On March 22, 2023 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period and full year ended December 31, 2022 (Press release, Spectrum Pharmaceuticals, MAR 22, 2023, View Source [SID1234629168]).

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Fourth Quarter 2022 and Recent Business Update

First launch quarter for ROLVEDON, with net sales for the quarter and year ended December 31, 2022, totaling $10.1 million.
Operating expenses decreased 45% year-over-year as the Company streamlined operations while continuing to invest in core business objectives, including the commercialization of ROLVEDON.
70 targeted accounts purchased ROLVEDON during the launch quarter, including the top three community oncology networks, representing approximately 22% of the total clinic market.
Received permanent J-Code, facilitating more efficient and predictable reimbursement in the outpatient setting.
National Comprehensive Cancer Network Supportive Care Guidelines (NCCN Guidelines) in oncology for Hematopoietic Growth Factors named ROLVEDON as an appropriate option for cancer patients who are at risk for febrile neutropenia.
Cash, cash equivalents and marketable securities of $75.1 million at December 31, 2022, giving us an expected runway through 2024.
"It’s been a transformative year for Spectrum as we have become a commercially focused company. We’ve approached the launch of ROLVEDON with a disciplined strategy and an understanding that Spectrum’s long-term growth is dependent upon the product’s success. We’re off to a solid start and are encouraged by the initial customer receptivity to ROLVEDON," said Tom Riga, President and Chief Executive Officer of Spectrum Pharmaceuticals. "Commercial success is foundational to the Company’s future and, with the right people in place, a lean infrastructure, and an ample cash runway, we have a tremendous opportunity moving forward."

Financial Results for the Quarter and Year Ended December 31, 2022 (All numbers are from Continuing Operations)

Net sales for the quarter and year ended December 31, 2022 were $10.1 million as we began to sell our sole commercial product, ROLVEDON, which was approved by the FDA on September 9, 2022.

During the quarter and year ended December 31, 2022, the cost of sales was $1.8 million, consisting primarily of packaging costs, freight and royalties associated with the net sales of ROLVEDON and $1.1 million of start-up expenses associated with stability and bio-burden testing. This figure did not include any direct costs associated with the manufacture of ROLVEDON, which were previously expensed in research and development expense.

Selling, general and administrative expenses for the quarter and year ended December 31, 2022 were $11.3 million and $38.8 million, respectively, as compared to $18.9 million and $60.4 million for the comparable periods in 2021. The decrease was primarily due to lower costs associated with personnel related expenses associated with the reduction in workforce announced in January 2022 and decreases in professional services and other general expenses.

Total research and development expenses were $8.7 million and $42.2 million for the quarter and year ended December 31, 2022, respectively, as compared to $18.0 million and $87.3 million for the comparable periods in 2021. The decrease was due to decreased program activities for ROLVEDON, poziotinib, and early-stage compounds, personnel-related expenditures associated with the reduction in workforce during the strategic restructuring that began in January 2022, as well as a concession provided by Hanmi Pharmaceutical Co. Ltd. for drug substance which had been accrued during 2021 and is no longer payable by Spectrum.

Net loss was $11.7 million, or $0.06 per basic and diluted share, for the quarter ended December 31, 2022, compared to a net loss of $39.8 million, or $0.26 per basic and diluted share, for the comparable period in 2021. Net loss for the year ended December 31, 2022 was $78.1 million, or $0.43 per basic and diluted share, compared to net loss of $158.4 million, or $1.02 per basic and diluted share, for the comparable period in 2021.

The Company had a total cash, cash equivalents, and marketable securities balance of approximately $75.1 million at December 31, 2022.

Conference Call

As previously announced, management will host a conference call as follows:

Date:

Wednesday, March 22, 2023

Time:

8:30 AM ET
Register:

Click Here
Webcast (Audio Only):

Click Here
The webcast will be archived under the "Events and Presentations" section of the Company’s investor relations website.

About ROLVEDON

ROLVEDON (eflapegrastim-xnst) injection is a long-acting granulocyte colony-stimulating factor (G-CSF) with a novel formulation. Spectrum has received an indication to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. ROLVEDON is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation. The BLA for ROLVEDON was supported by data from two identically designed Phase 3, randomized, open-label, noninferiority clinical trials, ADVANCE and RECOVER, which evaluated the safety and efficacy of ROLVEDON in 643 early-stage breast cancer patients for the management of neutropenia due to myelosuppressive chemotherapy. In both studies, ROLVEDON demonstrated the pre-specified hypothesis of non-inferiority (NI) in mean duration of severe neutropenia (DSN) and a similar safety profile to pegfilgrastim. ROLVEDON also demonstrated non-inferiority to pegfilgrastim in the mean DSN across all four cycles (all NI p<0.0001) in both trials.

Please see the Important Safety Information below and the full prescribing information for ROLVEDON at www.rolvedon.com.

Indications and Usage

ROLVEDON is indicated to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia.

Limitations of Use

ROLVEDON is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation.

Important Safety Information

Contraindications

ROLVEDON is contraindicated in patients with a history of serious allergic reactions to eflapegrastim, pegfilgrastim or filgrastim products. Reactions may include anaphylaxis.
Warnings and Precautions

Splenic Rupture

Splenic rupture, including fatal cases, can occur following the administration of recombinant human granulocyte colony-stimulating factor (rhG-CSF) products. Evaluate patients who report left upper abdominal or shoulder pain for an enlarged spleen or splenic rupture.
Acute Respiratory Distress Syndrome (ARDS)

ARDS can occur in patients receiving rhG-CSF products. Evaluate patients who develop fever, lung infiltrates, or respiratory distress. Discontinue ROLVEDON in patients with ARDS.
Serious Allergic Reactions

Serious allergic reactions, including anaphylaxis, can occur in patients receiving rhG-CSF products. Permanently discontinue ROLVEDON in patients who experience serious allergic reactions.
Sickle Cell Crisis in Patients with Sickle Cell Disorders

Severe and sometimes fatal sickle cell crises can occur in patients with sickle cell disorders receiving rhG-CSF products. Discontinue ROLVEDON if sickle cell crisis occurs.
Glomerulonephritis

Glomerulonephritis has occurred in patients receiving rhG-CSF products. The diagnoses were based upon azotemia, hematuria (microscopic and macroscopic), proteinuria, and renal biopsy. Generally, events of glomerulonephritis resolved after dose-reduction or discontinuation. Evaluate and consider dose reduction or interruption of ROLVEDON if causality is likely.
Leukocytosis

White blood cell (WBC) counts of 100 x 109/L or greater have been observed in patients receiving rhG-CSF products. Monitor complete blood count (CBC) during ROLVEDON therapy. Discontinue ROLVEDON treatment if WBC count of 100 x 109/L or greater occurs.
Thrombocytopenia

Thrombocytopenia has been reported in patients receiving rhG-CSF products. Monitor platelet counts.
Capillary Leak Syndrome

Capillary leak syndrome has been reported after administration of rhG-CSF products and is characterized by hypotension, hypoalbuminemia, edema and hemoconcentration. Episodes vary in frequency and severity and may be life-threatening if treatment is delayed. If symptoms develop, closely monitor and give standard symptomatic treatment, which may include a need for intensive care.
Potential for Tumor Growth Stimulatory Effects on Malignant Cells

The granulocyte colony-stimulating factor (G-CSF) receptor through which ROLVEDON acts has been found on tumor cell lines. The possibility that ROLVEDON acts as a growth factor for any tumor type, including myeloid malignancies and myelodysplasia, diseases for which ROLVEDON is not approved, cannot be excluded.
Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML) in Patients with Breast and Lung Cancer

MDS and AML have been associated with the use of rhG-CSF products in conjunction with chemotherapy and/or radiotherapy in patients with breast and lung cancer. Monitor patients for signs and symptoms of MDS/AML in these settings.
Aortitis

Aortitis has been reported in patients receiving rhG-CSF products. It may occur as early as the first week after start of therapy. Consider aortitis in patients who develop generalized signs and symptoms such as fever, abdominal pain, malaise, back pain, and increased inflammatory markers (e.g., c-reactive protein and white blood cell count) without known etiology. Discontinue ROLVEDON if aortitis is suspected.
Nuclear Imaging

Increased hematopoietic activity of the bone marrow in response to growth factor therapy has been associated with transient positive bone imaging changes. This should be considered when interpreting bone imaging results.
Adverse Reactions

The most common adverse reactions (≥20%) were fatigue, nausea, diarrhea, bone pain, headache, pyrexia, anemia, rash, myalgia, arthralgia, and back pain.
Permanent discontinuation due to an adverse reaction occurred in 4% of patients who received ROLVEDON. The adverse reaction requiring permanent discontinuation in 3 patients who received ROLVEDON was rash.
To report SUSPECTED ADVERSE REACTIONS, contact Spectrum Pharmaceuticals, Inc. at 1-888-713-0688 or FDA at 1800FDA1088 or www.fda.gov/medwatch

On March 22, 2023 Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company focused on developing next-generation therapeutics to target difficult-to-treat cancers, reported financial results for the full year ended December 31, 2022, and provided a corporate update (Press release, Pyxis Oncology, MAR 22, 2023, View Source [SID1234629167]).

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"Over the past year, our team has made significant progress in advancing PYX-201, our antibody-drug conjugate (ADC) product candidate, and PYX-106, our immunotherapy product candidate, into Phase 1 clinical trials," said Lara S. Sullivan, M.D., President and Chief Executive Officer of Pyxis Oncology. "Our strong balance sheet gives us a cash runway into the first half of 2025 as we continue to advance these programs for patients. We anticipate preliminary data from both trials, including biomarker results and potential early signs of clinical activity, in late 2023 to early 2024."

Recent Corporate Updates

•
Two Phase 1 trials initiated: Clinical sites are being activated and patient screening is ongoing in the Phase 1 trial of PYX-106, referred to as PYX-106-101, and dosing is expected to begin early in the second quarter of 2023. Subject dosing is underway in the Phase 1 trial of PYX-201, known as PYX-201-101. Preliminary data are anticipated from both trials in the late-2023 to early-2024 timeframe.
•
PYX-201 nonclinical data published: A peer-reviewed article titled "Quantification of antibody-drug conjugate PYX-201 in rat and monkey plasma via ELISA and its application in preclinical studies" was published online in Bioanalysis. This publication described the ELISA assay that was successfully validated for the measurement of PYX-201 concentrations in rat and monkey plasma in support of the Company’s successful IND application.
•
Abstracts submitted to ASCO (Free ASCO Whitepaper): Abstracts for trial in progress (TIP) posters describing the PYX-106-101 and PYX-201-101 Phase 1 clinical trials were submitted to the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held June 2-6, 2023, in Chicago.
•
Two INDs cleared by FDA: In November 2022, clearance for the PYX-106 and PYX-201 Investigational New Drug (IND) applications was received from the U.S. Food and Drug Administration (FDA).

Full-Year 2022 Financial Results

•
As of December 31, 2022, Pyxis Oncology had cash and cash equivalents (including restricted cash) of $180.7 million, which is expected to fund operations into the first half of 2025.
•
Research and development expenses were $86.1 million for the year ended December 31, 2022, compared to $51.0 million for the year ended December 31, 2021. The increase was primarily due to increased expenses associated with contract manufacturing of drug products and drug substance, preclinical costs related to toxicity studies and preclinical work in support of IND filings, clinical trial costs, and an increase in employee headcount to support research and development activities.
•
General and administrative expenses were $37.4 million for the year ended December 31, 2022, compared to $18.7 million for the year ended December 31, 2021. The increase was primarily due to higher personnel-related expenses, including stock-based compensation, and increases in legal and professional fees, rent and directors and officers insurance expenses to support our growth and operations.
•
Net loss was $120.7 million, or ($3.65) per common share, for the year ended December 31, 2022, compared to $76.0 million, or ($8.95) per common share, for the year ended December 31, 2021. Net losses for the years ended December 31, 2022 and 2021 included $15.8 million and $6.4 million, respectively, related to non-cash stock-based compensation expense.
•
As of March 21, 2023, the outstanding number of shares of Common Stock of Pyxis Oncology was 36,980,621.

Pam Connealy, Chief Financial Officer of Pyxis Oncology, added, "As our clinical programs have advanced, our team has established a strong financial foundation with no debt and a cash runway into the first half of 2025. We expect our balance sheet will enable us to evaluate early signs of clinical activity in our two clinical programs and initiate tumor-specific expansion cohorts following dose selection."

On March 22, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical-stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer, reported an Issue Notification for patent JP 7232845 titled "METHODS FOR PRODUCING (6S,15S)-3,8,13,18- TETRAAZAICOSANE-6,15-DIOL" (Press release, Panbela Therapeutics, MAR 22, 2023, View Source [SID1234629166]). This patent, developed in collaboration with Syngene International Ltd., an integrated research, development, and manufacturing services company, claims a novel process with a reduced number of synthetic steps from seventeen to six to produce SBP-101, a lead investigational product. The patent is valid till 2039.

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Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela Therapeutics, commented, "We’re excited to have this patent in Japan. Expansion of our patent portfolio further supports our global clinical programs. This patent is the outcome of the dedicated efforts of our valued long-term partner Syngene International Ltd. in helping us achieve this important goal." First issued in the United States in 2021, this patent covers a shorter synthesis of SBP-101, which provides many benefits including: 1) the ability to manufacture product with a reduced lead time 2) quicker access to drug supply facilitating expansion into additional indications and 3) enables a scalable, efficient and cost-effective manufacturing process for future commercialization.

Jonathan Hunt, Managing Director and Chief Executive Officer, Syngene International Ltd., said, "We have been partnering with Panbela for the last decade, and I am proud of the work achieved through the successful collaboration. In this case, reducing the number of steps in production and simplifying the manufacturing process means that the drug will reach patients faster. The protection of SBP-101 production through patents in the U.S. and now in Japan are significant milestones."

Dr. Simpson added, "The Company expects to continue innovation and patent portfolio building to support our clinical programs. This process in this patent utilizes a pharmaceutical starting material that is widely available, increasing the availability of drug supply moving forward. "

About Panbela’s Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

Ivospemin (SBP-101)
Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial.

Flynpovi
Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increasing polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X
CPP-1X (eflornithine) is being developed as a single agent tablet or high dose powder sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigatorinitiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.

On March 22, 2023 Oxford Drug Design, a biotechnology company with core expertise in computer-aided drug design, reported that it has been engaged by PhoreMost Ltd (PhoreMost), a UK-based biopharmaceutical company dedicated to ‘Drugging the Undruggable’ disease targets, to accelerate a targeted protein degradation discovery programme for novel cancer therapeutics (Press release, Oxford Drug Design, MAR 22, 2023, https://oxforddrugdesign.com/2023/03/22/oxford-drug-design-and-phoremost-collaborate-to-advance-novel-cancer-therapeutics-discovery/ [SID1234629165]).

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The project leverages Oxford Drug Design’s proprietary artificial intelligence (AI) computational platform consisting of multiple computational drug discovery capabilities for both ligand- and structure-based design, together with PhoreMost’s next-generation SITESEEKER phenotypic screening platform and PROTEINi libraries. In doing so, Oxford Drug Design has analyzed the structural biology data and known binding compounds to identify and advance the development of novel, drug-like, compounds for onward optimization.

Dr Paul Finn, CSO of Oxford Drug Design commented: "We’re delighted to start deploying our pioneering computational platform and help other companies accelerate their own drug discovery efforts and we’re excited to have collaborated with PhoreMost on this project. Oxford Drug Design’s pioneering AI drug discovery platform has been well placed to make an impact on this challenging target."

Dr Richard Boyce, VP Drug Discovery at PhoreMost added: "PhoreMost’s involvement in this project demonstrates the versatility of our SITESEEKER phenotypic screening platform and the potential of our PROTEINi libraries. Oxford Drug Design’s pioneering AI drug discovery platform has facilitated the rapid discovery of small-molecule drugs to newly identified druggable targets obtained from SITESEEKER."

The global AI drug discovery market is estimated to be worth $5 billion by 2027, according to a recent report by MarketsandMarkets. With traditional drug discovery being a costly and lengthy process, the highly distinctive AI and machine learning technologies developed by Oxford Drug Design offers the prospect of analysing vast datasets and develop viable drugs in an automated and more cost-efficient way.

On March 22, 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 recent activity, new positive data and expected near term milestones across its clinical development pipeline (Press release, Moleculin, MAR 22, 2023, View Source [SID1234629164]).

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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

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"We continue to make significant progress on multiple fronts across our development pipeline and ongoing clinical trials. Our team believes that the human activity demonstrated with our lead program, Annamycin, in two separate indications, normally treated with doxorubicin (or its equivalent), continues to support our expectation of generating pivotal data in 2023. Additionally, preliminary data continue to demonstrate a promising safety and efficacy profile with zero evidence of cardiotoxicity, as analyzed by our independent expert, which bolsters our confidence in Annamycin’s potential as we continue to advance development. Additionally, we are pleased with the rapid pace of enrollment in our Phase 2 STS lung metastases clinical trial that, we believe, has us on track to complete enrollment in the first half of 2023 and in a position to report topline data from this study in the second half of this year," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Mr. Klemp added, "2023 is poised to be an exciting year. We are actively recruiting in three Phase 1b/2 clinical trials and expect to add more trials during the year. 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. Additionally, in February 2023, our externally funded Phase 1 clinical trial studying WP1066 for the treatment of pediatric brain tumors concluded, and we expect to have at least three externally funded Phase 1b/2 clinical trials for WP1066 in the treatment of GBM and other brain tumors in 2023, including another pediatric clinical trial, in the U.S. and, possibly, in Southeast Asia."

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 patients in the Company’s three clinical trials, which total includes 32 patients 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 we 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.

●

Preliminary Phase 1b data showed no evidence of cardiotoxicity.

●

In the ongoing Phase 2 portion of the study, to date 14 of the up to 28 subjects have been identified, recruited, or dosed. Of those recruited, 11 have begun receiving doses and 6 have been dosed two cycles and received their end of Cycle 2 scans.

●

In this Phase 2 portion of the trial to date, Annamycin has demonstrated a preliminary response of 67% (4 of 6) of subjects showing SD through 2 or more cycles, with one subject continuing with the study drug. The main reason for subjects not continuing is due to myelosuppression attributed to the study drug, keeping in mind that most of these subjects have had multiple prior treatments for STS and some have received close to the lifetime maximum anthracycline dose (LTMAD) as determined by the FDA prior to entering the study. All four subjects will continue to be monitored for PFS while in the study. These data are preliminary and subject to change.

●

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.

●

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 began administering drug to subjects in late 2022, at dosages significantly below that being tested in the Company’s U.S. study, with no human activity noted in the first two subjects in the first cohort of the Phase 1b portion of the study. This study is ongoing.

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 (MB-106). clinicaltrialsregister.eu: EudraCT 2020-005493-10 or clinicaltrials.gov: NCT05319587

Recent Activity Highlights

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In 2022 Moleculin completed its Phase 1 single agent trial for Annamycin in AML (MB-105) with fifteen subjects receiving a full cycle per protocol, 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 means 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.

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It is noteworthy that one investigator designated two of the three PR subjects as such even though the subjects did achieve BMA’s showing a blast count below 5% at the end of therapy.

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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).

Upcoming Milestones Expectations

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STS (U.S.): Report Phase 2 interim data.

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STS (EU): Report Phase 1b/2 interim data.

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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 investigator-sponsored clinical trials or programs for the treatment of adult and pediatric brain tumors to commence in the first half of 2023.

Recent Activity Highlights

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In 2022, an investigational new drug (IND) application Moleculin filed in the U.S. for a Phase 1 clinical trial studying WP1066 for treating glioblastoma multiforme (GBM) in adults received FDA allowance or went into effect.

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

Expected Upcoming Milestones

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Report topline results from investigator-initiated Phase 1 study in pediatric brain tumors.

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Seek to capitalize on external funding with 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 alone 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 externally funded clinical trials to confirm such activity.

Recent Activity Highlights

●

The Company’s Phase 1a clinical trial of WP1122 in the United Kingdom for the treatment of COVID-19 began recruiting in 2022, and the Company safely completed the trial in late 2022, establishing an RP2D. This RP2D will possibly aid in future externally funded trials for the treatment of certain viruses and cancers as the Company looks for investigator led studies.

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In 2021 the Company filed an IND with the FDA that then went into effect, allowing it to proceed with a clinical trial using WP1122 for the treatment of glioblastoma multiforme (GBM). This may lead to an oncology investigator-initiated trial.

Expected Upcoming Milestones

●

Report preliminary findings of National Institutes of Health (NIH) funded animal testing of WP1096 in the Tacaribe Arenavirus.

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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 treatments designated for the FDA Rare Pediatric Disease Priority Review Voucher (PRV) Program. WP1122 has ODD for GBM, as well, and FTS for GBM. For more information about our trials, please visit clinicaltrials.gov.