On April 30, 2025 Bold Therapeutics Inc., a clinical-stage biopharmaceutical company pioneering novel oncology treatments, reported that its Sr. Director of Preclinical Development, Mark Bazett, PhD, is presenting a late-breaking poster on BOLD-100’s potent neuroprotective properties later today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, Bold Therapeutics, APR 30, 2025, View Source [SID1234652397]).

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Session: Late-Breaking Research: Experimental and Molecular Therapeutics 4

Neuropathy Rates from BOLD-100-001 vs. Historical Controls
Neuropathy Rates from BOLD-100-001 vs. Historical Controls
Bold Therapeutics’ Data in a Model of Cold Allodynia
Bold Therapeutics’ Data in a Model of Cold Allodynia
Date & Time: April 30, 2025, from 9:00 AM to 12:00 PM

Location: Poster Section 51, Poster Board #14

Poster Code: #LB432

Poster Title: "Clinical-stage anticancer agent BOLD-100 demonstrates protective effects against oxaliplatin-induced peripheral neuropathy in an in-vivo rat model"

Addressing a Critical Unmet Need: Oxaliplatin-Induced Peripheral Neuropathy

In Bold Therapeutics’ global Phase 1b/2a trial (BOLD-100-001, NCT04421820), patients with advanced gastrointestinal cancers — including colorectal (mCRC), gastric (GC), biliary tract (BTC), and pancreatic cancer (PDAC) — were treated with BOLD-100 in combination with FOLFOX (5-fluorouracil, oxaliplatin, leucovorin). Across 109 patients in six treatment arms, the study showed encouraging efficacy data, which was previously presented at ESMO (Free ESMO Whitepaper) GI and ASCO (Free ASCO Whitepaper) in 2024.

A surprising finding from this trial was a profound reduction in both the frequency and severity of oxaliplatin-induced peripheral neuropathy (OIPN), a relatively common and debilitating side effect of chemotherapy. In each patient cohort, rates of neuropathy were dramatically lower than those typically seen in historical benchmarks for FOLFOX alone:

These results were supported by feedback from trial investigators, many of whom noted the unexpectedly low incidence of neuropathy in their patients treated with BOLD-100 — particularly those who were heavily pretreated and/or receiving FOLFOX again where one would expect both a high incidence and severity of neuropathy.

To better understand this effect, Bold Therapeutics developed a preclinical rat model of cold allodynia, a common symptom of OIPN. Rats were treated with either high or low doses of oxaliplatin and then given BOLD-100 every three days for 17 days. The results confirmed the clinical findings: rats receiving BOLD-100 showed significantly less neuropathic pain. Once treatment with BOLD-100 stopped, neuropathy symptoms rapidly returned, further supporting its protective effect.

These findings prompted further efforts to elucidate BOLD-100’s neuroprotective mechanism and investigation into whether BOLD-100 can prevent or reduce neuropathy from other neuropathy-inducing agents, such as the taxanes. BOLD-100’s unique ability to both directly impact cancer while simultaneously preventing or reducing OIPN could redefine first-line treatment by helping patients:

Improve overall outcomes through direct anti-cancer activity and completing full chemotherapy cycles.
Stay on therapy longer without dose reductions or early discontinuation.
Experience less pain, tingling, and sensory issues that impact daily life.
Avoid long-term nerve damage that can persist long after treatment ends.
Ongoing Clinical Study and Future Outlook

Bold Therapeutics is currently enrolling FOLFOX-naïve second-line mCRC patients in a multinational randomized clinical study comparing FOLFOX vs. FOLFOX + BOLD-100 in various efficacy, safety, and quality-of-life endpoints. Bold Therapeutics anticipates this trial will further demonstrate BOLD-100’s potential as a transformative therapy in early-line colorectal cancer, biliary tract cancer and other solid tumor indications.

Bold Therapeutics invites investors, strategic partners, clinical investigators and key opinion leaders to connect with the team at AACR (Free AACR Whitepaper) to discuss these latest findings, the company’s ongoing clinical development plans, and potential strategic collaborations.

On April 30, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported that preclinical data will be presented on its proprietary RSwitch technology, which enables the fine-tuning of transgene levels in gene and cell therapy applications, at the American Society of Gene And Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, which will be held from May 13 -17th, 2025, in New Orleans, LA (Press release, Rgenta Therapeutics, APR 30, 2025, View Source [SID1234652396]).

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Title: RSwitch Enabled Gene Therapy to Fine Tune Frataxin Expression for the Treatment of Friedrich’s Ataxia
Authors: Samuel A. Hasson, Jon Dempersmier, Mariam Elhawary, Diane Hamann, Ian McLachlan, Chris Yates, Travis Wager, and Simon Xi
Session date and time: Wednesday, May 14, 2025, 5:30 – 7:00 PM CT
Location: Poster Hall I2
Poster ID: AMA251

About RSwitch
RSwitch is a proprietary regulatable gene therapy system that enables oral, small molecule drug control of transgene levels in gene and cell therapy applications. RSwitch encodes a "dimmer" switch that makes the expression of transgene dependent on the administration of an oral small molecule drug that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein. Rgenta has demonstrated the RSwitch system’s feasibility in vitro and in vivo, achieving dose-dependent expression of reporter transgenes following small molecule administration. RSwitch technology offers versatile control across multiple gene and cell therapy applications, and the company is actively exploring strategic partnerships.

On April 30, 2025 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the Company will participate in the BofA Securities 2025 Healthcare Conference. Company management will participate in a fireside chat on Wednesday, May 14, 2025, at 10:00 a.m. PT / 1:00 p.m. ET / 6:00 p.m. IST (Press release, Jazz Pharmaceuticals, APR 30, 2025, View Source [SID1234652395]).

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An audio webcast of the fireside chat will be available via the Investors section of the Jazz Pharmaceuticals website at View Source A replay of the webcast will be archived on the website for 30 days.

On April 30, 2025 Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that seek to overcome tumor immune evasion and drug resistance, reported that a poster presentation titled "Final analysis of the randomized Phase 2 cohort of CM24 with nivolumab and chemotherapy in pancreatic cancer & potential serum biomarkers" is being presented during the session "Liquid Biopsy: Circulating Nucleic Acids 4 / Predictive Biomarkers 1" at the Annual Meeting of the American Association of Cancer Research (AACR 2025) on Wednesday, April 30, 2025 (Press release, Purple Biotech, APR 30, 2025, View Source;id=347386&p=2372871&I=1206939-c7Z3G6f3m8 [SID1234652394]).

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"These statistically significant biomarker results, with up to a 90% reduction in risk of death over the control group, are highly encouraging and we believe warrant a biomarker-driven Phase 2b study," stated Purple Biotech CEO Gil Efron. "The new, previously unpublished data presented at AACR (Free AACR Whitepaper) demonstrate significant overall survival (OS) and progression-free survival (PFS) benefit for patients meeting the criteria of either serum CEACAM1 or tumor CEACAM1 biomarkers. With these final data reported, we believe CM24 could potentially be positioned as a therapy targeting CEACAM1 in cancers with large unmet needs."

At AACR (Free AACR Whitepaper) 2025, Purple Biotech presented final data from its randomized, controlled, open-label, multicenter Phase 2 study (NCT04731467), which established proof of concept in a biomarker-enriched subgroup of 31 patients with advanced/metastatic pancreatic ductal adenocarcinoma (PDAC) who were post-first line therapy, and compared treatment with CM24 + nivolumab + Nal-IRI/5FU/LV against treatment with Nal-IRI/5FU/LV alone.

The following is a summary of the findings.

The combination of CM24, nivolumab and Nal-IRI/5FU/LV chemotherapy was well tolerated and demonstrated quantitative improvement in all efficacy parameters, including OS, PFS, objective response rate (ORR), disease control rate (DCR) and CA19-9, in previously treated PDAC patients.

Without consideration of biomarkers, a consistent improvement in the treatment arm compared to the control arm was observed, as follows:

Prolongation of 2.4 months in OS HR=0.81 (p-0.575) and prolongation of 1.9 months in PFS HR=0.75 (p=0.463) vs. control arm
Higher ORR in experimental arm vs control arm (25% vs 6.7%)
Higher DCR in experimental arm vs control arm (62.5% vs 46.7%)
Consistent and continuous decrease in CA19-9 was observed in experimental arm vs control arm increase
Biomarker results included the following:

A statistically significant benefit to patients with defined pre-treatment ranges of serum CEACAM1 or tumor CEACAM1 expression (H score) compared to the control arm (representing a subgroup of 52% of patients):

Subgroup analyses of 16/31 patients meeting the criteria of CEACAM1+Tumor cell H score 115-275 or serum CEACAM1 levels 6K-15K pg/mL
78% reduction in risk of death (HR 0.22, 95% CI 0.07-0.7, p=0.006) and 95% reduction in the risk of progression or death (HR 0.05, 95% CI 0.01-0.44, p=0.0003)
Prolongation of 3.7 months in OS and 2.9 months in PFS
Statistically significant results in patients with defined pre-treatment serum CEACAM1 or myeloperoxidase (MPO) levels (representing a subgroup of 80% of patients):

Subgroup analyses for 24/30 patients meeting the criteria of MPO levels 200-600 ng/mL or serum CEACAM1 levels 6K-15K pg/mL
61% reduction in risk of death (HR = 0.39, 95% CI 0.16-0.98, p=0.039) and 72% reduction in the risk of progression or death (HR=0.28, 95% CI 0.11-0.73, p=0.006)
Prolongation of 2.4 months in OS and 2.2 months in median PFS
A statistically significant benefit for CM24-nivolumab treatment in patients with high CEACAM1+Tumor cell H score and low PD-L1 CPS (representing a subgroup of 38% of patients):

Subgroup analyses of 10/26 patients meeting the criteria of high CEACAM1+Tumor cell H score 115 and Low CPS 1 (
90% reduction in risk of death (HR = 0.1, 95% CI 0.01-0.89, p=0.013) and 81% reduction in the risk of progression or death (HR = 0.19, 95% CI 0.04-1.02, p=0.033)
Prolongation of 4 months in OS and 2 months in PFS
"The identification of CEACAM1 as a potential biomarker, both in serum and at the tumor, corresponds with the multi-faceted function of CEACAM1, as part of the neutrophil extracellular trap (NET) structure affecting NET-related tumor immune evasion, metastasis and other cancer-associated complications, such as thrombosis, at the level of the whole body, as well as modulation of the tumor microenvironment (TME)," stated Purple Biotech VP of Research and Development, Dr. Hadas Reuveni. "Targeting CEACAM1 by CM24 suggests a potential new approach that may address tumor-associated mechanisms affecting the patient at the levels of the tumor, the TME and the whole body. The biomarker data accumulated in this clinical study may help us to direct the treatment of patients who might have a higher chance to benefit from the treatment and could expand our understanding of CEACAM1 and NETs in cancer biology."

Final conclusions from the study:

The combination of CM24, nivolumab, and Nal-IRI/5FU/LV chemotherapy was well tolerated and
demonstrated quantitative improvement in all efficacy parameters, including OS, PFS, ORR and CA19-9, in previously treated PDAC patients.

Based on post-hoc analyses – serum CEACAM1 and MPO, a NET marker, are demonstrated as potential predictive biomarkers for CM24-based therapy, consistent with its mechanism of action (MoA) in targeting CEACAM1 to modulate immune evasion and NET activities.
Results imply that both serum and tumor CEACAM1 levels are potential predictive biomarkers for CM24 based therapy, suggesting multifaceted MoA and the crosstalk of the tumor with the TME and the whole body.
Improved outcome in patients with high tumor CEACAM1 expression and low PD-L1 CPS further support the mechanistic rationale of the CM24/nivolumab combination and highlight its potential in disease settings where immuno-oncology is less effective.
"The design of the randomized trial enabled us to evaluate the benefit of CM24 and nivolumab in combination with standard of care chemotherapy and to analyze potential biomarker data to better prepare for the next study." stated Purple Biotech Head of Clinical and Regulatory Affairs, Dr. Michael Schickler. "We currently expect the design of the next Phase 2b study to include an additional group testing CM24 alone in combination with standard of care with patients selected based on the identified biomarkers, potentially in indications such as gastric and/or biliary tract cancer, in addition to PDAC."

The poster will be available at the Publication section on Purple Biotech’s website following its presentation at the conference.

On April 30, 2025 Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE: 7JO), a biopharmaceutical company advancing next-generation cancer therapies through artificial intelligence (AI)-powered drug discovery reported the financial results for its fourth quarter and fiscal year ending December 31, 2024 and provided a corporate update (Press release, Rakovina Therapeutics, APR 30, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-inc-announces-2024-financial-results-and-provides-corporate-update [SID1234652392]).

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2024 Highlights and Recent Developments

On April 29, 2025, we announced the intention to appoint Yevgeniy Meshcherekov and David Kideckel to the Company’s board of directors, subject to receiving approval from the TSX Venture Exchange. The Company also announces that Michael Liggett has retired from the Board. Subject to TSXV approval, Mr. Meshcherekov will replace Mr. Liggett as the chair of the audit committee of the Board
On April 28 & 29, 2025, we presented pre-clinical data related to the development of our kt-2000 and kt-5000 programs at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting. These data demonstrate that select candidates derived from our AI collaborations may meet our target product profile for a PARP-1 selective lead candidate (kt-2000) and for an ATR-inhibitor (kt-5000).
On March 12, 2025, we announced that we had received the first synthesized batch of AI-generated ATR inhibitor compounds developed in collaboration with Variational AI.
On January 30, 2025, we announced the listing of common shares on the Frankfurt Stock Exchange (FSE) under the ticker symbol "7J0".
On January 13, 2025, we announced the successful achievement of a shortlist of AI-generated molecules targeting ATR (Ataxia Telangiectasia and Rad3-related protein) with specific designs for central nervous system (CNS) penetration.
On January 6, 2025, we announced the receipt of initial synthesized drug candidates: AI-generated PARP-inhibitor compounds for in vitro and in vivo validation in our laboratories.
On December 13, 2024, we announced the closing of an oversubscribed $3.0 million private placement offering (the "Private Placement"). The Private Placement consists of 50,000,000 units (the "Units") at a price of $0.06 per Unit. Each Unit consists of one common share of the Company (each, a "Common Share") and one Common Share purchase warrant (each, a "Warrant"). Each Warrant entitles the holder thereof to subscribe for and purchase one Common Share of the Company for a period of 24 months from the date of issue at a price of $0.10 per Common Share. Rakovina retains the right to accelerate the Warrant exercise period if, upon written notice to the holder, the 20-day volume-weighted average price of its Common Shares exceeds $0.30.
On November 22, 2024, we delivered a poster presentation highlighting initial results of our Deep Docking and generative Artificial Intelligence (AI) PARP-inhibitor program at the Neuro-Oncology Annual Meeting in Houston, Texas.
On October 25, 2024, we presented our research on kt-3283, our lead small-molecule bifunctional inhibitor of PARP and HDAC enzymes at the 36th EORTC-NCI-AARC Symposium in Barcelona, Spain.
On October 23, 2024, we announced the receipt of initial results from the Company’s Deep Docking AI partnership following the evaluation of billions of molecular structures. This process resulted in a short-list of drug candidates that have been optimized to a specific target-product profile.
On September 17, 2024, we announced a research collaboration with Variational AI to employ the proprietary Enki AI platform to identify and develop novel small-molecule therapies against select DDR kinase targets for the treatment of cancer.
On July 26, 2024, we announced the final closing of an over-subscribed non-brokered private placement financing of units priced at $0.10 per unit with each unit consisting of one common share and one common share purchase warrant with an exercise price of $0.20 per warrant and a term of three years for gross proceeds to the Company of $2 million (the "2024 Private Placement"). Each Warrant entitles the holder thereof to subscribe for and purchase one Common Share at a purchase price of $0.20 for a period of three years from the date of issuance. If the closing price for the Common Shares on the TSX Venture Exchange is $0.25 or greater for five consecutive trading days, the Company will have the right to accelerate the expiry date of the Warrants, upon written notice to the holder, to the date that is 30 days following such notice.
On May 8, 2024, we announced the expansion of our collaborations with the University of British Columbia ("UBC") and our medicinal chemistry partner, Pharma Inventor Inc. to support the Company’s AI drug discovery initiatives.
On March 27, 2024, we announced a collaboration agreement with Dr. Artem Cherkasov granting Rakovina exclusive access to the proprietary Deep Docking AI Platform for DNA-damage response targets. We are using the Deep Docking platform to quickly analyze billions of molecular structures to evaluate their potential as targeted cancer drugs.
Summary Financial Results for the fourth quarter and year ended December 31, 2024

At December 31, 2024, the Company had positive working capital of approximately $321,442.

For the three- and twelve-months ending December 31, 2024, the Company reported a net loss of $1,483,988 and $4,072,618, respectively. Research and development operating expenses were $744,533 and $2,341,600 for the three and twelve months ended December 31, 2024, respectively. General and administrative expenses were $650,268 and $1,446,451 for the three- and twelve-months ending December 31, 2024, respectively. Total cash operating expenses related to research and development and general and administrative expenses for the three and twelve months ended December 31, 2024, were $3,165,554 and $1,243,517 respectively.

Selected Financial Information As at December 31, 2024 $
Cash & cash equivalents 1,312,743
Working capital 321,442
Intangible assets 3,977,473
Total Assets 6,240,920
Total liabilities 1,942,005
Deficit (14,997,929)
Total equity 4,298,915
Statements of net loss and comprehensive loss data: For the three months ended December 31, 2024 $ For the year ended December 31, 2024 $
Research & Development 744,533 2,341,600
General and administrative 650,268 1,446,451
Net loss and comprehensive loss (1,483,988) (2,612,925)
Basic and diluted income (loss) per share (0.01) (0.05)
Operating cash burn 1,243,517 3,165,554
Weighted average shares outstanding 100,089,471 82,549,477
Rakovina Therapeutics’ financial statements as filed with SEDAR can be accessed from the Company’s website at: View Source