On May 11, 2023 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company developing Stealth Editors to perform in vivo gene editing without triggering the immune system, reported its financial results for the three-month period ended March 31, 2023 and other recent developments (Press release, NeuBase Therapeutics, MAY 11, 2023, View Source [SID1234631525]).

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"To date in 2023, we have made steady progress advancing the development of our gene editing application based on our PATrOL platform. One of the main drivers behind the initial success of this application area, and speed at which we are moving forward, is the ability to leverage our extensive experience in peptide nucleic acids ("PNAs"), the chemistry which forms the core of our platform. PNAs have been shown to engage the double-stranded human genome due to their high binding affinity and exquisite sequence selectivity. This chemistry would allow us to tag a locus in the genome that harbors a mutation with a simple synthetic compound and recruit the cell’s own machinery to repair the mutation. This is the approach behind our in vivo gene editing platform," stated Dietrich A. Stephan, Ph.D., Founder and Chief Executive Officer of NeuBase. "We believe our Stealth Editors can potentially address up to 90% of disease-causing mutations, compared to the estimated approximately 20% addressed by base editing platforms currently in development. In addition, our approach to gene editing offers several benefits that differentiate us from CRISPR/Cas editors, base editors, and prime editors. For example, Stealth Editors are non-immunogenic and have exquisite fidelity, maximizing the ability to re-dose should durability wane over time, which is a situation we have seen with gene therapy approaches in the past."

"Throughout the remainder of the year we look forward to presenting initial ex vivo and in vivo editing results against high-value genetic mutations, together with associated performance metrics, such as fidelity and efficiency. This includes preclinical data in two oral presentations the NeuBase team will provide at the ASGCT (Free ASGCT Whitepaper) 26th Annual Meeting taking place next week. We plan to follow up the presentations with an investor and analyst conference call on May 22, 2023 to discuss these data," concluded Dr. Stephan.

First Quarter of 2023 and Recent Operating Highlights

Gene Editing Program:

The Company is advancing development of the differentiated gene editing capabilities of its PATrOL platform and development of Stealth Editors.
Two abstracts accepted for oral presentations at the ASGCT (Free ASGCT Whitepaper) 26th Annual Meeting, which is being held in Los Angeles, CA and virtually on May 16-20, 2023. Management plans to hold an investor and analyst conference call on May 22, 2023.

Details of the gene editing pipeline expected to be provided during 2023.
Gene Editing Research Agreements:
The Company’s previously announced collaboration with a global healthcare company to evaluate the PATrOL platform for three monogenic genetic diseases remains on track. The global healthcare company will have the exclusive opportunity, subject to certain terms and conditions, to license and develop the drug candidates created under this research evaluation agreement.
Engaged in discussions with other healthcare companies on the potential for additional research agreements.
Gene Silencing Pipeline Collaborations:
NeuBase continued active pursuit of collaborative initiatives, including partnerships, for the Company’s myotonic dystrophy type 1, Huntington’s disease, and KRAS (G12D and G12V) programs. The Company currently expects that any collaborations that could result from these discussions would be announced in the second half of 2023.
Financial Results for the Quarter Ended March 31, 2023

As of March 31, 2023, the Company had cash and cash equivalents of approximately $13.8 million, compared with approximately $17.4 million as of December 31, 2022.
NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the second quarter of 2024.

For the quarter ended March 31, 2023, the Company reported a net loss of approximately $4.1 million, or a net loss of $0.12 per share, compared with a net loss of approximately $9.9 million, or a net loss of $0.30 per share, for the same period last year.

For the quarter ended March 31, 2023, total operating expenses were approximately $4.2 million, consisting of approximately $2.9 million in general and administrative expenses and $1.3 million in research and development expenses. This compares with total operating expenses of approximately $9.9 million for the same period last year, consisting of approximately $3.1 million in general and administrative expenses and $6.8 million in research and development expenses.

On April 21, 2023, the Company’s Board of Directors approved a change in its fiscal year from the twelve months beginning October 1 and ending September 30 to the twelve months beginning January 1 and ending December 31, which will be effective for the year ending December 31, 2023.

On May 11, 2023 Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, reported business highlights and financial results for the quarter ended March 31, 2023 (Press release, Monte Rosa Therapeutics, MAY 11, 2023, View Source [SID1234631524]).

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"During the first quarter, we made significant progress with our Phase 1/2 clinical trial of MRT-2359 in MYC-driven solid tumors. This trial continues to attract top oncologists and academic centers, and patient enrollment is on track for us to have initial data to share in the second half of the year. We remain excited about the prospect of bringing a potential therapeutic option to a range of indications driven by MYC, one of the most frequently altered families of oncogenes," said Markus Warmuth, M.D., CEO of Monte Rosa. "Furthermore, we expect to declare a development candidate in our VAV1 program in the second quarter. This milestone will mark a significant advancement in our pipeline of MGD-based medicines in the field of immunology and inflammation. We believe the underlying biology and our preclinical data support the potential of our VAV1-directed MGD to provide clinical benefit in multiple autoimmune diseases. Our continued progress with our pipeline speaks to the power of our QuEEN discovery engine and our position as a leading molecular glue degrader company. Backed by a strong balance sheet and a sharp focus on developing impactful medicines, we look forward to continuing to further the development of our molecular glue therapeutics."

FIRST QUARTER 2023 AND RECENT HIGHLIGHTS

•
Presented preclinical data highlighting the preferential activity of MRT-2359, an orally bioavailable GSPT1-directed MGD, in MYC-driven tumor cells at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023 in Orlando, FL
•
Appointed Jan Skvarka, Ph.D., MBA, an accomplished biopharmaceutical executive with extensive operational, strategic, and financial expertise to the Company’s Board of Directors

UPCOMING MILESTONES

•
On track to announce initial clinical data, including pharmacokinetic (PK), pharmacodynamic (PD), safety, and available initial efficacy data from the Phase 1 part of the ongoing Phase 1/2 clinical trial evaluating MRT-2359 in MYC-driven tumors, including non-small cell lung cancer, small cell lung cancer, and other MYC-driven tumors, in the second half of 2023
•
On track to advance multiple preclinical programs to development candidates in immunology, inflammation, and oncology, including declaring a VAV1 development candidate in the second quarter of 2023

FIRST QUARTER 2023 FINANCIAL RESULTS

Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2023 were $26.8 million, compared to $17.9 million for the first quarter of 2022. These increases were driven by the successful achievement of key milestones in our R&D organization, including the advancement of MRT-2359 in the clinic, the progression of our preclinical pipeline in lead optimization, and the continued development of the Company’s QuEEN platform for discovery efforts. The increase in R&D expenses was driven by increased headcount and laboratory-related expenses to achieve these milestones. Non-cash stock-based compensation constituted $2.1 million of R&D expenses for Q1 2023, compared to $1.2 million in the same period in 2022.

General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2023 were $7.5 million compared to $6.4 million for the first quarter of 2022. The increase in G&A expenses was a result of increased headcount and expenses in support of the company’s growth and operations as a public company. G&A expenses included non-cash stock-based compensation of $1.8 million for the first quarter of 2023, compared to $1.1 million for the same period in 2022.

Net Loss: Net loss for the first quarter of 2023 was $32 million, compared to $23.9 million for the first quarter of 2022.

Cash Position and Financial Guidance: Cash, cash equivalents, restricted cash, and marketable securities as of March 31, 2023, were $237 million, compared to cash, cash equivalents, and restricted cash of $268.1 million as of December 31, 2022. The decrease of $31.1 million was primarily related to cash used to fund operations in the first quarter, including a seasonal reduction in accrued expenses. The company expects that its cash and cash equivalents will be sufficient to fund currently planned operations and capital expenditures into 2025.

About MRT-2359

MRT-2359 is a potent, selective and orally bioavailable molecular glue degrader (MGD) that induces the interaction between the E3 ubiquitin ligase component cereblon and the translation termination factor GSPT1, leading to the targeted degradation of GSPT1 protein. The MYC transcription factors (c‑MYC, L-MYC and N-MYC) are well-established drivers of human cancers that maintain high levels of protein translation, which is critical for uncontrolled cell proliferation and tumor growth. Preclinical studies have shown that this addiction to MYC-induced protein translation creates a dependency on GSPT1. By inducing degradation of GSPT1, MRT-2359 exploits this vulnerability, disrupting the protein synthesis machinery, leading to anti-tumor activity in MYC-driven tumors.

About VAV1

VAV1, a Rho-family guanine nucleotide exchange factor, is a key signaling protein downstream of both the T and B cell receptors, whose expression is restricted to blood and immune cells, including T and B cells. Preclinical studies have shown that targeted degradation of VAV1 protein via an MGD modulates both T- and B-cell receptor-mediated activity in vitro and in vivo, as displayed by a significant decrease in cytokine secretion, as well as activity in preclinical models of immune diseases. VAV1-directed MGDs have the potential to provide therapeutic benefits in multiple autoimmune indications, such as multiple sclerosis, rheumatoid arthritis, and dermatological disorders.

On May 11, 2023 Monopar Therapeutics Inc. (Monopar or the Company) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported first quarter 2023 financial results and summarized recent developments (Press release, Monopar Therapeutics, MAY 11, 2023, View Source [SID1234631523]).

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Recent Developments

Camsirubicin – Phase 1b Dose-Escalation Trial, Currently Enrolling Fifth Dose-Level Cohort Phase 1b data to date show an improvement in median progression free survival in patients with advanced soft tissue sarcoma (ASTS) from what was observed in the prior camsirubicin Phase 2 trial (265 mg/m2). This is supportive of our dose-response hypothesis with camsirubicin. Additionally, one of the three patients in the 520 mg/m2 dose-level cohort recently went from having what was initially determined to be an unresectable cancer to, after several cycles of camsirubicin treatment and a corresponding 21% reduction in tumor dimensions, being determined to be resectable. This changed the course of treatment for this patient, who recently did undergo surgical resection of the cancer.

Monopar is currently enrolling patients into the fifth dose-level cohort (650 mg/m2), which is nearly 2.5x the highest dose evaluated in any prior camsirubicin clinical trial (265mg/m2).

To date, no drug-related cardiotoxicity has been observed with camsirubicin treatment as evaluated by the industry standard left ventricular ejection fraction (LVEF). This compares favorably to the well-documented dose-restricting cardiotoxicity experienced with doxorubicin, the current first-line treatment for ASTS.

75% of camsirubicin patients in this trial have experienced no hair loss. Of the 25% with any hair loss, only 8% experienced >50% hair loss and only 17% experienced low grade hair loss. This compares favorably to the approximately 50% of doxorubicin treated patients in recent ASTS clinical trials reporting some amount of hair loss, with the majority of these patients experiencing >50% hair loss.

Only 8% of camsirubicin patients in the trial have experienced low grade, mild oral mucositis. This compares favorably to the roughly 35-40% of doxorubicin treated patients in recent ASTS clinical trials that experienced mild-to-severe oral mucositis.

MNPR-101 for Radiopharmaceutical Use – Promising Preclinical Studies Support FIH Study

Based on promising preclinical imaging results with MNPR-101-Zr showing high uptake across multiple tumor types, and with preclinical therapeutic efficacy and biodistribution studies utilizing the radioisotopes Ac-225 and Lu-177, Monopar and its collaborator, NorthStar Medical Radioisotopes, committed to additional funding with the aim of initiating a first-in-human (FIH) imaging study with MNPR-101-Zr as early as end of this year.

MNPR-101-Zr is a zirconium-89 labeled version of MNPR-101 (a highly selective antibody against the urokinase plasminogen activator receptor, also known as uPAR). Positron emission tomography (PET) imaging of preclinical mouse models for triple-negative breast, colorectal, and pancreatic tumors displayed high and selective uptake of MNPR-101-Zr in these uPAR-expressing tumors.

Preclinical triple negative breast cancer mouse model studies with Ac-225 and Lu-177 radiolabeled MNPR-101 showed a promising dose-dependent-anti-cancer-effect and favorable biodistribution profile. The imaging and therapeutic preclinical results to date demonstrate the potential utility of MNPR-101 as a precision targeting agent for both imaging and treatment in multiple cancer indications.

MNPR-202 – Promising Preclinical Data Ignites Further Research

MNPR-202 is designed to retain the same potentially non-cardiotoxic backbone as camsirubicin but is modified at other positions which may enable it to work in certain cancers that are resistant to camsirubicin and doxorubicin.

Monopar’s collaborator at the National University of Singapore, Cancer Science Institute, has reported data from blood cancer preclinical studies showing that MNPR-202:
has a similar cytotoxic potency to doxorubicin generates increased DNA damage in the cancer cells compared to doxorubicin has a unique immune activation profile versus doxorubicin demonstrates increased apoptosis (programmed cell death) compared to doxorubicin causes a distinct set of genes to be upregulated and downregulated versus doxorubicin and may also be superior to doxorubicin in certain combination treatment regimens.

A combination drug screen with 183 compounds was performed, revealing distinct differences in the synergy profile between doxorubicin versus MNPR-202 when used along with other compounds. For example, MNPR-202 demonstrated a more favorable synergy profile with the experimental anti-cancer agent volasertib compared to doxorubicin.

Validive Clinical Update

On March 27, 2023, the Company discontinued its Validive Phase 2b/3 VOICE trial based upon its independent Data Safety Monitoring Board’s determination that the trial did not meet the pre-defined threshold for efficacy of a 15% absolute difference in severe oral mucositis prevention between Validive and placebo. Other than clinical site close-out related expenses to be incurred in Q2 2023, the Company will not incur any license or royalty obligations and is not anticipating any significant expenses beyond Q2 2023 related to Validive.

Results for the First Quarter Ended March 31, 2023, Compared to the First Quarter Ended March 31, 2022

Cash and Net Loss

Cash, cash equivalents and short-term investments as of March 31, 2023, were $11.7 million. Monopar expects that its current funds will be sufficient for Monopar to obtain topline results from its ongoing open-label Phase 1b camsirubicin clinical trial as planned by the end of 2023 (but this may not be the case if camsirubicin reaches even higher dose levels than anticipated and topline results are deferred as dosing continues beyond 2023), advance the Company’s MNPR-101 radiopharmaceutical program into its first in human clinical trial and close out Monopar’s terminated Validive Phase 2b/3 (VOICE) clinical program. The Company estimates its cash, cash equivalents and short-term investments will fund the Company’s planned operations at least through June 2024. Monopar will require additional funding to advance its clinical and preclinical programs beyond that and anticipates seeking to raise additional capital within the next 12 months to fund its future operations.

Net loss for the first quarter of 2023 was $2.4 million or $0.19 per share compared to net loss of $2.5 million or $0.19 per share for the first quarter of 2022.

Research and Development (R&D) Expenses

R&D expenses for the first quarter of 2023 were $1,653,000 compared to $1,678,000 for the first quarter of 2022. This decrease of $25,000 was primarily due to a decrease of $120,000 in R&D personnel costs, partially offset by an increase of $79,000 in Validive and camsirubicin clinical trial-related and clinical material manufacturing-related expenses.

General and Administrative (G&A) Expenses

G&A expenses for the first quarter of 2023 were $872,000 compared to $779,000 for the first quarter of 2022. This increase of $93,000 was primarily due to (1) an increase in G&A salaries and benefits and (2) an increase in accounting and audit fees.

On May 11, 2023 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a growing pipeline, including Phase 2 clinical programs, for hard-to-treat tumors and viruses, reported its financial results for the quarter ended March 31, 2023 (Press release, Moleculin, MAY 11, 2023, View Source [SID1234631522]). As previously announced, the Company will host a conference call and live audio webcast, today, Thursday, May 11, 2023, at 8:30 AM ET (details below).

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"Through our team’s continued operational and clinical execution throughout 2022, we have set the stage for numerous clinical data readouts in 2023," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We believe that with the progress we’ve made and the milestones ahead, we are well positioned to unlock significant potential for all stakeholders, and most importantly, address unmet needs for people with highly resistant cancers and viruses."

Recent Highlights

Successfully completed the first cohort in that Phase 1b portion of its ongoing Phase 1b/2 trial evaluating Annamycin in combination with Cytarabine (Ara-C) for the treatment of AML (MB-106).
Announced presentation of positive pharmacokinetics and tissue-organ distribution data demonstrating high anti-tumor activity of Annamycin in preclinical cancer models in a poster titled, Exploration of Annamycin Organotropism to Target Primary and Metastatic Liver Cancers, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023.
Summary of Financial Results for the First Quarter 2023

Research and development (R&D) expense was $5.7 million and $4.6 million for the three months ended March 31, 2023 and 2022, respectively. The increase of $1.1 million is mainly related to the WPD sublicense termination.

General and administrative (G&A) expense was $2.6 million and $2.4 million for the three months ended March 31, 2023 and 2022, respectively. The increase of $0.2 million is mainly related to an increase in regulatory and legal services, and consulting & advisory fees.

As of March 31, 2023, the Company had cash and cash equivalents of $37.3 million and believes that this cash is sufficient to meet its projected operating requirements into the third quarter of 2024.

Conference Call and Webcast

Moleculin management will host its quarterly conference call and live audio webcast for investors, analysts, and other interested parties today, Thursday, May 11, 2023, at 8:30 AM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.

On May 11, 2023 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported its interim management statement for the quarter ending March 31, 2023 (Press release, Molecular Partners, MAY 11, 2023, View Source [SID1234631521]).

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"This quarter we made advances across our portfolio, including two clinical-stage oncology programs, MP0317 for solid tumors and MP0533 for AML. MP0317 is now recruiting patients at the top doses planned. We anticipate analyzing these data and working with potential partners to determine the best combinations and indications for the program. MP0533 recruitment has commenced, dose escalation is ongoing and progressing seamlessly. We look forward to the progress in this study and sharing initial data from the trial later this year," said Patrick Amstutz, Ph.D., Molecular Partners’ Chief Executive Officer. "We are also progressing well with our Radio-DARPin platform, comprising both in-house and Novartis-partnered programs, presenting at two leading scientific conferences, documenting the growing data in support of our thesis that RDTs have the potential to overcome many of the current limitations in the radiotherapy field."

Financial and Business Outlook
For the full year 2023, at constant exchange rates, the Company expects total expenses of CHF 70 – 80 million, of which approximately CHF 9 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciation. This guidance does not include any potential receipts from R&D partnerships.

With CHF 232.4 million in cash and short-term time deposits and no debt as of March 31, 2023, the Company expects to be funded into 2026, excluding any potential receipts from R&D partners.

Research & Development Highlights:

MP0317
In November 2022, Molecular Partners presented early results from the ongoing Phase 1 trial of MP0317, the Company’s DARPin candidate targeting fibroblast activation protein (FAP) and CD40, for the treatment of solid tumors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting. These data demonstrated the first clinical observation of tumor localized CD40 activation provided by MP0317. The candidate was also seen to be safe and well tolerated. MP0317 is designed to resolve the historical limitations of systemic CD40 agonists by activating immune cells within the tumor microenvironment through the simultaneous binding of the immune stimulator CD40 and FAP, a protein highly expressed within tumors. The dose escalation of the Phase 1 study remains on track.

Additionally, a clinical update will be provided at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in Chicago in early June:

Abstract Title: Phase I study of MP0317, a FAP-dependent DARPin, for tumor-localized CD40 activation in patients with advanced solid tumors.

Session Title: Developmental Therapeutics—Immunotherapy
Abstract Number for Publication: 2584
Session Date and Time: 6/3/2023, 8:00 AM-11:00 AM

MP0533
In January 2023, the first patient was dosed and recruiting and dose escalation is going according to plan in the Phase 1 study of MP0533, a novel trispecific T-cell engager for the treatment of Acute Myeloid Leukemia (AML). The first clinical results from this trial are expected by the fourth quarter of 2023. MP0533 engages CD3 on T-cells while binding up to three tumor-associated antigens (TAAs) CD33, CD70, and CD123 on AML cells. By modulating the affinity to each TAA, Molecular Partners designed MP0533 to induce T-cell-mediated killing preferentially when the cancer cells express two or three of the TAAs. This avidity-driven T-cell activation ensures preferential killing of AML cells, which consistently express two or three of the target antigens. At the same time, it is designed to reduce the damage to healthy cells (which tend to express only one of the target antigens), a recurrent issue with other T-cell engagers in AML.

In an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2022, Molecular Partners presented preclinical results showing MP0533 can induce preferential killing of cells expressing two or three tumor-associated antigens (TAAs) compared to cells expressing a single TAA. MP0533 was demonstrated to activate T-cells and destroy AML cells in samples from newly diagnosed and previously treated AML patients with different TAA expressions. Humanized mouse models confirmed MP0533’s ability to activate intra-tumoral T-cells and control tumor growth. The research also showed that MP0533 was able to directly target and kill leukemic stem cells (LSCs), while sparing a variety of healthy cells including hematopoietic stem cells. The unique preclinical safety profile of MP0533 was further supported by several other parameters including a lower level of cytokine release relative to benchmark mono-targeted T-cell engagers, both in vitro in a whole blood assay and in vivo in the humanized mouse AML models.

Radio DARPin Therapy Platform
Molecular Partners has continued to progress its Radio DARPin Therapy (RDT) platform by reducing the kidney uptake of DARPin radio conjugates to overcome nephrotoxicity (toxicity in the kidney), the key limitation of small protein-based radiotherapies. In 2023, the Company presented positive preclinical data from its RDT platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting and the 12th International Symposium on Targeted Alpha Therapy (TAT 12) supporting its potential to significantly reduce accumulation in the kidney, a common challenge with small protein-based delivery vectors. In preclinical models, the surface engineering did not affect tumor uptake or uptake in other healthy organs and in combination with another kidney reduction strategy provided a cumulative benefit.

The Company also selected tumor-associated protein Delta-like ligand 3 (DLL3) as the first target of its proprietary RDT programs. Expression of DLL3 is low in healthy tissue but significantly increased in certain tumor types, providing an opportunity for selective targeting through the high affinity and specificity offered by DARPins. These attributes, along with their small size, suggest that DARPins represent ideal delivery vectors for therapeutic radionuclides to efficiently target cancer cells with minimal systemic side effects. Molecular Partners is developing RDT candidates as part of its proprietary pipeline as well as in its collaboration with Novartis in the radioligand area.

Virology
Molecular Partners and Novartis signed a non-binding letter of intent to negotiate a Research Framework Agreement with a primary focus on emerging infectious global health threats.

Ophthalmology
In November 2021, Molecular Partners regained global development and commercial rights to abicipar for the treatment of neovascular age-related macular degeneration (nAMD) and Diabetic Macular Edema (DME). Abicipar completed two positive Phase 3 studies, CEDAR and SEQUOIA, which supported the non-inferior efficacy of its quarterly dosing regimen compared to monthly ranibizumab.

The Company continues to evaluate potential business opportunities for abicipar outside of internal development at Molecular Partners.

Financial Calendar

24 August 2023 – Publication of Half-year Results 2023 (unaudited)

26 October 2023 – Interim Management Statement Q3 2023