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.

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 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune and inflammatory diseases, reported the company will participate in a fireside chat at the 4th Annual Evercore ISI HealthCONx Conference on Wednesday, December 1st, 2021, at 12:35 p.m. ET/9:35 a.m. PT (Press release, Alpine Immune Sciences, NOV 22, 2021, View Source [SID1234595899]).

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A live webcast of the fireside chat will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

On November 22, 2021 EpiAxis Therapeutics reported that it will be taking its new corporate presentation to the J.P. Morgan 40th Annual Healthcare Conference (Press release, EpiAxis Therapeutics, NOV 22, 2021, View Source;utm_medium=rss&utm_campaign=epiaxis-heads-to-the-jp-morgan-40th-annual-healthcare-conference [SID1234595898]).

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The conference will be held in San Francisco from January 10-13, 2022.

"The J.P. Morgan 40th Annual Healthcare Conference is the largest and most informative health care investment symposium in the industry," said EpiAxis CEO Dr Jeremy Chrisp. "It connects global industry leaders, emerging fast-growth companies, innovative technology creators and members of the investment community."

In 2021, public and private companies delivered presentations to more than 8000 conference participants.

For 2022, AusBiotech is hosting a virtual Australian Showcase during the conference, highlighting companies at the forefront of medical innovation. EpiAxis will share its new corporate presentation with global investors and potential partners at this conference.

Taiwan biotech forum

Together with AusBiotech, EpiAxis attended the Australia-Taiwan Biotech Investment & Partnership Forum in early November. Organised by the Australian Office Taipei and the Biotechnology and Pharmaceutical Industries Promotion Office (BPIPO), the forum aimed to connect the biotech and pharma industries in the two nations.

More than 60 guests joined the forum, which was held during the BioTaiwan Exhibition, including several major biotech companies, venture capitalists, BIO industry members, event partners and journalists from bio magazines.

"It was exciting to share the latest updates from EpiAxis with industry and investors in Taiwan," said Dr Chrisp. "There is great potential for our epigenetics pipeline in the Asian market and we look forward to following up on the valuable connections that were made during the forum."

On November 22, 2021 AB Science SA (Euronext – FR0010557264 – AB) reported that its Phase I/II study (AB18001) evaluating AB8939 in patients with refractory and relapsed AML and refractory myelodysplastic syndrome (MDS) has received Investigational New Drug (IND) approval by the U.S. Food and Drug Administration (FDA) (Press release, AB Science, NOV 22, 2021, View Source [SID1234595896]).

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Study AB18001 is titled ‘A Phase 1/2 Study to Assess the Safety, Pharmacokinetics, and Efficacy of Daily Intravenous of AB8939 in patients with Relapsed/Refractory Acute Myeloid Leukemia’. The study has a multi-stage design. The first part is a dose escalation study that aims to determine the safety and tolerability of intravenous AB8939 in patients with refractory or relapsed AML or patients with refractory MDS, and to determine the recommended dose for the second-stage dose expansion study. This dose expansion study aims to determine the schedule for a Phase 2 trial in patients with relapsed/refractory AML and to also provide an early efficacy (response rate) assessment of AB8939.

Study AB18001 has also been approved in France and in Canada.

AB8939 is a new synthetic microtubule-destabilizing drug. Preclinical data show that AB8939 has broad anticancer activity, with a notable advantage over standard chemotherapies that target microtubules of being able to overcome P-glycoprotein (Pgp) and myeloperoxidase (MPO) mediated drug resistance. Development of drug resistance often restricts the clinical efficacy of microtubule-targeting chemotherapy drugs (for example, taxanes and vinca alkaloids); thus, AB8939 has strong potential to be developed in numerous oncology indications.

The first indication AB8939 is being developed for is acute myeloid leukemia (AML). Cytarabine (Ara-C) and azacytidine are standard chemotherapies for AML treatment, however, drug resistance is a major limitation to successful therapy. In vivo data from a highly resistant Ara-C patient derived xenograft (PDX) mouse model showed that AB8939, administered alone or in combination with Ara-C, increased survival relative to single agent Ara-C, with an accompanying significant reduction of blasts in blood and decrease in tumor growth. Further evidence of therapeutic potential was demonstrated using an azacytidine resistant PDX model with AB8939, administered alone or in combination with azacytidine, showing a significant reduction of blasts relative to single agent azacytidine. Moreover, while azacytidine was associated with strong treatment related hematotoxicity, AB8939 did not induce hematotoxicity throughout its 4-week treatment period.

AB8939 was granted orphan drug designation for AML from the U.S. Food and Drug Administration (FDA).

AB8939 was entirely discovered by the laboratories of AB Science, which retains full ownership of intellectual rights, and is an example of AB Science’s focus on innovative drug development focused on improving patients’ lives.

About acute myeloid leukemia (AML)
Acute myeloid leukemia (AML) is a serious, life-threating condition and the most common cause of leukemia-related mortality, with a majority of patients facing a highly unsatisfactory prognosis. As such, AML represents an unmet medical need, with limited therapeutic options for patients who are refractory or too frail to benefit from potentially curative but highly toxic treatment, or for those patients that have relapsed following a first complete response. The prevalence of AML in western countries is around 1 per 5,000 persons, corresponding to around 100,000 cases in Europe and 60,000 in the USA. Among AML patients, it is estimated that approximately 50% of the patients will not have stem cell transplantation and will relapse. Therefore, the estimated targeted population of AB8938 in AML is around 80,000 people in Europe and the US.