On May 10, 2023 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported first quarter 2023 financial results and business updates (Press release, Precigen, MAY 10, 2023, View Source [SID1234631399]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Precigen continues to execute on our strategy to maintain corporate strength while advancing our most promising programs. This quarter, we successfully closed a public offering and more recently announced that Precigen has regained rights to two validated targets (CD19 and BCMA) that will further bolster our already robust UltraCAR-T portfolio and provides an opportunity to advance potential best-in-class UltraCAR-T drug candidates. We continue to advance our vision to transform the personalized cell therapy landscape using Precigen’s library approach to build the most comprehensive clinical and preclinical CAR-T portfolios with antigen-specific targets spanning both hematological and solid tumors where there is high unmet medical need for cancer patients, including CD33, MUC16, ROR1, CD19, BCMA and MSLN," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are pleased with the progress of our programs so far this year. We successfully dosed the first patient with PRGN-3007 and showcased preclinical data for our MSLN next generation UltraCAR-T at the 2023 AACR (Free AACR Whitepaper) annual meeting. We also presented positive Phase 1 data for our PRGN-2012 AdenoVerse immunotherapy in RRP at our R&D day and completed enrollment in the Phase 2 study. Finally, we look forward to sharing additional data at the 2023 ASCO (Free ASCO Whitepaper) annual meeting for our PRGN-3005 UltraCAR-T and PRGN-2009 AdenoVerse immunotherapy."

"We remain focused on strengthening our financial footing while containing costs to support our business objectives," said Harry Thomasian Jr., CFO of Precigen. "Our program of financial discipline, combined with a public offering and early retirement of most of our debt, has provided a solid cash runway to support priorities into late 2024."

Program Highlights

Exclusive Rights to UltraCAR-T Targets, CD19 and BCMA, and IL-12 Gene Therapy

· The Company amended its exclusive license agreement with Alaunos Therapeutics to bolster its portfolio and broaden strategic opportunities.
· The Company regained exclusive rights to CD19 and B-cell maturation antigen (BCMA) targets to enable unencumbered development and commercialization of two validated targets utilizing the UltraCAR-T platform.

· The Company also regained exclusive rights to its interleukin (IL)-12 gene therapy, including application through the off-the-shelf AdenoVerse immunotherapy platform, paving the way for potential future treatments in oncology given the important role of IL-12 cytokines in targeting many types of tumors such as human papillomavirus (HPV)-associated cancers.

PRGN-2012 AdenoVerse Immunotherapy in RRP

· PRGN-2012 is an investigational off-the-shelf (OTS) AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with HPV 6 or HPV 11 for the treatment of RRP. The US Food and Drug Administration (FDA) granted orphan drug designation for PRGN-2012 for patients with recurrent respiratory papillomatosis (RRP).
· The Company announced positive Phase 1 dose escalation and expansion cohort data (N=15) in January 2023 at its R&D Day virtual event.
· The Company completed enrollment in the Phase 2 portion of the study (N=23) bringing the total number of enrolled patients to 35 at Dose Level 2. Patient follow up is ongoing.
· The Company plans to outline the regulatory strategy as FDA discussions advance.

PRGN 2009 AdenoVerse Immunotherapy in HPV-associated Cancers

· PRGN-2009 is an OTS investigational immunotherapy utilizing the AdenoVerse platform designed to activate the immune system to recognize and target HPV-positive (HPV+) solid tumors.
· The Company completed enrollment in the Phase 1 monotherapy (N=6) and combination therapy (N=11) arms in patients with recurrent or metastatic HPV-associated cancers. An abstract for the clinical data of the PRGN-2009 Phase 1 study (Abstract # 2628) titled, "Phase I evaluation of PRGN-2009 alone and in combination with bintrafusp alfa in patients (pts) with recurrent/metastatic (R/M) HPV-associated cancers (HPV-C)" has been selected for presentation at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 3, 2023 from 8:00 to 11:00 AM CT.
· Enrollment was completed in the Phase 2 monotherapy arm with 20 evaluable patients in newly diagnosed oropharyngeal squamous cell carcinoma (OPSCC) patients. An interim clinical data presentation from the Phase 2 monotherapy arm is expected in the second half of 2023.

PRGN-3006 UltraCAR-T in AML

· PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T cell (CAR-T) therapy engineered to simultaneously express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a kill switch. The FDA granted orphan drug designation and fast track designation for PRGN-3006 UltraCAR-T for patients with relapsed or refractory (r/r) acute myeloid leukemia (AML).
· The Company completed the Phase 1 dose escalation study and announced positive data at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. Subsequently, the Company initiated a multicenter Phase 1b dose expansion study of PRGN-3006. The Company received FDA clearance to incorporate repeat dosing in the Phase 1b trial. A Phase 1b clinical data presentation is expected in 2024.

PRGN-3005 UltraCAR-T in Ovarian Cancer

· PRGN-3005 UltraCAR-T is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a kill switch.
· The Company completed enrollment in the Phase 1 dose escalation cohorts of the intraperitoneal (IP) and intravenous (IV) arms without lymphodepletion as well as in the lymphodepletion cohort in the IV arm. An abstract for the clinical data of the PRGN-3005 Phase 1 dose escalation study (Abstract # 5590) titled, "Phase 1/1b study of PRGN-3005 autologous UltraCAR-T cells manufactured overnight for infusion next day to advanced stage platinum resistant ovarian cancer patients" has been selected for presentation at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting on June 5, 2023 from 1:15 to 4:15 PM CT.
· The Company initiated a Phase 1b dose expansion trial of PRGN-3005. The Company received FDA clearance to incorporate repeat dosing in the Phase 1b study. A Phase 1b clinical data presentation is expected in 2024.

PRGN-3007 UltraCAR-T in Advanced ROR1+ Hematological and Solid Tumors

· PRGN-3007, based on the next generation of the UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), mbIL15, a kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.
· The Company announced dosing of the first patient in the Phase 1/1b dose escalation/dose expansion study of PRGN-3007 in advanced ROR1-positive (ROR1+) hematological and solid tumors. The target patient population for the Phase 1/1b study includes hematological cancers (chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), acute lymphoblastic leukemia (ALL), and diffuse large B-cell lymphoma (DLBCL)) and solid tumors (triple negative breast cancer (TNBC)).

Next Generation UltraCAR-T Platform

· The Company showcased advances in the UltraCAR-T platform with a preclinical data presentation for the next generation UltraCAR-T platform utilizing mesothelin (MSLN) CAR from Precigen’s library of non-viral plasmids at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023. Enhancement of efficacy due to incorporation of a novel mechanism for PD-1 blockade in MSLN UltraCAR-T in preclinical models was presented in the abstract titled, "Next Generation UltraCAR-T Cells with Intrinsic Checkpoint Inhibition and Overnight Manufacturing Overcome Suppressive Tumor Microenvironment Leading to Sustained Antitumor Activity."

Financial Highlights

· In January 2023, the Company completed an underwritten public offering of approximately 44 million shares of common stock, including a partial exercise of the underwriters’ option to purchase additional shares, at a price to the public of $1.75 per share, which resulted in net proceeds to the Company of $72.8 million (after deducting underwriting discounts, fees and other expenses).
· During the three months ended March 31, 2023, the Company successfully retired, through open market purchases, $29.5 million of outstanding convertible notes due in July 2023 at a discount to par bringing the total outstanding balance to $13.8 million. Any remaining outstanding convertible notes will be retired using the Company’s restricted cash balance. Early retirements have saved the Company close to $7 million through retirements at discounts to par and reduced interest costs.
· Cash, cash equivalents, short-term and long-term investments and restricted cash totaled $125.4 million as of March 31, 2023.
· Selling, general and administrative (SG&A) costs decreased by 15% for the three months ended March 31, 2023 compared to the prior year period.

First Quarter 2023 Financial Results Compared to Prior Year Period

Research and development expenses increased $0.4 million, or 3%, from the three months ended March 31, 2022. This increase was primarily driven by a continued prioritization of clinical product candidates.

Total other income, net, increased $2.5 million over the three months ended March 31, 2022. This increase was primarily due to reduced interest expense associated with the Company’s Convertible Notes as a significant portion of the original $200 million face value of the Convertible Notes has been retired. In addition, interest income increased due to higher interest rates on the Company’s investments.

SG&A expenses decreased $2.1 million, or 15%, from the three months ended March 31, 2022. This decrease was primarily driven by a reduction in professional fees of $2 million, primarily due to decreased legal fees associated with certain litigation matters.

Total revenues decreased $3.7 million, or 66%, from the three months ended March 31, 2022. This decrease related to the recognition of revenue in the first quarter of 2022 related to agreements for which revenue was previously deferred that did not occur in the first quarter of 2023 of $1.0 million, as well as declines in services performed at Exemplar.

Loss from continuing operations was $22.7 million, or $(0.10) per basic and diluted share, compared to loss from continuing operations of $23.9 million, or $(0.12) per basic and diluted share, in Q1 2022.

On May 10, 2023 PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53, reported financial results for the first quarter ended March 31, 2023, and provided a corporate update (Press release, PMV Pharma, MAY 10, 2023, View Source [SID1234631398]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased with the ongoing progress in the PYNNACLE study of PC14586, a first-in-class p53 Y220C reactivator, in patients with advanced solid tumors. We look forward to providing the next clinical and regulatory update in the second half of 2023," said David Mack, Ph.D., President and Chief Executive Officer. "Additionally, we are actively enrolling patients in the combination arm of PYNNACLE to explore the potential synergistic effects between PC14586 and KEYTRUDA."

Corporate Highlights:

•
The ongoing Phase 1/2 PYNNACLE study is evaluating PC14586 in patients with advanced solid tumors harboring a p53 Y220C mutation. The next clinical and regulatory update is expected in 2H 2023.
•
Commenced enrollment in the combination arm of PYNNACLE evaluating PC14586 with KEYTRUDA (pembrolizumab). PMV Pharma and Merck entered into a collaboration in 2022 under the terms of which Merck will supply KEYTRUDA for this study.

First Quarter 2023 Financial Results

•
PMV Pharma ended the first quarter with $229.4 million in cash, cash equivalents, and marketable securities, compared to $243.5 million as of December 31, 2022. Net cash used in operations was $15.0 million for the three months ended March 31, 2023, compared to $18.0 million for the three months ended March 31, 2022.
•
Net loss for the quarter ended March 31, 2023, was $19.1 million compared to $18.4 million for the quarter ended March 31, 2022.
•
Research and development (R&D) expenses were $15.1 million for the quarter ended March 31, 2023, compared to $11.8 million for the quarter ended March 31, 2022. The increase in R&D expenses was primarily related to increased clinical expenses to advance research on PC14586, the Company’s lead drug candidate.
•
General and administrative (G&A) expenses were $6.4 million for the quarter ended March 31, 2023, compared to $6.8 million for the quarter ended March 31, 2022. The decrease in G&A expenses was primarily due to reduced spend for insurance and outside services.

KEYTRUDA (pembrolizumab) is a registered trademark of Merck Sharp & Dohme LLC., a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About PC14586

PC14586 is a first-in-class, small molecule, p53 reactivator designed to selectively bind to the crevice present in the p53 Y220C mutant protein, hence, restoring the wild-type, or normal, p53 protein structure and tumor-suppressing function. The U.S. Food and Drug Administration granted Fast Track designation to PC14586 for the treatment of patients with locally advanced or metastatic solid tumors that have a p53

Y220C mutation. For more information about the Phase 1/2 PYNNACLE trial (PMV-586-101), refer to www.clinicaltrials.gov (NCT study identifier NCT04585750).

On May 10, 2023 Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer, and other indications, reported its financial results for the quarter ended March 31, 2023, and provided a business update (Press release, Pieris Pharmaceuticals, MAY 10, 2023, View Source [SID1234631397]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to be excited by the potential of our inhaled biologics pipeline to reshape the treatment paradigm for patients living with uncontrolled asthma and other chronic respiratory diseases. Our top priority remains the study completion and clinical data read out from the elarekibep Phase 2a study in asthma, which is benefitting from increased operational resources from our partner, AstraZeneca," said Stephen S. Yoder, President and CEO of Pieris. "Pieris continues to make measured investments in PRS-220 and PRS-400 while also expecting additional progress across partnered programs through our capital-efficient collaborations with Boston Pharmaceuticals, Genentech, Seagen and Servier."

Respiratory Pipeline:

Elarekibep and AstraZeneca Collaboration: Enrollment is ongoing in the multi-center, placebo-controlled Phase 2a study of dry powder inhaler-formulated elarekibep, an IL-4 receptor alpha (IL-4Rα) inhibitor being developed for the treatment of moderate-to-severe asthma. Topline results measuring placebo-adjusted FEV1 improvement at four weeks, the study’s primary efficacy endpoint, are expected by the middle of 2024. AstraZeneca previously communicated to the Company that completion of the Phase 2a study remains an important priority and that additional resources have been provided to achieve study completion. This includes a commitment to adding several new countries and a significant number of additional clinical sites, bringing the anticipated total number to more than 100 sites. As part of this commitment, AstraZeneca is on track to add three new geographies and related sites in the current quarter. Together with the previously announced protocol amendments, which are positively impacting study screening, we anticipate this will enable the achievement of the enrollment targets and timelines. In addition, the safety review of the 10 mg dose cohort in mild controlled asthmatics was successfully completed, which provides additional data supporting the elarekibep safety profile and enables doses greater than 3 mg to be evaluated in the future, if needed.

Previously reported elarekibep Phase 1 results demonstrated reduced fractional exhaled nitric oxide (FeNO) levels in mild asthma patients, and a favorable safety profile. Elarekibep is further validated by dupilumab, an FDA-approved inhibitor of IL-4Rα that has demonstrated reduced levels of FeNO and clinical efficacy in uncontrolled, moderate-to-severe asthma. Furthermore, dupilumab Phase 3 study results have shown efficacy in chronic obstructive pulmonary disease (COPD).

Pieris retains co-development and U.S. co-commercialization rights for elarekibep, which are exercisable following completion of the ongoing Phase 2a study.
PRS-220: Pieris continues clinical development of PRS-220, a potential best-in-class inhaled Anticalin protein targeting connective tissue growth factor (CTGF) for the treatment of IPF, a disease with a large unmet medical need, and other fibrotic lung diseases. Preclinically, PRS-220 demonstrated superior on-target potency compared to pamrevlumab, an intravenously infused CTGF antagonist in late-stage clinical development. The Company believes inhaled administration will deliver high lung exposure, optimal pulmonary target engagement and superior clinical outcomes, while offering convenience of at-home administration.

The Company is dosing healthy volunteers in a Phase 1 study with PRS-220 and expects to report results in the second half of this year. On May 21, 2023, preclinical data will be presented at the ATS 2023 International Conference, including data demonstrating that inhaled PRS-220 significantly reduced collagen deposition in a silica-induced lung fibrosis mouse model. Pieris continues to benefit from a meaningful grant from the Bavarian government, which supports early-stage development of this program.
PRS-400: Pieris continues its preclinical advancement of PRS-400, an inhaled anti-Jagged-1 Anticalin therapeutic program with transformative potential in a wide range of respiratory diseases driven by mucus hypersecretion. PRS-400 was designed to allow patients to exit the vicious cycle of mucus hypersecretion, infection and airway obstruction, while avoiding inhibition of healthy, normal mucus production outside of the lungs. On May 22, 2023, preclinical data will be presented at the ATS 2023 International Conference demonstrating that PRS-400 reduced inflammation-driven goblet cell metaplasia and mucus hypersecretion in a therapeutic disease model. PRS-400 is advancing toward development candidate nomination in the second half of this year.
Immuno-Oncology Pipeline:
Pieris’ immuno-oncology pipeline continues to progress in a cost-efficient manner with the benefit of its partners. The Company believes that multiple opportunities exist to generate value from this portfolio based on promising data generated to date.

In April, clinical results from the Company’s clinical study of cinrebafusp alfa (PRS-343) in 2L+ HER2-positive gastric cancer were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, including an unconfirmed 100% objective response rate and promising emerging durability profile in the five patients enrolled into the study prior to study discontinuation of enrollment for strategic reasons. Pieris is considering a range of transactions to facilitate program continuation, from an immuno-oncology focused spinout to a traditional partnering transaction.
Boston Pharmaceuticals continues to advance BOS-342 (also known as PRS-342), a 4-1BB/GPC3 bispecific Mabcalin (antibody-Anticalin protein) compound, toward the clinic, with Phase 1 expected to begin in the coming months.
Pieris and Servier continue to progress the escalation portion of the Phase 1/2 study of PRS-344/S095012, a 4-1BB/PD-L1 bispecific Mabcalin compound for the treatment of solid tumors, for which Pieris holds full U.S. rights.
As previously announced, Seagen initiated a Phase 1 study for SGN-BB228 (also known as PRS-346), triggering a $5 million milestone payment to Pieris. SGN-BB228 is a first-in-class CD228/4-1BB bispecific antibody-Anticalin compound designed to provide a potent costimulatory bridge between tumor-specific T cells and CD228-expressing tumor cells. Pieris and Seagen continue to collaborate on two other undisclosed bispecific programs.
Fiscal Year End Financial Update:

Cash Position – Cash, cash equivalents, and investments totaled $48.4 million for the quarter ended March 31, 2023, compared to a cash and cash equivalents balance of $59.2 million for the year ended December 31, 2022. The decrease was due to funding operations in the first quarter of 2023. The Company believes operations are sufficiently funded for more than the next 12 months.

R&D Expense – R&D expenses were $13.4 million for the quarter ended March 31, 2023, compared to $14.1 million for the quarter ended March 31, 2022. The decrease was due primarily to lower clinical costs for cinrebafusp alfa and lower personnel costs, license fees and software costs. These lower costs were partially offset by higher overall program costs for PRS-220 and higher preclinical costs for discovery-stage programs, both partnered and proprietary.

G&A Expense – G&A expenses were $4.0 million for the quarter ended March 31, 2023, compared to $4.4 million for the quarter ended March 31, 2022. The period-over-period decrease was driven primarily by lower professional services, consulting and insurance costs.

Other Income – For the quarter ended March 31, 2023, $2.0 million of grant income was recorded with respect to PRS-220, compared to $2.1 million for the quarter ended March 31, 2022, indicating that costs incurred on PRS-220 were comparable for both periods. Interest income was $0.4 million for the quarter ended March 31, 2023, given the impact of rising interest rates compared to a de minimis amount of interest income in the quarter ended March 31, 2022

Net Loss – Net loss was $13.2 million or $(0.45) per share for the quarter ended March 31, 2023, compared to a net loss of $5.1 million or $(0.07) per share for the quarter ended March 31, 2022.

Conference Call:

Pieris management will host a conference call beginning at 8:00 AM EDT on Wednesday, May 10, 2023, to discuss the first quarter 2023 financial results and provide a corporate update. Individuals can join the call by dialing 866-682-6100 (Toll Free US & Canada) or +1 862-298-0702 (International) at least five minutes prior to the start of the call. Alternatively, a listen-only audio webcast of the call can be accessed here.

A replay will be available on the Investors section of the Company’s website, www.pieris.com.

On May 10, 2023 Nuvectis Pharma, Inc (NASDAQ: NVCT) ("Nuvectis" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of innovative precision medicines for the treatment of serious conditions of unmet medical need in oncology, reported its financial results for the first quarter 2023 and provided an update on recent business progress (Press release, Nuvectis Pharma, MAY 10, 2023, View Source [SID1234631395]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis commented, "In the last few months, the Nuvectis team accomplished several important milestones that significantly advance our NXP800 and NXP900 development programs. For NXP800, we initiated the Phase 1b study, in which the preliminary efficacy of NXP800 in a target population, in this case platinum-resistant, ARID1a-mutated ovarian carcinoma, will be evaluated for the first time. The study is a multicenter, open label, single-arm clinical trial that will be conducted in the United States, United Kingdom and Europe, in collaboration with the GOG Foundation and the European Network of Gynecological Oncological Trial Group ("ENGOT"), two of the world’s premier gynecology oncology clinical trials consortia. The Phase 1b study follows the preliminary safety, tolerability and dosing findings from the Phase 1a in advanced solid tumors." Mr. Bentsur continued, "For NXP900, the Investigational New Drug ("IND") application with the US Food and Drug Administration ("FDA") is pending and we’re preparing to commence the first-in-human clinical trial shortly after the IND becomes effective. Lastly, recent publications for both NXP800 and NXP900 provided substantial scientific evidence for additional clinical development opportunities, including recent data from patient-derived xenograft ("PDX") in vivo models presented at this year’s American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting ("AACR") by investigators from the Mayo Clinic that demonstrated the potential of NXP800 to treat cholangiocarcinoma, a deadly disease for which only limited treatment options exist and new treatment options are greatly needed." Mr. Bentsur concluded, "We expect to continue to make strides in our development programs while effectively managing our cash position and we look forward to providing additional updates in the coming months."

First Quarter 2023 Financial Results

Cash, cash equivalents, and short-term investments were $15.5 million as of March 31, 2023, compared to $20.0 million as of December 31, 2022. The decrease of $4.5 million was primarily a result of the Company’s operations and payment of one-time items. The Company expects that its cash, cash equivalents, and short-term investments will be sufficient to meet its operating expense requirements into the second half of 2024.

The Company’s net loss was $4.0 million for the three months ended March 31, 2023, compared to $2.9 million for the three months ended March 31, 2022, an increase of $1.1 million. Net loss for the first quarter 2023 fiscal three months included $1.4 million in non-cash expenses related to stock-based compensation, $0.2 million in one-time development costs in connection with NXP900 and $0.1 million in interest income.

Research and development expenses were $2.4 million for the three months ended March 31, 2023, compared to $1.8 million for the three months ended March 31, 2022, an increase of $0.6 million. The increase in research and development expenses were primarily attributed to non-cash expenses related stock-based compensation, an increase in preclinical and clinical development costs and an increase in manufacturing costs. Research and development expenses for the first quarter 2023 included $0.7 million in non-cash expenses related to stock-based compensation.

General and administrative expenses were $1.7 million for the three months ended March 31, 2023, compared to $1.1 million for the three months ended March 31, 2022, an increase of $0.6 million. The increase in general and administrative expenses is primarily attributed to costs associated with operating as a public company following our 2022 initial public offering. General and administrative expenses for the first quarter 2023 included $0.7 million in non-cash expenses related to stock-based compensation.

On May 10, 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 cancers and viruses, reported an update on recent activity and expected near term milestones across its clinical development pipeline (Press release, Moleculin, MAY 10, 2023, View Source [SID1234631394]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"While it is important that Annamycin has proven safe in the last eight subjects in MB105 and MB106, we believe of greater significance is the activity shown by Annamycin in AML in subjects mostly heavily pre-treated by anthracyclines and other agents and mainly in elderly subjects. In the last eight subjects treated in those trials, we have had five or 63% respond with two complete responses or CRs and three partial responses or PRs. Two of the PRs had bone marrow aspirates below 5%," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Couple this with the activity we are seeing in the MB107 Phase 2 portion of our Phase 1b/2 clinical trial of Annamycin for the treatment of soft tissues sarcoma pulmonary metastases and we are excited about the potential for further positive data readouts as we move deeper into 2023."

"I am extremely proud of the progress we demonstrated across our development pipeline and ongoing clinical trials. With three Phase 1b/2 clinical trials underway, we are focused on driving recruitment forward and meeting the milestones ahead for each program. Importantly, these three currently active clinical trials are open label and provide us with the opportunity to announce clinical activity as it is being demonstrated throughout 2023," added Mr. Klemp.

Ongoing Clinical Trial Updates

Next Generation Anthracycline – Annamycin

Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic (unlike currently prescribed anthracyclines) and has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (a commonly prescribed anthracycline), as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. An independent expert has reported no signs of cardiotoxicity in the first 42 subjects in the Company’s three clinical trials, which total includes 32 subjects treated over the lifetime maximum anthracycline dose set by the U.S. Food and Drug Administration (FDA). Annamycin is currently in development for the treatment of STS lung metastases (STS lung mets) and relapsed or refractory acute myeloid leukemia (AML) and the Company believes the drug may have the potential to treat additional indications.

STS Lung Mets

The Company is currently in the Phase 2 portion of its ongoing U.S. Phase 1b/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases (MB107). clinicaltrials.gov: NCT04887298

Recent Activity Highlights

As previously announced, in the Phase 1b portion of the MB107 study, 15 subjects were enrolled, and the Company determined the Recommended Phase 2 Dose (RP2D) as safe and tolerable and demonstrated a preliminary response of stable disease or better in 60% (n=15) – defined as stable disease (SD) with STS lung mets or better after two cycles (approximately six weeks) of Annamycin. The median progression free survival (PFS) was 2.6 months for subjects receiving the study drug dose per the protocol in the Phase 1b portion. It is important to note that while most published studies of second line or later therapy in "advanced soft tissue sarcoma" include patients without lung metastases, MB107 includes only subjects with lung metastases, which are generally considered to be less responsive to such therapies. For this reason, the Company believes that the preliminary PFS results are encouraging.
Preliminary Phase 1b data showed no evidence of cardiotoxicity.
In the ongoing Phase 2 portion of the study, to date 13 of the up to 28 subjects have been identified, recruited, or dosed. Ten subjects have been dosed two cycles and received their end of Cycle 2 scans. One subject has received their initial dose and has not received cycle 2 scans.
Up to 28 subjects will be enrolled at the RP2D in Phase 2 to focus more on quantifying efficacy as well as providing additional safety information.
In this Phase 2 portion of the trial to date, Annamycin has demonstrated a progression free response of 60% (6 of 10) of subjects showing SD through 2 or more cycles, with three subjects continuing with the study drug. One such subject has received six cycles after their end of cycle 4 scan and has exhibited stable disease. For the three subjects with SD not continuing with the study drug, they are being followed for progression free survival and, for all subjects, overall survival is being monitored. These data are preliminary and subject to change.
An investigator sponsored Phase 1b/2 clinical trial was initiated in Poland in 2022 to study an alternative dosing regimen for Annamycin in the treatment of STS lung mets. This trial continues to enroll subjects and began dosing subjects in late 2022.
AML

The Company is currently conducting its Phase 1b/2 clinical trial evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with AML who are refractory to or relapsed after induction therapy (MB106). clinicaltrialsregister.eu: EudraCT 2020-005493-10 or clinicaltrials.gov: NCT05319587

Recent Activity Highlights

In 2022 Moleculin completed its Phase 1 single agent trial for Annamycin in AML (MB105), identifying an RP2D and demonstrating an 80% Overall Response Rate (ORR) in the five subjects treated with the RP2D in the final cohort. Three subjects achieved a Partial Response (PR) and one achieved a complete response with incomplete recovery of peripheral blood count (CRi). For purposes of this clinical trial, the protocol defines a CR to mean that the subject’s bone marrow aspirate assessments (BMA) reduced to 5% or less with recovery of neutrophils (or white blood cells) and platelets, CRi means a CR where there was incomplete recovery of neutrophils and/or platelet counts and a PR means the subject’s BMA reduced by 50% and resulted in a blast count of 25% or less.
It is noteworthy that one investigator designated two of the three PR subjects as such even though the subjects achieved BMA’s showing a blast count below 5% at the end of therapy. Notwithstanding this designation by the investigator, our own medical experts consider these subjects to have achieved CRs.
The MB106 clinical trial application (CTA) in Poland for a Phase 1b/2 trial of Annamycin in combination with Cytarabine (AnnAraC) for the treatment of AML was allowed in 2022. This trial was later approved to expand into Italy in 2022 and dosing subjects began in March 2023.
Five clinical sites in Poland and Italy have been recently activated for the MB-106 trial. The Company is planning for a total of up to eleven sites in the European Union (EU).
The Company recently completed the first cohort in the Phase 1b portion of the MB106 study. In the first cohort, three subjects were treated, all of whom are believed to be relapsed from multiple prior therapies. Annamycin was dosed at 190 mg/m2, along with Cytarabine at 2.0 g/m2/day for 5 days (total dose of 10 g/m2), consistent with the familiar 7+3 regimen, combining daunorubicin and Cytarabine, typically used as a first-line induction therapy. In the Company’s study therapy Annamycin replaces daunorubicin. The Company, at the recommendation of the data safety committee, deemed the first cohort dose as safe and opened the second cohort with the Annamycin dose being increased to 230 mg/m2.
Expected Upcoming Milestones

STS (U.S.): Report Phase 2 interim data.
STS (EU): Report Phase 1b/2 interim data.
AML: Present CSR for MB-105 Phase 1 results at an upcoming conference.
Flagship Immune/Transcription Modulator – WP1066

Moleculin is in ongoing discussions with multiple academic institutions in separate programs evaluating WP1066 for the treatment of brain tumors. The Company expects to finalize agreements in the second quarter with at least one investigator-sponsored clinical trial or program for the treatment of adult and pediatric brain tumors to commence in the second half of 2023.

Recent Activity Highlights

The Company supplied drug product to an externally funded pediatric brain tumor trial with WP1066 up to its conclusion in February 2023 and expects additional externally funded clinically trials for WP1066 (in combination with radiation) in 2023 in the U.S. and, possibly, in Southeast Asia.
Expected Upcoming Milestones

Report topline results from investigator-initiated Phase 1 study in pediatric brain tumors.
Seek external funding opportunities for an investigator-initiated clinical trial in adult and pediatric cancer patients in 2023.
Metabolism/Glycosylation Inhibitor – WP1122 Portfolio

WP1122 was developed as a prodrug of 2 deoxy-D-Glucose (2-DG) to provide a more favorable pharmacological profile and was found to have greater potency than 2-DG monotherapy in preclinical models where tumor cells require higher glycolytic activity than normal cells. WP1122 has also been shown to have a greater antiviral effect than 2-DG against SARS-CoV-2 in MRC-5 cells in culture. The improved pharmacokinetic and pharmacodynamic (PK/PD) profile of WP1122 compared to 2-DG was noted in mice following oral dosing at equimolar (i.e., equivalent levels of 2-DG) doses. The WP1122 Portfolio includes numerous analogs, including WP1096, which has demonstrated the potential for broad antiviral capabilities in a wide range of in vitro models including multiple arenaviruses, Zika virus, and HIV. The Company looks forward to the potential of externally funded research to confirm such activity.

Expected Upcoming Milestones

Report preliminary findings of National Institutes of Health (NIH) funded animal testing of WP1096 in the Tacaribe Arenavirus.
Identify investigators interested in initiating a clinical trial to study the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM.
General Information on the Company’s Core Technologies

Annamycin currently has Fast Track Status (FTS) and Orphan Drug Designation (ODD) from the FDA for the treatment of soft tissue sarcoma, in addition to ODD for the treatment of acute myeloid leukemia. WP1066 has ODD for the treatment of GBM and has four indications designated for the FDA Rare Pediatric Disease Priority Review Voucher (PRV) Program. WP1122 has ODD and FTS for GBM, as well. For more information about our trials, please visit clinicaltrials.gov.