4). According to data disclosed recently, UV1 in combination with pembrolizumab as a first-line treatment in advanced melanoma was shown to be safe with promising early efficacy data; an objective response rate (ORR) of 57% was reached with 30% of patients achieving a complete response (CR), or complete disappearance of their tumors. At 24 months follow up, 80% of patients were alive. In a second cohort, similar levels of tumor destruction were seen with 90% of patients remained alive after one year. Peer-reviewed data published in May 2021 in Frontiers in Immunology demonstrated that UV1 with ipilimumab was safe and showed signals of prolonged efficacy in advanced melanoma, the combination achieving ORR of 33% and 5-year overall survival (OS) rate of 50%.

UV1 is currently being investigated in combination with checkpoint inhibitors in four Phase II trials: in unresectable or metastatic melanoma, ovarian cancer, head and neck squamous cell carcinoma and malignant pleural mesothelioma.

Ultimovacs’ webcast on the Fast Track designation and clinical data at 13.00 CET (7.00 am EST) on Thursday 21 October 2021 will be accessible here and from the company website.

On October 21, 2021 Silence Therapeutics plc (AIM:SLN and Nasdaq: SLN), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (siRNA) therapeutics for the treatment of diseases with significant unmet medical need, reported it is hosting its R&D Day in New York City (Press release, Silence Therapeutics, OCT 21, 2021, View Source [SID1234591770]).

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During the event, Silence plans to showcase its proprietary mRNAi GOLD platform and current clinical programs, including SLN360 for cardiovascular disease due to high lipoprotein(a), or Lp(a), and SLN124 for thalassemia and myelodysplastic syndrome (MDS) as well as a newly added program in polycythemia vera (PV). Silence will also provide an update on its growing discovery pipeline and plans to deliver 2-3 INDs per year from 2023.

Mark Rothera, President and Chief Executive Officer of Silence Therapeutics, said: "The vision at Silence has always been to build an exquisite platform that would enable the development of longer-lasting precision medicines to transform patients’ lives. We are pleased to report strong progress across both our proprietary and partnered pipelines, including plans to further expand the SLN124 program into a third indication, PV, where endogenous hepcidin modulation can potentially improve outcomes for 3.5 million people worldwide who currently have very limited treatment options. Through our hybrid business model, we are committed to maximizing the substantial opportunity for our mRNAi GOLD platform and remain well positioned to deliver 2-3 INDs per year from 2023."

Giles Campion, MD, EVP, Head of R&D and Chief Medical Officer of Silence Therapeutics, said: "We are very encouraged by the outcomes from our recent safety review committee meeting for SLN360. Based on their recommendations, we have now defined our dose range and are ready to start the multiple-ascending dose study. We are also extending the follow-up period in the single-ascending dose study to fully characterize SLN360’s duration of action, which may be even longer than we initially anticipated. We look forward to further evaluating SLN360 in the clinic and remain on-track to report topline data in the single-ascending dose study in the first quarter of 2022."

mRNAi GOLD Proprietary Program Updates

SLN124 Program
New highlighted updates include:

Plans to pursue new PV indication and start a phase 1 trial in the second half of 2022.
FDA acceptance of the US IND in myelodysplastic syndrome (MDS).
Enrollment continues in the two single-ascending dose studies for thalassemia and MDS. Silence anticipates topline data from both studies in the third quarter of 2022.
Positive results from the healthy volunteer study reported in May 2021 accepted for poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 11-14, 2021.
SLN360 Program
New highlighted updates include:

On-track to report top-line data in the single-ascending dose study in the first quarter of 2022.
Key outcomes from the independent safety review committee meeting:
Recommendation to extend follow-up period in the single-ascending dose study from 150 days to 365 days to fully assess the duration of action, which may be longer than initially anticipated based on preclinical modelling.
The therapeutic dose range has been established based on Cohorts 1-4 (optional Cohort 5 not needed) and study can now proceed to the multiple-ascending dose phase.
On-track to initiate phase 2 development in the second half of 2022, pending regulatory discussions.

Building Proprietary Pipeline

Advancing two new undisclosed proprietary programs following the Hansoh collaboration announced last week (read full release here). Hansoh has the option to license China region rights at the end of phase 1.

mRNAi GOLD Partnered Program Updates

AstraZeneca Collaboration for Cardiovascular, Renal, Metabolic and Respiratory Diseases

Continuing work with AstraZeneca to develop siRNAs for two undisclosed targets.
On-track to initiate work on five targets within the first three years of the collaboration.
Mallinckrodt Collaboration for Complement-Mediated Diseases

Progressing IND-enabling studies for SLN501 C3-targeting program and expect to initiate a phase 1 study in the first half of 2022.
Developing siRNAs for two other undisclosed complement-mediated disease targets.
Hansoh Collaboration

In addition to the two proprietary programs outlined above, Silence will work with Hansoh on a third undisclosed program which Hansoh has the option to license worldwide at the point of IND filing.
As part of the Hansoh agreement, Hansoh made a $16 million upfront cash payment and Silence has the potential for up to $1.3 billion in additional milestones plus royalties tiered from low double-digit to mid-teens on Hansoh net product sales.
Targeting 2-3 INDs per year from 2023

Silence remains on-track to deliver 2-3 INDs per year from 2023 through both its proprietary and partnered mRNAi GOLD platform programs. Key highlighted updates include:
Expanded translational genomics efforts, contributing both to finding new, genetically validated disease-causing genes and continuously fine-tuning the siRNA design algorithm using machine learning
A fine-tuned drug discovery process
Aiming for a mixed-risk portfolio of validated and novel targets with projects at different stages of drug discovery ensuring multiple shots on goal
The Company’s R&D Day will be held today, October 21, 2021 from 9:00 am to 11:30 am ET at the Convene, 530 Fifth Avenue, in New York City and will include a live video stream.

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 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, reported the company will host a conference call and live webcast on Wednesday, Nov. 3, 2021, at 8:00 a.m. ET to report its third quarter 2021 financial results and other business highlights (Press release, Agios Pharmaceuticals, OCT 21, 2021, https://investor.agios.com/news-releases/news-release-details/agios-webcast-conference-call-third-quarter-2021-financial [SID1234591761]).

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A live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The conference call can be accessed by dialing 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 5748074. The webcast will be archived and made available for replay on the company’s website beginning approximately two hours after the event.

On October 21, 2021 Exacis Biotherapeutics, Inc., a development-stage immuno-oncology company working to harness the immune system to cure cancer, reported initiation of a strategic partnership with Toronto-based Centre for Commercialization of Regenerative Medicine (CCRM) for specialty manufacturing services related to the development of Exacis’ innovative, iPSC-derived mRNA-engineered NK cell products to treat cancer (Press release, Exacis Biotherapeutics, OCT 21, 2021, View Source [SID1234591758]). The partnership also includes a cash investment into Exacis by CCRM Enterprises Holdings Ltd., the for-profit venture investment arm of CCRM, which will be used to fund operations.

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Exacis CEO Gregory Fiore, MD, commented, "We welcome CCRM as a key partner to allow us to rapidly advance our virus-free manufacturing processes to make novel NK cell products that are engineered for performance and to avoid rejection. CCRM is a recognized leader in iPSC-derived cell therapy development and manufacturing and we are thrilled to have them as a partner. Their confidence in Exacis is evidenced by the accompanying investment, by CCRM Enterprises Holdings Ltd., underscoring the unique value proposition offered by Exacis’ differentiated platform and approach to cell therapies. We look forward to partnering with CCRM’s CDMO experts to apply our mRNA based technologies to develop best-in-class products to treat challenging hematologic and solid tumors."

Cynthia Lavoie, PhD, President and CIO of CCRM Enterprises Inc. added, "We are pleased to support Exacis by way of an investment, and with our sector expertise and specialized infrastructure. This is a successful model that we have employed in the past to support promising technologies and together we will develop leading cell therapy products that utilize the substantial potential of the Exacis platform as it advances its iPSC-derived cell programs.