On March 3, 2022 Pepper Bio, the world’s first transomics drug discovery company, reported a collaboration with the Dean Felsher Laboratory at Stanford University (Press release, Pepper Bio, MAR 3, 2022, View Source [SID1234638702]). Leveraging Pepper Bio’s unique platform on phosphoproteomics, the two teams aim to identify and validate novel targets for hepatocellular carcinoma and lymphoma within the year.

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Hepatocellular carcinoma, the most prevalent type of liver cancer, is diagnosed in over 35 thousand people, and lymphoma is diagnosed in over 80 thousand people each year. To treat these cancers and others, researchers need to target the broken genes that either rapidly spur or fail to suppress tumor growth. The Felsher Lab has identified one of these processes, known as oncogene addiction, in which specific tumor cells depend on a single activated oncogenic protein or pathway to maintain malignancy. When the activated pathway the cancer relies on is defeated, so is the cancer. Defeating the activated pathway is a nontrivial scientific task.

In studying oncogene addiction, Felsher’s Lab found protein phosphorylation is a critical mechanism of oncogenesis and, thus, crucial to understand for treating these highly aggressive cancers. Pepper Bio is the first and only techbio company to provide academic researchers and pharmaceutical partners with technology to identify functional, actionable insights on protein phosphorylation, known as phosphoproteomics.

"Pepper Bio’s platform is able to integrate and translate large amounts of different kinds of measurements across the omic layers into actionable insights, and provides an immense advantage in identifying disease pathways that are implicated in the proliferation of cancer," said head of the Felsher Lab and Stanford Medicine’s Associate Chief of Medical Oncology, Translational Research & Applied Medicine, Dean Felsher, MD, PhD. "As such, Pepper’s technology is highly valuable to my lab’s current research focus of surfacing insight around which phospho-proteins are linked to specific oncogenes."

By fully stacking and integrating these four layers of biological data — genomics, transcriptomics, and proteomics, and phosphoproteomics — researchers have a complete, real-time map of what happens in cells before and during disease. Pepper Bio will collaborate with the Felsher Lab and contribute to oncogene addiction and target validation research. Co-founder and Chief Science Officer Samantha Dale Strasser, PhD., will lead efforts on the startup’s side.

"As our technology teases out changes in pathway activity, notably phosphoproteomics, we can directly support the goal of finding the linchpins necessary to defeat pathways of oncogene addiction. There is a natural fit between the work the Felsher lab is doing and the transomics capabilities we have unlocked," said Dr. Strasser. "Dean is an excellent mentor, resource, and partner as we bring our technology to an application where it can advance research outcomes and improve the lives of patients and their loved ones."

Just three months after Pepper Bio launched from stealth with backing from VC firm, NFX Bio, this announcement underscores the readiness and viability of its platform for drug discovery and target validation.

To learn more about Pepper Bio, please visit: Pepper.bio

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Puma Biotechnology has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission .

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On March 3, 2022 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported its financial results for the year ended December 31, 2021 and provided a business update (Press release, INmune Bio, MAR 3, 2022, View Source [SID1234609522]).

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In December, the Company reported data from the first patient treated with INKmuneTM in the myelodysplastic syndrome (MDS) Phase I clinical trial. More than 100 days after the course of INKmuneTM therapy, 60% of the patient’s NK cells showed the activated, tumor killing memory like NK cells phenotype, a fourfold increase from pre-treatment. The patient’s memory like NK cells killed >70% of NK resistant tumor cells in an in vitro assay. The patient remains well and with an ECOG status of 0, a two-point drop from pre-treatment. Additionally, two patients were treated with INKmuneTM under compassionate use after having failed at least one allogeneic bone marrow transplant. One of the two patients has been discharged home, one remains hospitalized. In all cases, INKmuneTM therapy was well tolerated, safe and was given without any type of pre-medication or cytokine therapy.

"These patients demonstrate the unique attributes of INKmuneTM therapy in patients with high-risk MDS/AML. INKmuneTM converted the patient’s resting NK cells into cancer killing memory like NK cells. The memory like NK cells killed NK-resistant cancer cells in an in vitro assay. Both these attributes lasted four months, a trait we are calling therapeutic persistence." stated RJ Tesi, M.D., Chief Executive Officer of INmune Bio. "We are continuing to screen patients for enrollment into the trial and are in process of expanding the number of clinical trial sites."

The Phase II clinical trial using XProTM to treat patients with AD and elevated biomarkers of neuroinflammation (ADi) is open, screening patients and seeking to enroll the first patient. A second Phase II trial in patients with Mild Cognitive Impairment (MCI) should begin enrolling patients soon. Patients must have ApoE4 to be included in the MCI trial. INMB is treating MCI and Mild AD in separate clinical trials due to differences in disease severity with the potential for different rates of response to therapy. The MCI trial is a three-month trial designed to determine the extent to which XProTM has an impact on biological and cognitive biomarkers in MCI patients. The mild AD trial is a six-month trial with EMACC as the primary endpoint and secondary endpoints of CDR, ADL, NPI, MRI, and blood biomarkers of neurodegeneration. Top line data is expected in the first half of 2023 from the MCI trial and the second half of 2023 from the mild ADi trial. A video released today can be found on the company’s website which explains our rationale for XPro in treating neuroinflammation to potentially reduce white and gray matter degeneration which can be found by clicking here.

Q4 2021 and Recent Corporate Highlights

IINKmuneTM Platform Highlights:

The Phase I program has been peer-reviewed by the UK National Cancer Research Institute (NCRI) – Myelodysplastic Syndrome Expert Group and has been accepted for listing on their national MDS trials site. This is unique to the UK; no equivalent system is present in the US. This allows specialist MDS trials centers to refer patients to the current UK clinical trial sites for treatment under the clinical trial protocol or to request to become an additional trial site. We hope this added validation of the trial and exposure to additional expert centers will provide more patients for enrollment into the Phase I MDS program and facilitate future phase II trials.
Presented 119-day data on first patient in company’s ongoing Phase 1 clinical trial of its Natural Killer (NK) cell priming platform, INKmuneTM, as a potential treatment for high-risk myelodysplastic syndrome (MDS). A single course of INKmuneTM has benefited the course the patient’s disease for more than 6 months. The data were presented at the 2021 British Society of Immunology Congress.
Announced a pre-clinical research collaboration with the Chinese University of Hong Kong to evaluate INKmuneTM, the company’s pseudokine NK cell priming platform, in nasopharyngeal cancer (NPC), a type of head and neck cancer. The project provides INMB scientists working at University College of London with access to the only three proven NPC cancer cell lines to test the ability of INKmuneTM-primed NK cells to kill NPC tumors. This pre-clinical work, if successful, will provide data for a future clinical trial in NPC.
DN-TNF Platform Highlights (XProTM and INB03TM):

Opened the Phase II trial using XProTM to treat patients with mild ADi for enrollment. The primary endpoint will examine cognition using the Early AD/MCI Alzheimer’s Cognitive Composite (EMACC). Multiple secondary endpoints of cognition will also be measured, including CDR-SB, ADAS-COG13 and other endpoints. Data is anticipated in the second half of 2023.
Delivered multiple oral and poster presentations at the AAIC 2022 (Alzheimer’s Association International Conference) and the 14th Clinical Trials on Alzheimer’s Disease (CTAD) Annual Meeting.
Presented new breast cancer data at the 2021 San Antonio Breast Cancer Symposium. A significant percentage of women with triple negative breast cancer (TNBC) express MUC4. MUC4 expression predicts a worse survival and increased risk of developing metastatic disease. In addition, MUC4 expressing patients have "cold" tumors with fewer tumor infiltrating lymphocytes. These data suggest the use of INmune’s DN-TNF candidate, INB03TM, may help reverse resistance to immunotherapy women with TNBC.
Upcoming Milestones:

Initiate XProTM Phase II program for Mild Cognitive Impairment (MCI) in patients with APOE4 allele 1H 2022.
Initiate XProTM Phase II program for treatment resistant depression (TRD), funded in part by a $2.9 million NIH grant, by 2H 2022.
Initiate INKmune Phase I program in ovarian cancer in 2H 2022.
Additional open-label Phase 1 trial data of INKmuneTM in high-risk MDS.
Report top-line data from Phase 2 trial of XProTM in MCI patients in 1H 2023.
Report top-line data from Phase 2 trial of XProTM in mild Alzheimer’s patients in 2H 2023.
Present INKmuneTM clinical data and new pre-clinical data on mechanism of action at the Innate Killer Summit conference in San Diego in March.
Report pre-clinical INKmuneTM data in at least two new solid tumor indications, renal cell carcinoma and nasopharyngeal carcinoma.
Oral and Poster presentations at AD/PD 2022, the largest European AD meeting. The meeting will be held in Barcelona in March.
Financial Results for the Year Ended December 31, 2021:

Net loss attributable to common stockholders for the year ended December 31, 2021 was approximately $30.3 million, compared to approximately $12.1 million for the year ended December 31, 2020.

Revenues were approximately $0.2 million for the year ended December 31, 2021, compared to $0.0 million for the year ended December 31, 2020.

Research and development expense totaled approximately $20.5 million for the year ended December 31, 2021, compared to approximately $5.9 million during the year ended December 31, 2020.

General and administrative expense was approximately $8.8 million for the year ended December 31, 2021, compared to approximately $6.3 million during the year ended December 31, 2020.

Other expense was approximately $1.2 million during the year ended December 31, 2021 compared to other income of approximately $0.1 million during the year ended December 31, 2020.

As of December 31, 2021, the Company had cash of approximately $74.8 million.

As of March 3, 2022, the Company had approximately 17.9 million common shares outstanding.

Earnings Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call.

A live audio webcast of the call can be accessed using this link: View Source;tp_key=636ec61ab2

A transcript will follow approximately 24 hours from the scheduled call. A replay will also be available through March 10, 2022 by dialing 1-844-512-2921 or 1-412-317-6671 (international) and entering PIN no. 13726701.

About XProTM

XProTM is a next-generation inhibitor of tumor necrosis factor (TNF) that is currently in clinical trial and acts differently than currently existing TNF inhibitors in that it neutralizes soluble TNF (sTNF), without affecting trans-membrane TNF (tmTNF) or TNF receptors. XProTM could have substantial beneficial effects in patients with neurologic disease by decreasing neuroinflammation. For more information about the importance of targeting neuroinflammation in the brain to improve cognitive function and restore neuronal communication visit this section of the INmune Bio’s website.

About INKmuneTM

INKmune is a pharmaceutical-grade, replication-incompetent derivative of our proprietary INB16 cell line, a human tumor cell line, which conjugates to resting NK cells and delivers multiple essential priming signals akin to treatment with a combination of at least three cytokines. INKmuneTM is stable at -80 °C and is delivered by a simple IV infusion. The INKmuneTM:NK interaction ligates multiple activating and co-stimulatory molecules on the NK cell and enhances its avidity of binding to tumor cells; notably those resistant to normal NK-mediated tumor lysis. Tumor-primed NK (TpNK) cells can lyse a wide variety of NK-resistant tumors, including: leukemias, lymphomas, myeloma, ovarian cancer, and breast cancer.

On March 3, 2022, X4 Pharmaceuticals, Inc. (the "Company"), reported that it entered into a securities purchase agreement (the "Securities Purchase Agreement") with BCLS II Investco, LP (the "Investor"), an affiliate of Bain Capital Life Sciences, pursuant to which the Company agreed to issue and sell to the Investor in a private placement (the "Private Placement") (i) 900,000 shares (the "Shares") of the Company’s common stock, par value $0.001 per share (the "Common Stock"), at a purchase price of $1.80 per share, which represents the volume weighted average price per share of the Common Stock as quoted on the Nasdaq Stock Market for the thirty (30) consecutive-day trading day period ending on March 2, 2022, and (ii) pre-funded warrants (the "Pre-Funded Warrants") to purchase 766,666 shares of Common Stock at a price of $0.01 per share, at a purchase price of $1.79 per Pre-Funded Warrant (Filing, 8-K, X4 Pharmaceuticals, MAR 3, 2022, View Source [SID1234609507]). The price per Pre-Funded Warrant represents the price of $1.80 per share to be sold in the Private Placement, minus the $0.01 per share exercise price of each such Pre-Funded Warrant. The Pre-Funded Warrants are exercisable at any time after their original issuance and will not expire.

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The Pre-Funded Warrants to be issued in the Private Placement will provide that the holder of the Pre-Funded Warrants will not have the right to exercise any portion of its Pre-Funded Warrants if such holder, together with its affiliates, would beneficially own in excess of 9.99% of the number of shares of the Company’s Common Stock outstanding immediately after giving effect to such exercise (the "Beneficial Ownership Limitation"); provided, however, that the holder may increase or decrease the Beneficial Ownership Limitation by giving 61 days’ notice to the Company, but not to any percentage in excess of 19.99%.

The Private Placement is expected to close on or about March 7, 2022 (the "Closing Date"), subject to the satisfaction of certain customary closing conditions. The Company expects to receive aggregate gross proceeds from the Private Placement of approximately $3.0 million, before deducting estimated offering expenses payable by the Company. The Company expects the net proceeds from the Private Placement to be used for advancement of the Company’s clinical development pipeline, business development activities, working capital and general corporate purposes.

The foregoing descriptions of the Securities Purchase Agreement and the Pre-Funded Warrants do not purport to be complete and are qualified in their entirety by reference to such agreements, copies of which are filed as Exhibits 10.1 and 4.1 hereto, respectively, and incorporated by reference herein.

Registration Rights Agreement
Also, on March 3, 2022, the Company entered into a registration rights agreement (the "Registration Rights Agreement") with the Investor, pursuant to which the Company agreed to register for resale the Shares and the shares of Common Stock underlying the Pre-Funded Warrants held by the Investor (the "Registrable Securities"). Under the Registration Rights Agreement, the Company has agreed to file a registration statement covering the resale of the Registrable Securities by no later than April 30, 2022 (the "Filing Deadline"). The Company has agreed to use commercially reasonable efforts to cause such registration statement to become effective as soon as practicable and to keep such registration statement effective until the date the Shares and the shares of Common Stock underlying the Pre-Funded Warrants covered by such registration statement have been sold or may be resold pursuant to Rule 144 without restriction. The Company has agreed to be responsible for all fees and expenses incurred in connection with the registration of the Registrable Securities.

In the event (i) the registration statement has not been filed by the Filing Deadline, (ii) the registration statement has not been declared effective prior to the earlier of (A) five business days after the date which the Company is notified by the U.S. Securities and Exchange Commission (the "SEC") that the registration statement will not be reviewed by the SEC staff or is not subject to further comment by the SEC staff, or (B) 60 days following the Filing Deadline (or, in the event the SEC reviews and has written comments to the registration statement, 120 days following the Filing Deadline) or (iii) after the registration statement has been declared effective by the SEC, sales cannot be made pursuant to the registration statement for any reason including by reason of a stop order or the Company’s failure to update such registration statement, subject to certain limited exceptions, then the Company has agreed to make pro rata payments to the Investor as liquidated damages in an amount equal to 1% of the aggregate amount invested by the Investor in the Registrable Securities per 30-day period or pro rata for any portion thereof for each such month during which such event continues, subject to certain caps set forth in the Registration Rights Agreement.
The Company has granted the Investor customary indemnification rights in connection with the registration statement. The Investor has also granted the Company customary indemnification rights in connection with the registration statement.

The foregoing description of the Registration Rights Agreement does not purport to be complete and is qualified in its entirety by reference to the Registration Rights Agreement, a copy of which is filed as Exhibit 10.2 hereto and incorporated by reference herein.