On November 22, 2021 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal year ended September 30, 2021 (Press release, Arrowhead Research Corporation, NOV 22, 2021, View Source [SID1234595900]). The company is hosting a conference call today, November 22, 2021, at 4:30 p.m. ET to discuss the results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 8074256.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 8074256.

Selected Recent Events

Entered into an exclusive license agreement with GlaxoSmithKline (GSK) under which GSK will develop and commercialize ARO-HSD, Arrowhead’s investigational RNA interference (RNAi) therapeutic in a Phase 1/2 trial that is currently being developed as a treatment for patients with nonalcoholic steatohepatitis (NASH)
Presented new clinical data at The Liver Meeting, the Annual Meeting of the American Association for the Study of Liver Disease (AASLD), for the following investigational candidates:
JNJ-73763989 (JNJ-3989), formerly called ARO-HBV, being developed by collaborator Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen)
ARO-HSD, the investigational RNAi therapeutic being developed as a treatment for patients with NASH and recently licensed to GSK
ARO-AAT, also known as TAK-999, the investigational RNAi therapeutic being co-developed with Takeda Pharmaceutical Company Limited as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency
Presented additional Phase 1/2 clinical data on ARO-APOC3, the investigational RNA RNAi therapeutic targeting apolipoprotein C-III (APOC3) being developed as a treatment for patients with hypertriglyceridemia, severe hypertriglyceridemia, and familial chylomicronemia syndrome, at the American Heart Association (AHA) Scientific Sessions 2021
Initiated and began dosing patients in AROAPOC3-2002, now called MUIR, a Phase 2b clinical study of ARO-APOC3
Initiated and began dosing patients in AROANG3-2001, now called ARCHES-2, a Phase 2b clinical study of ARO-ANG3, the company’s investigational RNAi therapeutic being developed as a treatment for patients with mixed dyslipidemia
Received Breakthrough Therapy designation from the U.S. Food and Drug Administration for ARO-AAT
Announced that Janssen disclosed its collaboration with Arrowhead on investigational compound JNJ-75220795, which in a Phase 1 clinical study and is designed to reduce expression in the liver of patatin like phospholipase domain containing 3 (PNPLA3) as a potential treatment for patients with NASH
Earned a $10 million milestone from Janssen after Janssen dosed the fifth patient in a Phase 1 clinical study
Filed a CTA to begin clinical studies of previously undisclosed candidate ARO-C3, an investigational therapeutic designed to reduce production of complement component 3 as a potential therapy for various complement mediated diseases, and hosted a key opinion leader webinar to discuss the complement pathway and the diseases Arrowhead will initially focus on

On November 22, 2021 NOXXON Pharma N.V. (Euronext Growth Paris: ALNOX) (Paris:ALNOX), a biotechnology company focused on improving cancer treatments by targeting the tumor microenvironment (TME), reported that new data from the ongoing Phase 1/2 GLORIA trial with NOX-A12 and radiotherapy in brain cancer (glioblastoma multiforme, GBM) were presented at the Society for Neuro-Oncology (SNO) Annual Meeting (Press release, NOXXON, NOV 22, 2021, View Source [SID1234595917]). The presentation was held by Frank A. Giordano, M.D., Director and Chair of the Department of Radiation Oncology, University Hospital Bonn, Germany, and lead investigator of the ongoing GLORIA study.

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The oral presentation, entitled "CXCL12 inhibition in MGMT unmethylated glioblastoma – results of an early proof-of-concept assessment in the multicentric phase I/II GLORIA trial", included results from 9 chemotherapy refractory (MGMT promoter unmethylated) patients participating in the proof-of-concept study on CXCL12 inhibition during and after radiotherapy of glioblastoma. Eight of 9 patients (89%) receiving NOX-A12 showed reductions in tumor size (2 patients with objective responses [>50% reduction] and 6 patients with stable disease [<50% reduction], while one patient progressed. These results compare favorably with historic patient outcomes from a matched cohort that received standard of care, where only 1 out of 13 patients (8%) showed a reduction in tumor size with an objective response and 12 patients’ tumors progressed.

Also, data from tissue analysis of a patient on NOX-A12 therapy shows [1] a significant reduction of the NOX-A12 target, CXCL12, on tumor blood vessels, [2] a significant decrease in tumor cell proliferation and [3] an increase in tumor infiltration of activated killer immune cells. Interestingly and very importantly, such benefits were observed across all available tumor tissue and not only in small subsections.

These benefits are strongly supportive of the dual mechanism of action of NOX-A12:
– inhibiting repair of blood vessels damaged by radiotherapy
– promoting of immune-response
This dual mechanism of action could prove transformational since this is not consistently observed in historical samples including patients treated with immune checkpoint inhibitors.

"The data presented by Dr. Giordano at the SNO meeting are a significant step forward in bringing NOX-A12 to glioblastoma patients. While a diagnosis of chemotherapy-resistant glioblastoma leads almost inevitably to systematic rapid progression of the disease, NOX-A12 combined with radiotherapy managed to achieve stable disease or an objective response in 8 out of the 9 patients. We very much look forward to presenting and explaining the transformational nature of these new data at our upcoming KOL event on Tuesday, November 23, when Dr. Giordano will also be available to answer questions," commented Aram Mangasarian, CEO of NOXXON.

Details of the Key Opinion Leader webinar are as follows:
Title: NOX-A12 and Radiotherapy combination: A Differentiated and Promising New Approach to Treating Brain Cancer
Presenter: Dr. Frank A. Giordano, Director and Chair of the Department of Radiation Oncology, University Hospital Bonn, Germany
Webinar time and date: November 23, 2021 at 02:00 p.m. CET (08:00 a.m. EST)
Registration: To register for the event, please click here

NOX-A12 acts via a unique mechanism of action, which was confirmed by the presented results: by removing the CXCL12 chemokine from the tumor blood vessels the revascularization of the irradiated tumor area is blocked and a significant increase in activated killer immune cell infiltration to the tumor can be seen.

More information about the GLORIA study can be found at ClinicalTrials.gov number NCT04121455.

Dr. Frank A. Giordano, is Director and Chair of the Department of Radiation Oncology at the University Hospital Bonn, Germany. He is an expert in precision radiation therapy and intraoperative irradiation of malignant tumors and has received international recognition for his brain tumor research, including an award from the American Society of Radiation Oncology (ASTRO) and an honorary membership of the Spanish Society of Radiation Oncology (SEOR). Dr. Giordano received his medical degree from the University of Heidelberg, Germany, and did his post-doctoral training as a Peter Engelhorn fellow at the German Cancer Research Center (DKFZ). He received clinical training at the National Center for Tumor Diseases (NCT) Heidelberg and the University Medical Center Mannheim, where he served as acting chairman and director of the Department of Radiation Oncology before moving to Bonn. For many years, his research has focused on optimized radiation therapy of brain cancers to offer cancer patients personalized and even more effective treatment. As one of the few Else-Kröner-Fresenius Excellence Fellows, Dr. Giordano developed innovative therapy options that even found their way in clinical practice. He sees great potential in the combination of radiotherapy and immunomodulatory therapy.

On November 22, 2021 DNAtrix, a biotech company advancing virus-driven immunotherapies for cancer, reported that data from the Phase 1 study of DNX-2401 in diffuse intrinsic pontine glioma (DIPG) was presented in an oral presentation at the 26th Annual Meeting and Education Day of the Society for Neuro-oncology (SNO), which was held from November 18-21, 2021 in Boston, MA (Press release, DNAtrix, NOV 22, 2021, View Source [SID1234595934]). DNX-2401 is an adenovirus-based immunotherapy that is engineered to selectively kill tumor cells and trigger a robust anti-tumor immune response. It has received FDA Fast Track and Rare Pediatric Disease designations for DIPG.

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"DIPG is a rapidly progressing tumor with the worst prognosis of any pediatric cancer," said Jeffrey Knapp, chief executive officer of DNAtrix. "Patients receiving conventional radiation therapy typically have a median overall survival of between eight and eleven months, with less than ten percent of the patients reaching the two-year survival mark. The preliminary overall survival data from the DNX-2401 Phase 1 study show a median survival of close to 18 months, with three patients still in follow-up. This is a significant and remarkable impact on overall survival in this difficult-to-treat tumor type, and we look forward to completing the study."

The Phase 1 study evaluated DNX-2401 followed by conventional radiation therapy in 12 newly diagnosed DIPG patients. Tumor reductions were reported for 9 patients (75%), including 3 confirmed (25%) responses per RAPNO criteria. As of the data cutoff, median overall survival was 17.8 months with follow-up ongoing for three patients. No dose-limiting toxicities were observed, and the treatment regimen was well-tolerated.

Oral Presentation Details:

Title:

Safety, efficacy, and survival results from a Phase 1 study of the oncolytic adenovirus DNX-2401 followed by standard of care radiotherapy for newly diagnosed diffuse intrinsic pontine glioma (DIPG)

Abstract Number:

CTIM-08

Presenter:

Jaime Gallego Perez-Larraya, M.D.

Date/Time:

November 21, 2021 at 12:00PM EST

About DNX-2401
DNX-2401 is an oncolytic adenovirus engineered specifically to infect, replicate in, and directly kill cancer cells, as well as elicit a broader anti-tumor immune response. DNX-2401 is currently being evaluated as a potential treatment for highly aggressive brain tumors, including recurrent glioblastoma in adults and newly-diagnosed diffuse intrinsic pontine glioma (DIPG) in children. Clinical studies have demonstrated that DNX-2401 was well tolerated and extended survival for patients with recurrent glioblastoma. DNX-2401 has been granted Fast Track for recurrent glioblastoma and Orphan designation by the FDA and PRIME and Orphan designation by the EMA for high grade glioma, as well as Fast Track and Rare Pediatric Disease designations by the FDA for DIPG.

About DIPG
Diffuse intrinsic pontine glioma (DIPG), also known as diffuse midline glioma, is a rare and highly aggressive infiltrative tumor of the brainstem with the worst prognosis of any pediatric cancer. No effective treatments are available and novel treatment approaches are needed.

On November 22, 2021 Lantern Pharma Inc. (NASDAQ: LTRN) ("Lantern" or the "Company"), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform the cost, pace, and timeline of oncology drug discovery and development, reported that its board of directors has authorized a share repurchase program to acquire up to $7 million of the Company’s common stock (Press release, Lantern Pharma, NOV 22, 2021, View Source [SID1234595882]). The Company may purchase common stock on the open market, through privately negotiated transactions, or otherwise, in compliance with the rules of the United States Securities and Exchange Commission and other applicable legal requirements. As of September 30, 2021, the Company had approximately $73.8 million of cash, cash equivalents and marketable securities. The Company had approximately 11.2 million shares of common stock outstanding as of October 29, 2021.

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"Initiating a share repurchase program at this time is in line with our ongoing focus on creating value for our stockholders, which we are committed to," stated Panna Sharma, CEO and President of Lantern Pharma Inc. "It also demonstrates our confidence in advancing our clinical pipeline and our growing RADR A.I. platform — Lantern is capitalized to achieve its upcoming clinical milestones. In light of our strong balance sheet, the board has decided to implement the share repurchase program enabling Lantern to opportunistically return value to its stockholders."

The timing, amount of shares repurchased and prices paid for the stock under this program will depend on market conditions as well as corporate and regulatory limitations, including blackout period restrictions. The repurchase program does not obligate the Company to acquire any particular amount of shares, and the repurchase program may be suspended or discontinued at any time at the Company’s discretion.

On November 22, 2021 Catapult Therapeutics, a clinical-stage biopharmaceutical company developing novel cancer treatments, reported dosing of the first patient in the company’s Phase I clinical trial of CAP-100 in patients with relapsed and/or refractory chronic lymphocytic leukemia (CLL) (Press release, Catapult Therapeutics, NOV 22, 2021, View Source [SID1234595901]). The trial will enroll an estimated number of 25 patients and is performed in 3 Academic Sites in the US. CAP-100 is Catapult’s lead product candidate, an anti-CCR7 antibody with a unique mechanism of action interfering with the fundamental pathology of hematological malignancies. CAP-100 prevents cancer cells from entering and hiding in lymph nodes in addition to being able to kill the cancer cells directly. This unique mode of action is directly preventing cancers cells to escape into niches where they are more difficult to eradicate.

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"The dosing of the first patient with CAP-100 is an important step forward in the development of our lead product. The pre-clinical data of CAP-100 is very promising, and we are looking forward to receiving data that potentially confirms what we believe CAP-100 could become: an effective treatment for patients with hematological malignancies and with the potential to change the treatment paradigm for different cancers," says Dominik Höchli, MD, CEO of Catapult Therapeutics.

The first in-human clinical trial was designed in close collaboration with the Dana-Farber Cancer Institute in Boston. "The CCR7 signaling pathway is a major driver directing cells to the lymph node. Targeting this pathway with CAP-100 has the potential to lead to more rational and effective therapies for CLL and other malignancies," says Jennifer R. Brown, MD, PhD, Director of the CLL Center of the Division of Hematologic Malignancies at Dana-Farber Cancer Institute, Professor of Medicine at Harvard Medical School in Boston, Massachusetts and the principal investigator of the phase 1 study.

CAP-100 is an anti-CCR7 antibody with a unique and biologically independent therapeutic mechanism of action for treatment of CCR7-positive hematological malignancies. Preclinical studies have demonstrated CAP-100’s unique ability to treat primary tumor cells from patients with human CLL, and different non-Hodgkin lymphomas (NHL) at a fundamental point, by interrupting tumor cell migration to and survival in lymph nodes. In addition, CAP-100 provides strong cell killing while preserving normal blood cells and inhibition of survival of tumor cells in the lymph nodes. Furthermore, CAP-100 may also be an effective treatment for other CCR7-expressing B- and T-cell leukemias and lymphomas as well as for graft-versus-host disease.

About the study

Catapult Therapeutic’s Phase I clinical trial (clinicaltrials.gov #NCT04704323) will investigate the safety and efficacy of increasing doses of CAP-100 in relapsed or refractory patients to at least two prior standard systemic treatment regimens for CLL or SLL (small lymphocytic lymphoma) and having no available therapies known to provide clinical benefit. The trial will be divided into two phases. The aim of the Phase Ia (dose escalation) is to define the Recommended Phase 2 Dose (RP2D). Phase Ib of the trial (expansion phase) will evaluate the safety and preliminary clinical benefit of CAP-100 at RP2D, to support the design of future trials investigating CAP-100 in earlier lines of CLL and in other malignancies either as monotherapy or in a combination setting.

About CCR7 & CAP-100

The chemokine receptor CCR7 is essential for the migration, maturation, and survival of CCR7 expressing cells to lymphoid organs. This pivotal receptor is over-expressed in hematological malignancies with lymph node involvement, such as CLL and diffuse large B-cell lymphoma (DLBCL) amongst others. Anti-hCCR7 antibody CAP-100 offers a unique and biologically independent therapeutic mechanism to treat these hematological cancers at a fundamental point, by interrupting tumor cell migration to lymph nodes. In addition, CAP-100 provides strong cell killing and inhibition of survival of tumor cells in the lymph nodes.

About CLL

Despite advances in therapy and improved outcome, in most instances CLL is an incurable disorder, and most patients relapse or become refractory to their treatment. CLL is the most common type of leukemia in Western countries, predominates in the elderly, and the incidence of the disease increases exponentially with age. Thus, the number of CLL patients is expected to rise in the future, given the increase in the aging population, bringing to light new clinical challenges and public health issues.