Ocuphire Pharma to Present in the BIO CEO & Investor Conference

On February 23, 2024 Ocuphire Pharma, Inc. (Nasdaq: OCUP) ("Ocuphire"), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of retinal and refractive eye disorders, reported that George Magrath, M.D. M.B.A, M.S. CEO of Ocuphire, will be presenting a company overview at the BIO CEO & Investor Conference being held February 26-27 2024 in New York City (Press release, Ocuphire Pharma, FEB 23, 2024, View Source [SID1234640424]). Company management will also be participating in one-on-one meetings throughout the conference.

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BIO CEO & Investor Conference – February 26-27, 2024
Title: Ocuphire Pharma, Inc. (OCUP) Company Presentation
Presenter: George Magrath, M.D. M.B.A, M.S.
Date: Tuesday, February 27, 2024
Time: 2:45 – 3:00pm ET
Location: Plymouth Room, Marriott Marquis, New York, NY

If you are interested in arranging a 1×1 meeting, please contact your conference representative or send an email to [email protected]. For more details, please see the Investors and Events section of Ocuphire’s corporate website.

Actinium Highlights Improved Survival with Iomab-B in TP53 Positive Relapsed Refractory Acute Myeloid Leukemia Patients in the SIERRA Trial and Other Presentations at the 2024 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT® and CIBMTR

On February 23, 2024 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of targeted radiotherapies, reported results from three poster presentations at the 2024 Tandem Meetings | Transplantation & Cellular Therapy (TCT) Meetings of ASTCT (American Society for Transplantation and Cellular Therapy and CIBMTR (Center for International Blood and Marrow Transplant Research) being held in San Antonio, Texas (Press release, Actinium Pharmaceuticals, FEB 23, 2024, View Source [SID1234640423]). The posters detailed results and findings from the Phase 3 SIERRA trial of Iomab-B (131I-Apamistamab) including; improved rates of Complete Remission (CR), durable Complete Remission (dCR) and survival in patients with a TP53 mutation, key aspects of radiopharmaceutical dosimetry as related to Iomab-B, and early results from a Phase 1 study demonstrating safety and lymphodepletion from Iomab-ACT conditioning with CD19 CAR-T therapy.

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Dr. Avinash Desai, Actinium’s Chief Medical Officer, said, "Patients with a TP53 mutation have a desperate need for viable treatment options to improve outcomes. As seen in the SIERRA trial, Iomab-B led bone marrow transplant can overcome the negative impact of a TP53 mutation, producing complete remissions in more than 50% of patients as well as significant durable complete remissions. This is in stark contrast to the 0% complete remission and durable complete remission rate seen in the TP53 positive patients on the control arm who received best available treatment based on physician’s choice. We are excited to further highlight these important outcomes to the transplant community and look forward to presenting additional findings from the SIERRA trial in our upcoming oral presentations."

The presented Iomab-B Phase 3 SIERRA trial results and highlights include:

Response Rate by TP53 Mutational Status and Treatment

Iomab-B + Crossover

Conventional Care

TP53 Positive

CR

Durable CR

N=27

N=15

N=4

55.56%

14.81%

N=10

N=0

N=0

0%

0%

TP53 Wildtype

CR

Durable CR

N=93

N=54

N=15

58.06%

16.13%

N=23

N=4

N=0

17.39%

0%

CR = Complete Remission

Improved Survival with Iomab-B

Iomab-B + Crossover

Conventional Care

N=27

N=10

Median Overall Survival

(95% CI)

5.49

(3.94, 8.25)

1.66

(0.99,2.96)

Hazard Ratio

(95% CI)

0.23

(0.1., 0.52)

p-value (log-rank)

0.0002

Upcoming Iomab-B Phase 3 SIERRA Trial 2024 TCT Oral Presentations:

Title: 131I-Apamistamab Improves Outcomes in Patients 65 Years and Older with Relapsed or Refractory AML

Date & Time: Saturday, February 24, 2024, at 11:45 AM

Title: Targeted Myeloablative Radiation Using 131I-Apamistamab Prior to Allogeneic Hematopoietic Cell Transplant for Patients with R/R AML Results in Robust Engraftment

Date & Time: Saturday, February 24, 2024, at 10:30 AM

In addition, Actinium presented results from its ongoing phase 1 trial using Iomab-ACT as conditioning prior to CD19 CAR-T therapy for patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia or Diffuse Large B-cell Lymphoma. Importantly, no patients (0/4) developed immune effector cell-associated neurotoxicity syndrome (ICANS) of any grade, a major safety measure of the study, as ICANS is observed in 25% or more of pts w/ R/R B-ALL and DLBCL treated with various CAR T-cell products. Iomab-ACT demonstrated transient depletion of peripheral blood lymphocytes and monocytes. Persistence of CAR T-cells up to 8 weeks and minimal non-hematologic toxicities have been observed to date.

About the TCT Tandem Meetings

The Tandem Meetings I Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR are the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR). Administrators, clinicians, data manager / clinical research professionals, fellows-in-training, investigators, laboratory technicians, MD/PhDs, nurses, nurse practitioners, pharmacists, physician assistants, and other allied health professional attendees benefit from a full scientific program that addresses the most timely issues in hematopoietic cell transplantation and cellular therapy.22211111111

TG Therapeutics to Host Conference Call on Fourth Quarter and Year-End 2023 Financial Results and Business Update

On February 23, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that a conference call will be held on Wednesday, February 28, 2024, at 8:30 AM ET to discuss results for the fourth quarter and year-end 2023 and provide a business outlook for 2024. Michael S. Weiss, Chairman and Chief Executive Officer, will host the call (Press release, TG Therapeutics, FEB 23, 2024, https://ir.tgtherapeutics.com/news-releases/news-release-details/tg-therapeutics-host-conference-call-fourth-quarter-and-year-3 [SID1234640422]).

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In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Fourth Quarter and Year End Update Call. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

TG Therapeutics will announce its financial results for this period in a press release to be issued prior to the call.

Silence Therapeutics Achieves $10 Million Milestone Payment from AstraZeneca Collaboration Following the Initiation of Phase 1 Trial

On February 23, 2024 Silence Therapeutics plc, Nasdaq: SLN ("Silence" or "the Company"), an experienced and innovative biotechnology company committed to transforming people’s lives by silencing diseases through precision engineered medicines, reported that the initiation by AstraZeneca of a phase 1 clinical trial of the first product candidate under its siRNA (short interfering RNA) collaboration, has triggered a $10.0 million milestone payment to Silence (Press release, Silence Therapeutics, FEB 23, 2024, View Source [SID1234640421]).

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"This represents the first clinical milestone under our collaboration with AstraZeneca and the third program to enter the clinic from our mRNAi GOLD platform," said Craig Tooman, President and CEO of Silence. "This is a very exciting time for Silence as we are beginning to establish ourselves as a platform company progressing multiple clinical programs targeting both rare and common genetic diseases. In addition to this great achievement under our collaboration with AstraZeneca, we are also pleased with the continued advancement of our proprietary pipeline with encouraging clinical data now emerging from our zerlasiran program in high Lp(a) and divesiran program in polycythemia vera."

"We are pleased to have achieved this important clinical milestone through our collaboration with Silence Therapeutics. Our goal in working together is to advance these novel programs and develop the next wave of innovative therapies that address cardiovascular, renal and metabolic diseases," said Regina Fritsche Danielson, SVP, Early Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D, AstraZeneca.

Silence and AstraZeneca initiated a multi-target collaboration in March 2020 focused on using Silence’s proprietary mRNAi GOLD platform to develop siRNA therapeutics for cardiovascular, renal, metabolic and respiratory diseases. Under the agreement, AstraZeneca will pay Silence an option fee of $10 million for each selected target at the point of candidate nomination. The deal covers up to ten targets. For each target selected, Silence is eligible for up to $140 million in development milestones and up to $250 million in commercialization milestones as well as tiered royalties on net sales ranging from high single digit to low double digit.

Biodexa Announces Positive Top Line Phase I Clinical Trial Results for Diffuse Midline Glioma and Provides R&D Update

On February 23, 2024 Biodexa Pharmaceuticals PLC, a clinical stage biopharmaceutical company developing a pipeline of innovative products for the treatment of diseases with unmet medical needs including Type 1 diabetes and rare / orphan brain cancers, reported an R&D update including positive top-line clinical trial results of a recently completed Phase 1 study of MTX110 in patients with diffuse midline glioma, or DMG, and results of a preclinical experiment designed to demonstrate tolimidone’s potential for beta cell proliferation in an in vitro model (Press release, Midatech Pharma, FEB 23, 2024, View Source [SID1234640419]).

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MTX110

In an investigator initiated study conducted by Columbia University Irving Medical Center, patients newly diagnosed with DMG were administered MTX110 via convection enhanced delivery ("CED") using a subcutaneous pump connected to a catheter directly implanted into the pons in a 3+3 dose-escalating design (NCT 04264143). As this was the first ever study of repeated infusions to the pons via an implanted CED catheter, the primary objective of the study was safety and tolerability and, accordingly, the number of infusions was limited to two, each of 48 hours, 7 days apart. Nine patients were treated in the study (30 M group, n=3; 60 M group, n=4; 90 M group (optimal dose), n=2). One patient in the 60 M group suffered a severe adverse event assessed by the investigators as not related to the study drug but related to the infusion and tumor anatomy. Although the study was not powered to reliably demonstrate efficacy, median overall survival (OS) of patients in the study was 16.5 months. This compares favourably with median survival rate in a cohort of 316 cases of 10.0 months (Jansen et al, 2015. Neuro-Oncology 17(1):160-166).

Study investigators are planning to present detailed results of the trial at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) being held on June 28-July 2, 2024 in Philadelphia, PA.

Tolimidone

On the Company’s behalf, a CRO conducted an in vitro experiment designed to demonstrate tolimidone’s potential for beta cell proliferation using reaggregated pancreatic islets. The results of the experiment were inconclusive in that they did not correlate with the results previously seen in in vitro and in vivo studies of tolimidone. The Company believes there are a few possible explanations to the outcome of this in vitro study and accordingly, plans to move ahead rapidly with an in vivo preclinical study with similar objectives while continuing preparations for its planned Phase IIa open-label study of tolimidone in patients with Type 1 diabetes due to start recruitment later this year.