On March 22, 2023 Atossa Therapeutics, Inc. (Nasdaq: ATOS), a clinical stage biopharmaceutical company developing innovative proprietary medicines to address significant unmet needs in cancer, reported financial results for the fiscal quarter and fiscal year ended December 31, 2022 and provided an update on recent company developments (Press release, Atossa Therapeutics, MAR 22, 2023, View Source [SID1234629169]).

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Key developments from Q4 2022 and year to date include:

Initiation and First Patient Dosed in Phase 2 EVANGELINE Study – EVANGELINE (Endoxifen Versus exemestANe GosEreLIn), is our Phase 2 randomized non-inferiority study of (Z)-endoxifen compared to exemestane plus goserelin as a neoadjuvant treatment for premenopausal women with Grade 1 or 2 ER+ / HER2- breast cancer. Participants receive neoadjuvant treatment for up to six months, followed by surgery. Several FDA-approved neoadjuvant therapies exist for ER- breast cancers, but few exist for ER+ patients, which account for approximately 78% of breast cancers. We expect to enroll approximately 175 patients at up to 25 sites across the United States.

Continued Enrollment in Phase 2 Karisma-Endoxifen Study – The Karisma-Endoxifen study is our randomized, double-blind, placebo-controlled trial of healthy, pre-menopausal women with increased breast density. The treatment cohort receives daily doses of (Z)-endoxifen for six months, over the course of which mammograms will be conducted to measure reduction in mammographic breast density (MBD). Patients will also be given a mammogram at 24 months to assess the durability of the MBD changes. MBD affects more than 10 million women in the United States and many millions more worldwide. Increased MBD reduces the ability of mammograms to detect cancer. Studies have also shown that women with MBD have an increased risk of developing breast cancer and that the higher the MBD, the higher the incidence of breast cancer. We expect to fully enroll the study by the end of 2023.

Initiation of New Study Arm in the Ongoing Phase 2 I-SPY 2 Clinical Trial – (Z)-endoxifen is being evaluated in a new study arm of the ongoing I-SPY 2 clinical trial. The I-SPY 2 trial evaluates neoadjuvant treatments for locally advanced breast cancer and is a collaborative effort among academic investigators from major cancer research centers across the United States, Quantum Leap Healthcare Collaborative, the U.S. Food and Drug Administration, and the Foundation for the National Institutes of Health (FNIH) Cancer Biomarkers Consortium. Approximately 20 patients will be treated with (Z)-endoxifen for up to 24 weeks prior to surgery.

Additional Intellectual Property Protection for (Z)-endoxifen – The United States Patent and Trademark Office granted a new patent (No. 11,572,334) directed to (Z)-endoxifen encapsulated in an enteric capsule. Enteric capsules have an acid resistant coating to prevent them from dissolving when they pass through the stomach. Enteric capsules are dissolved when they pass through an alkaline environment, which is usually when they reach the small intestine. Delivering oral (Z)-endoxifen via an enteric capsule prevents breakdown of the endoxifen in the stomach. This patent further reinforces Atossa’s broad Intellectual Property portfolio related to (Z)-endoxifen.

Investment in Dynamic Cell Therapies, Inc. (DCT) – DCT is a privately held, venture capital backed developer of CAR-T therapies in the pre-clinical phase of developing controllable CAR-T cells to address difficult-to-treat cancers. Its platform technology of dynamic control of engineered T-cells is designed to improve the safety, efficacy, and durability of CAR-T cell therapies. Our investment totaled $4.7 million and resulted in Atossa owning approximately 19% of the outstanding capital stock of DCT.

"Our focus remains on both helping reduce the incidence of breast cancer and changing the treatment paradigm for patients who are not benefiting from, or are unable to tolerate, currently approved therapies," said Dr. Steven Quay, Atossa’s President and Chief Executive Officer. "With a strong balance sheet and three Phase 2 trials underway, we are well positioned to continue accelerating the development of (Z)-endoxifen, which we feel has the potential to address significant unmet needs across the continuum of breast cancer."

YEAR ENDED DECEMBER 31, 2022, FINANCIAL RESULTS (IN THOUSANDS)

As of December 31, 2022, we had cash, cash equivalents and restricted cash of $111,000.

Results of Operations

Comparison of Years Ended December 31, 2022 and 2021

Revenue and Cost of Revenue:

For the years ended December 31, 2022 and 2021, we have no source of sustainable revenue and no associated cost of revenue.

Operating Expenses:

The following table provides a breakdown of major categories within Research and Development (R&D) and General and Administrative (G&A) expenses for the years ended December 31, 2022 and 2021, together with the dollar change in those categories:

2022 2021 Period-Period
Change
Research and Development
Clinical trials $ 10,225 $ 4,656 $ 5,569
Compensation 1,875 1,482 393
Stock-based compensation 2,393 1,591 802
Professional fees 1,242 454 788
Exclusivity agreements (700 ) 1,000 (1,700 )
Other 48 27 21
Research and Development Total $ 15,083 $ 9,210 $ 5,873
General and Administrative
Compensation $ 3,034 $ 2,371 $ 663
Stock-based compensation 4,395 3,676 719
Professional fees 1,625 2,317 (692 )
Legal 1,135 534 601
Insurance 1,640 1,576 64
Other 779 837 (58 )
General and Administrative Total $ 12,608 $ 11,311 $ 1,297
Total operating expenses were $27,691 for the year ended December 31, 2022, which was an increase of $7,170, or 35% from the year ended December 31, 2021. Operating expenses for 2022 consisted of R&D expenses of $15,083 and G&A expenses of $12,608. Operating expenses for 2021 consisted of R&D expenses of $9,210 and G&A expenses of $11,311. Factors contributing to the increased operating expenses in the year ended December 31, 2022 are explained below.

R&D Expenses: R&D expenses for the year ended December 31, 2022, were $15,083, an increase of $5,873 or 64% from total R&D expenses for the year ended December 31, 2021 of $9,210. Key changes were as follows:

The increase in R&D expense was attributed primarily to increased spending on clinical and non-clinical trials of $5,569 compared to the prior year period due to additional pre-clinical toxicology studies in our (Z)-endoxifen and AT-H201 programs as well as increased trial costs and manufacturing expenses for (Z)-endoxifen.
R&D compensation increased $393 in 2022 compared to the prior year period due to increased headcount, salary, bonuses and benefits during 2022.
Stock-based compensation, which is a non-cash charge, increased $802 due to the increased number of options being expensed as well as the weighted average fair value of options amortizing in 2022 was higher compared to the prior year period.
Professional fees increased $788 compared to the prior year period, due primarily to a CAR-T technology market analysis performed during 2022.
In 2022, the Company received a refund of $1,000 from the research institution with which the Company had an exclusive right to negotiate for the acquisition of the worldwide rights to two oncology R&D programs. In 2021, R&D expenses included $1,000 attributable to the same one-time exclusivity fee. Finally, in 2022, we paid $300 for the exclusive right to negotiate with another CAR-T Company. Net, the exclusivity agreements caused a decrease in expenses of $1,700 compared to the prior year period.

G&A Expenses: G&A expenses were $12,608 for the year ended December 31, 2022, an increase of $1,297, or 11% from total G&A expenses for the year ended December 31, 2021 of $11,311. Key changes were as follows:
The increase in G&A expense for the year ended December 31, 2022 compared to the prior year period, was in part attributable to the increase in compensation expense of $663 in 2022 due to an increase in headcount, hourly wages, salaries and bonus accruals.
Non-cash stock-based compensation expense also increased by $719 due to the increased number of options being expensed as well as the weighted average fair value of options amortizing in 2022 was higher compared to the prior year period.
Professional fees decreased by $692 in 2022 compared to the prior year period, due primarily to a decrease in proxy costs for investor outreach.
Legal fees increased $601 in 2022 compared to the prior year period, due to higher patent activity in 2022 for (Z)-endoxifen and our immunotherapy research.

Interest Income: Interest income was $877 for the year ended December 31, 2022 compared to the prior year period of $6. The increase is due to the investment of an additional $50,000 in a money market account during 2022 and higher average interest rates for the year ended December 31, 2022 compared to 2021.

ATOSSA THERAPEUTICS, INC.
CONSOLIDATED BALANCE SHEETS
(amounts in thousands, except for par value)
As of December 31,
2022 2021
Assets
Current assets
Cash and cash equivalents $ 110,890 $ 136,377
Restricted cash 110 110
Prepaid expenses 4,031 2,488
Research and development tax rebate receivable 743 1,072
Other current assets 2,423 1,193
Total current assets 118,197 141,240
Investment in equity securities 4,700 -
Other assets 635 22
Total Assets $ 123,532 $ 141,262
Liabilities and Stockholders’ Equity
Current liabilities
Accounts payable $ 2,965 $ 1,717
Accrued expenses 1,059 204
Payroll liabilities 1,525 1,184
Other current liabilities 19 21
Total current liabilities 5,568 3,126
Total Liabilities 5,568 3,126
Commitments and contingencies
Stockholders’ equity
Series B convertible preferred stock – $0.001 par value; 10,000 shares authorized; 1 shares issued and outstanding as of December 31, 2022 and December 31, 2021 - -
Additional paid-in capital – Series B convertible preferred stock 582 582
Common stock – $0.18 par value; 175,000 shares authorized; 126,624 shares issued and outstanding as of December 31, 2022 and December 31, 2021 22,792 22,792
Additional paid-in capital – common stock 250,784 243,996
Accumulated deficit (156,194 ) (129,234 )
Total Stockholders’ Equity 117,964 138,136
Total Liabilities and Stockholders’ Equity $ 123,532 $ 141,262

ATOSSA THERAPEUTICS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(amounts in thousands, except for per share amounts)
For the Year Ended
December 31,
2022 2021
Operating expenses
Research and development $ 15,083 $ 9,210
General and administrative 12,608 11,311
Total operating expenses 27,691 20,521
Operating loss (27,691 ) (20,521 )
Interest income 877 6
Other expense, net (146 ) (91 )
Loss before income taxes (26,960 ) (20,606 )
Income taxes - -
Net loss (26,960 ) (20,606 )
Loss per share of common stock – basic and diluted $ (0.21 ) $ (0.18 )
Weighted average shares outstanding – basic and diluted 126,624 116,950

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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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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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.