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

On May 11, 2023 MEI Pharma, Inc. (Nasdaq: MEIP), a clinical-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for the quarter ended March 31, 2023, and highlighted recent corporate events (Press release, MEI Pharma, MAY 11, 2023, View Source [SID1234631520]).

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"We are making steady progress in advancing both of our clinical-stage pipeline programs: voruciclib for hematologic malignancies and ME-344 for relapsed colorectal cancer, both expected to report data by around the end of the calendar year," said Dan Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "With the clinical-data expected from these two programs around year-end, funds to support operations for at least two years based upon our current development plans, and the opportunity to further strengthen our value proposition via the pending merger with Infinity Pharmaceuticals, we are well positioned to deliver progress in our mission to deliver improved benefits to patients with cancer, as well as value to our shareholders."

Third Quarter Fiscal Year 2023 and Recent Developments

•
Kyowa Kirin has been evaluating whether to continue developing zandelisib in Japan and after meeting with the PMDA has concluded this month that conducting a randomized study consistent with agency guidance to support a marketing application would likely not be feasible to complete within a time period that would support further investment. As a result, Kyowa Kirin decided to discontinue development of zandelisib in Japan. The discontinuation of zandelisib in Japan was a business decision by Kyowa Kirin based on the most recent regulatory guidance from the PMDA and is not related to the zandelisib clinical data generated to date.

In light of Kyowa Kirin’s decision to discontinue development of zandelisib in Japan, the parties intend to terminate the global license, development and commercialization agreement executed in April 2020.

•
In April 2023, MEI conducted a 1-for-20 reverse stock split. The reverse stock split was approved by MEI’s stockholders on January 5, 2023, and was implemented with the intent to increase the per share trading price of the Company’s common stock to enable the Company to satisfy the minimum bid price requirement for continued listing on Nasdaq. As per a notice received from the Nasdaq dated May 2, 2023, MEI regained compliance with the Nasdaq minimum bid requirement.

•
In March 2023, the Safety Review Committee of the Phase 1 study evaluating voruciclib, MEI’s orally administered cyclin-dependent kinase 9 (CDK9) inhibitor, plus venetoclax (Venclexta) completed a safety assessment of the initial dose escalation cohort evaluating the combination in patients with acute myeloid leukemia (AML) and recommended opening the next cohort. The combination stage of the study started after completing the single-agent dose exploration stage of the Phase 1 study in patients with either AML or B-cell malignancies. CDK9 inhibition disrupts Mcl-1 production and upregulation of Mcl-1 is a known escape mechanism of treatment with venetoclax. Thus, the combination being evaluated presents an opportunity to explore the synergistic potential to disrupt the cell cycle and inhibition of pro-survival cell cycle pathways.
•
In February 2023, MEI Pharma and Infinity Pharmaceuticals announced a definitive merger agreement for an all-stock transaction pursuant to which Infinity will become a wholly owned subsidiary of MEI Pharma. The combined company would have a projected cash balance of approximately $100 million at Closing that would be expected to fund operations through mid-2025, and to clinical data over the next 12 to 24 months across three clinical-stage oncology development programs: eganelisib, an oral immuno-oncology macrophage reprogramming product candidate, voruciclib, an oral CDK9 inhibitor, and ME-344, a novel tumor selective mitochondrial inhibitor.

Expected Drug Candidate Pipeline Developments

Voruciclib – Oral CDK9 inhibitor for the treatment of B-cell malignancies and acute myeloid leukemia
•
Report clinical data from the ongoing Phase 1b trial evaluating voruciclib plus Venclexta (venetoclax) in patients with acute myeloid leukemia around calendar year-end 2023.

ME-344 – Tumor selective mitochondrial inhibitor

•
Initiate a Phase 1b clinical trial evaluating ME-344 plus Avastin in relapsed colorectal cancer patients in the first half of calendar year 2023.
•
Report clinical data from the Phase 1b clinical trial evaluating ME-344 plus Avastin in patients with relapsed colorectal cancer around calendar year-end 2023.

Third Quarter Fiscal Year 2023 Financial Results

•
As of March 31, 2023, MEI had $112.0 million in cash, cash equivalents, and short-term investments with no outstanding debt.

•
For the quarter ended March 31, 2023, cash used in operations was $12.1 million, compared to $17.0 million used in operations during the quarter ended March 31, 2022. The decrease in cash used in operations is due to a reduction in zandelisib costs as we continued the close down of development activities and other changes in working capital.

•
Research and development expenses were $15.1 million for the quarter ended March 31, 2023, compared to $22.3 million for the quarter ended March 31, 2022. The decrease was primarily related to a reduction in zandelisib costs as we continued the close down of development activities in December 2022.

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General and administrative expenses were $7.2 million for the quarter ended March 31, 2023, compared to $8.9 million for the quarter ended March 31, 2022. The decrease primarily relates to personnel costs related to the reduction in workforce.

•
MEI recognized revenue of $5.9 million for the quarter ended March 31, 2023, compared to $9.7 million for the quarter ended March 31, 2022. The decrease in revenue comes as a result of the discontinuation of the zandelisib program in December 2022 and the associated reduction in expense reimbursement under our global License, Development and Commercialization Agreement with Kyowa Kirin.

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Net loss was $15.4 million, or $2.32 per share, for the quarter ended March 31, 2023, compared to net loss of $8.7 million, or $1.31 per share for the quarter ended March 31, 2022. The Company had 6,662,857 shares of common stock outstanding as of March 31, 2023, compared with 6,657,602 shares as of March 31, 2022.

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The adjusted net income (a non-GAAP measure) for the quarter ended March 31, 2023, excluding non-cash expenses related to changes in the fair value of the warrants, was $15.4 million, compared to an adjusted net loss of $21.5 million for the quarter ended March 31, 2022.

On May 11, 2023 Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, reported financial and operating results for the first quarter ended March 31, 2023 and will host a conference call today at 4:30 p.m. Eastern Time to discuss these results (Press release, Lineage Cell Therapeutics, MAY 11, 2023, View Source [SID1234631519]).

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"During the first quarter of this year, the Lineage team executed on multiple fronts, advancing our clinical and preclinical cell transplant programs while continuing support of our existing alliances and establishing new ones," stated Brian M. Culley, Lineage CEO. "A key area of focus was our partnership with Roche and Genentech and supporting the ongoing Phase 2a clinical study of OpRegen in patients with GA secondary to AMD, which is enrolling and treating patients at multiple sites in the U.S. Through presentations at various medical and scientific conferences, we also broadened our efforts to enhance awareness of OpRegen’s potential to provide durable anatomical and functional improvements in dry AMD patients. In addition, we established new collaborations with CIRM and the Christopher & Dana Reeve Foundation to support a new SCI conference which aims to identify, discuss, and address gaps in the product development process and elevate the patient’s voice in the treatment process. Looking ahead, we will be working on regulatory interactions for OPC1 while continuing to maintain our commitment to disciplined spending and making responsible investments in disease settings where we believe the use of differentiated cell transplants can provide a meaningful impact for patients."

Recent milestones and activities included:

– RG6501 (OpRegen)

Continued execution under our collaboration with Roche and Genentech across multiple functional areas, including support for the ongoing Phase 2a multi-center clinical study in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD):
Additional sites expected to come online this year.
Long-term follow-up of patients from the Phase 1/2a clinical study of OpRegen:
Preliminary evidence of durable anatomical and functional improvements following administration of OpRegen cells was presented at 2023 Association for Research in Vision and Ophthalmology Annual Meeting and other medical and scientific meetings.
– Received CIRM grant to support 1st Annual Spinal Cord Injury Investor Symposium

California Institute for Regenerative Medicine (CIRM) awarded Lineage an educational grant with a total award value of $50,000, recognizing this event as an important mission-specific conference, which will allow for the exchange of scientific information, create opportunities to accelerate the development of stem cell therapies to patients, increase the likelihood of successful treatments reaching patients, addresses an unmet clinical need, and can be impactful to CIRM’s overall mission.
– Strengthened OPC1 intellectual property portfolio

United States Patent and Trademark Office has granted a patent for the Company’s U.S. patent application No. 16/750,975, now U.S. Patent No. 11,603,518, entitled "Dorsally-Derived Oligodendrocyte Progenitor Cells From Human Pluripotent Stem Cells," with claims covering proprietary manufacturing processes developed by Lineage for its oligodendrocyte progenitor cell therapy candidate (OPC1) for the treatment of spinal cord injury. The patent has a term that would expire no earlier than 2040.
– Executed option and license agreement with Eterna Therapeutics

Enables development of novel beta 2 microglobulin (B2M)-deficient iPSC lines, which Lineage will evaluate for development into differentiated cell transplant therapies, specifically for the treatment of certain central nervous system disorders and other neurology indications.
– Initiated preclinical testing of ANP1

Preclinical testing underway through a collaboration with the University of Michigan and Yehoash Raphael, Ph.D., The R. Jamison and Betty Williams Professor of Otolaryngology, Department of Otolaryngology-Head and Neck Surgery and Lab Director at the University of Michigan Kresge Hearing Research Institute.
Some of the events anticipated by Lineage include:

– Type B Meeting with FDA to discuss a proposed amendment to the Investigational New Drug Application (IND) for OPC1 to enable clinical testing of a novel spinal cord delivery system.

– Amendment of an IND for OPC1 to enable clinical testing of a novel spinal cord delivery system.

– Submission of an additional OPC1 manuscript describing magnetic resonance imaging (MRI) findings from the subacute studies in both thoracic and cervical spinal cord injury.

– Updates from ongoing ANP1 preclinical testing at the University of Michigan Kresge Hearing Research Institute under a collaboration with the University of Michigan.

Balance Sheet Highlights

Cash, cash equivalents, and marketable securities totaled $46.8 million as of March 31, 2023, which is expected to support planned operations into Q3 2024.

First Quarter Operating Results

Revenues: Lineage’s revenue is generated primarily from licensing fees, royalties, collaboration revenues, and research grants. Total revenues for the three months ended March 31, 2023 were approximately $2.4 million, a net decrease of $2.8 million as compared to $5.2 million for the same period in 2022. The decrease was driven by less collaboration and licensing revenue recognized from deferred revenues from the Roche Agreement.

Operating Expenses: Operating expenses are comprised of research and development ("R&D") expenses and general and administrative ("G&A") expenses. Total operating expenses for the three months ended March 31, 2023 were $8.9 million, a decrease of $2.5 million as compared to $11.4 million for the same period in 2022.

R&D Expenses: R&D expenses for the three months ended March 31, 2023 were $4.2 million, a net increase of $1.2 million as compared to $3.0 million for the same period in 2022. The net increase was primarily driven by $0.5 million for nonclinical-related expenses to support the OPC1 program, and $0.2 million in OpRegen program expenses to support the Roche collaboration. Another $0.4 million and $0.2 million of the increase was related to R&D spending on the new auditory neuron and photoreceptor cell therapy programs, respectively.

G&A Expenses: G&A expenses for the three months ended March 31, 2023 were $4.7 million, a net decrease of $3.7 million as compared to approximately $8.4 million for the same period in 2022. The decrease was primarily driven by $3.5 million in lower litigation and legal expenses, mostly due to the Asterias litigation settlement expense accrued in the prior year, and $0.2 million in lower expense for accounting and tax services.

Loss from Operations: Loss from operations for the three months ended March 31, 2023 was $6.6 million, an increase of $0.2 million as compared to $6.4 million for the same period in 2022.

Other Income/(Expenses), Net: Other income (expenses), net for the three months ended March 31, 2023 reflected other income, net of $0.4 million, compared to other expense, net of ($0.7) million for the same period in 2022. The net income was primarily driven by fair market value changes in marketable equity securities, interest income from our marketable debt securities, and other income recorded in the current period related to the employee retention credit program, partially offset by exchange rate fluctuations related to Lineage’s international subsidiaries.

Net Loss Attributable to Lineage: The net loss attributable to Lineage for the three months ended March 31, 2023 was $4.4 million, or $0.03 per share (basic and diluted), compared to a net loss attributable to Lineage of $7.1 million, or $0.04 per share (basic and diluted), for the same period in 2022.

Conference Call and Webcast

Interested parties may access today’s conference call and webcast, by dialing (800) 715-9871 from the U.S. and Canada and should request the "Lineage Cell Therapeutics Call". A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 18, 2023, by dialing (800) 770-2030 from the U.S. and Canada and entering conference ID number 8339383.