Avalo Therapeutics Successfully Eliminates $35 Million Debt

 In a major achievement, Avalo Therapeutics (Nasdaq: AVTX) proudly announces the payoff of the remainder of its $35 million debt owed to Horizon Technology Finance Corporation (Nasdaq: HRZN). This significant milestone not only signifies the company's steadfast commitment to financial stability but also paves the way for future accelerated growth and progress toward executing its ambitious plans to advance its most promising drug candidates, including its anti-LIGHT mAb (quisovalimab or AVTX-002) and its BTLA agonist fusion protein (AVTX-008).

https://finance.yahoo.com/news/avalo-therapeutics-successfully-eliminates-35-110000091.html

Phathom: Vonoprazan NDA Submission for Non-Erosive GERD

 

• Submission based on positive results from Phase 3 PHALCON-NERD-301 in which vonoprazan 10 mg and 20 mg controlled heartburn symptoms through the entire 6 months of the study with a safety profile consistent with prior vonoprazan studies
  

• New drug application (NDA) seeks U.S. Food and Drug Administration (FDA) approval for vonoprazan as a daily treatment for Non-Erosive GERD, the largest subcategory of GERD with an estimated U.S. adult population of 38 million
  

• FDA action date for Non-Erosive GERD NDA expected in the third quarter of 2024

https://finance.yahoo.com/news/phathom-pharmaceuticals-announces-vonoprazan-nda-120100625.html

BioVie near completion of Phase 3 Trial of NE3107 in Mild to Moderate Alzheimer's Disease

• Expects to announce topline data expected in the November/December timeframe.

• Enrolled patients had underlying medical conditions that are known risk factors for dementia that NE3107 has the potential to improve.

https://finance.yahoo.com/news/biovie-announces-completion-last-patient-120000875.html

 

Omega Therapeutics Inc OMGA announced preliminary data from the initial two dose level cohorts (n=8) from Part 1 of its Phase 1/2 MYCHELANGELO I study of OTX-2002 in hepatocellular carcinoma and other solid tumors associated with the c-MYC (MYC) gene. 

OTX-2002, the company's lead development candidate, is designed to pre-transcriptionally downregulate MYC, a master oncogene implicated in more than 50% of all cancers and approximately 70% of HCC cases.

MYCHELANGELO I is an ongoing Phase 1/2 open-label trial evaluating OTX-2002 as a monotherapy (Part 1) and in combination with standard-of-care therapies (Part 2).

Highly specific on-target engagement and intended epigenetic changes at the target genomic loci were observed for all eight patients across both dose levels, as evidenced by a robust increase in cell-free DNA MYC methylation signal following administration with OTX-2002. 

The increased methylation signal persisted throughout the two-week dosing interval.

Epigenetic modulation of MYC translated to rapid, robust, and durable downregulation of MYC expression in all eight patients, with mean reductions across both dose levels of approximately 55% observed seven days following administration with OTX-2002.

The increased methylation and corresponding downregulation of MYC expression observed clinically are within the ranges that led to anti-tumor activity in preclinical xenograft models.

Consistent pharmacokinetic (PK) data across both dose levels with rapid clearance and minimal variability observed within and between patients.

No accumulation was observed following repeat administration and low, transient levels of immune response were observed with no related adverse events or impact on PK.

OTX-2002 was generally well tolerated, with no dose-limiting toxicities.

https://www.benzinga.com/general/biotech/23/09/34911032/omega-therapeutics-cancer-program-shows-encouraging-anti-tumor-activity-in-liver-cancer

Candel: Early Survival Data From Lung Cancer Trial

 Candel Therapeutics Inc CADL announced updated activity data from its ongoing, open-label, phase 2 trial of CAN-2409 plus valacyclovir in combination with continued immune checkpoint inhibitor (ICI) treatment in patients with non-resectable, stage III/IV non-small cell lung cancer (NSCLC), who have an inadequate response to front line anti-PD(L)1 therapy. 

These patients historically have had an expected median overall survival of 10-13 months. 

The aim of the CAN-2409 immunotherapy antitumor strategy is to raise the tail on the survival curve by increasing the number of long survivors beyond 10-13 months.

Of the 40 evaluable patients, 15 have lived ≥ 12 months; of these, 10 patients have lived > 18 months, of whom 70% (7/10) were alive as of the last follow-up. 

All four patients (100%) with OS > 24 months were alive at the last follow-up, with the longest reaching 31.7 months.

An additional 18 out of the 40 evaluable patients are also alive but have not yet reached 12 months of follow-up.

Candel continued to observe a favorable safety/tolerability profile after CAN-2409 treatment.

Candel expects to share topline overall survival data for Cohort 2 in the second quarter of 2024, assuming mature data then.

https://www.benzinga.com/general/biotech/23/09/34915228/candel-therapeutics-reveals-early-survival-data-from-lung-cancer-trial

Takeda Puts Nearly $600M on the Line for AcuraStem’s ALS Program

 Takeda on Monday inked a development and commercialization agreement with California-based biotech AcuraStem to target amyotrophic lateral sclerosis and other neurodegenerative diseases.

Under the terms of the deal, Takeda will pledge upfront and milestone payments of up to approximately $580 million if the collaboration meets all future clinical, regulatory and commercial milestones. AcuraStem will also be eligible to receive tiered royalties on potential net sales of any product that emerges from the partnership.

In return, the Japanese multinational pharma will gain access to AcuraStem’s PIKFYVE program, which includes AS-202, an investigational antisense oligonucleotide (ASO) and the biotech’s most mature candidate. Takeda will have exclusive worldwide license to AS-202 as well as to AcuraStem’s other PIKFYVE-directed assets.

AcuraStem will take charge of certain studies to enable an Investigational New Drug application for AS-202, while Takeda will be responsible for all development activities, including clinical studies, regulatory efforts and commercialization, according to the announcement.

PIKFYVE is an emerging therapeutic target in amyotrophic lateral sclerosis (ALS), according to AcuraStem’s website. The company’s studies have shown that using an ASO therapeutic to inhibit the gene that encodes for the kinase helps neurons discard toxic proteins and pathologic aggregates. In turn, this restores healthy neuron function and prevents neurodegeneration.

This mechanism of action also points to the potential of AcuraStem’s PIKFYVE approach to frontotemporal dementia (FTD) and other diseases caused by the misfolding, and eventual clumping, of the protein TDP-43.

In many pre-clinical models of ALS and FTD, using PIKFYVE-targeted ASOs, such as AS-202, can reverse the pathological protein deposition in neurons, according to AcuraStem.

In a statement, Sarah Sheikh, head of Takeda’s Neuroscience Therapeutic Area Unit, said that “through its unique dual mechanism of action, which addresses TDP-43 aggregation and improves TDP-43 function,” AS-202 has the potential to address the existing unmet need among patients with “debilitating neurological diseases.”

Takeda has also inked other high-value deals this year. In July 2023, the company signed a strategic discovery collaboration and licensing agreement with British biotech F-star Therapeutics to discover next-generation and multi-specific antibodies for cancer targets. Though the complete financial details of the deal were not revealed, the companies said that F-star could receive up to $1 billion in milestones.

In January, Takeda entered into another potential billion-dollar deal with Hutchmed to develop fruquintinib, a VEGF inhibitor being trialed for colorectal cancer.

https://www.biospace.com/article/takeda-puts-nearly-600m-on-the-line-for-acurastem-s-als-program/

EU in talks with Moderna over new deal for COVID vaccines

 The European Union is in talks with Moderna over a new supply deal for the company's COVID-19 vaccines amid concerns over a rise in infections in the region, the Financial Times reported on Tuesday, citing two people familiar with the matter.

The company supplied its COVID vaccines to the EU during the pandemic, but the contract for its Omicron-adjusted vaccines ended in August last year and was not renewed.

At least eight countries in the union are interested in a new supply deal, the report said, citing one of the people.

"Moderna manufactured its updated COVID-19 vaccine at risk to ensure we could support member states with their upcoming vaccination campaigns," a company spokesperson said, adding the updated shot was approved in Europe and ready to be supplied.

Updated COVID shots that will be used in the vaccination campaign this year will target the XBB.1.5 variant of Omicron.

The spokesperson said an agreement with the European Commission is the fastest way to procure the shots, which would help ensure a diversified supply of COVID vaccines with vaccination campaigns set to start in October.

A spokesperson for the European Commission declined to comment on the talks over Moderna's supply contract, but added it "has been assessing the needs and interests of the Member States for additional COVID-19 vaccines".

Currently, Pfizer and its German partner BioNTech's updated COVID shot is the most widely used vaccine in the region.

Pfizer and BioNTech in May reached a deal to amend their COVID-19 vaccine contract by cutting the number of shots the EU must buy and pushing the delivery deadline to 2026 due to gradually falling demand for the shots

https://finance.yahoo.com/news/1-eu-talks-moderna-over-123334736.html