On May 11, 2022 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported first quarter 2022 financial results and provided a business update (Press release, Cyclacel, MAY 11, 2022, View Source [SID1234614218]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are pleased to report another productive quarter for Cyclacel, which included continued expansion of our three, registration-directed, clinical trials and publication of research findings supporting our drug development strategy," said Spiro Rombotis, Chief Executive Officer of Cyclacel. "Oral fadraciclib, our CDK2/9 drug candidate, is proving to be well tolerated in 065-101, our Phase 1/2 solid tumor and lymphoma study, having reached dose level 5 in the dose escalation stage which provides for daily dosing over 4 out of 4 weeks. In our PLK1 program, we have dosed the first patients in the streamlined Phase 1/2 trial of CYC140 for the treatment of solid tumors and lymphomas. We have optimized the properties of CYC140 to fit its apoptosis-driven mechanism, including short half-life and differentiated structural and biological properties, compared to other PLK1 inhibitors in development. We therefore believe CYC140 has the potential to demonstrate activity across a wide range of solid tumors, as a single agent and in combinations."

"A growing body of preclinical research supports the clinical development plan of fadraciclib. In April, we announced publication of research from The University of Texas MD Anderson Cancer Center highlighting fadraciclib’s antileukemic activity in CLL. Fadraciclib treatment resulted in suppression of MCL1, a key target protein. In addition, synergy of fadraciclib in combination with venetoclax was observed against primary CLL cell lines, including those with 17p deletion. With funding estimated through mid-2023, we are continuing to execute on our clinical development plan. We look forward to presenting initial fadraciclib clinical data in solid tumors and lymphomas, in the coming weeks, determining the recommended Phase 2 dose and entering proof of concept stage in the second half of 2022."

Key Highlights

·Fadraciclib 065-101 Phase 1/2 study in advanced solid tumors: Phase 1 dose escalation has reached dose level 5 (100mg given twice a day for 5 days for 4 weeks in a 4-week cycle) with a favorable patient safety profile and appropriate pharmacokinetic data observed thus far. The study is enrolling at four sites with several additional sites planning to join the proof-of-concept stage of this registration-directed study in 2H 2022. The Phase 2 part includes seven histologically defined cohorts thought to be sensitive to the drug’s mechanism: breast, colorectal (including KRAS mutant), endometrial/uterine, hepatobiliary, ovarian cancers and lymphomas. The study also includes an eighth basket cohort which will enroll patients regardless of histology with biomarkers relevant to the drug’s mechanism, including MCL1, MYC and/or cyclin E amplified.

·Fadraciclib 065-102 Phase 1/2 study in patients with leukemias or myelodysplastic syndromes. This study is now enrolling at City of Hope and MD Anderson Cancer Center and is treating patients at dose level one. Once the recommended Phase 2 dose (RP2D) for single-agent, oral fadraciclib is determined, the study will enter into proof-of-concept, cohort stage, where fadraciclib will be administered, both as a single agent and in combinations, to patients in up to seven cohorts relevant to the drug’s mechanism of action and informed by the clinical activity of fadraciclib in previous studies. Single-agent cohorts will include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) who have an inadequate response or have progressed on venetoclax combinations with hypomethylating agent (HMA) or low dose Ara C and relapsed/refractory AML or MDS patients with FLT3, KIT or MAPK pathways (including N and K RAS, BRAF, PTPN11, NF1). The trial will also include patients with CLL who have progressed after at least two lines of therapy including a BTK inhibitor and/or venetoclax.

Announced publication confirming fadraciclib suppresses MCL1 and synergizes with venetoclax in chronic lymphocytic leukemia. Results from the study confirmed that fadraciclib inhibited CDK9 mediated transcription, reduced levels of the short-lived, anti-apoptotic protein MCL1, and induced apoptosis in primary CLL cells. The data highlighted the importance of continuous treatment to prevent recovery of MCL1 protein levels. Furthermore, fadraciclib was shown to combine synergistically with the BCL2 antagonist, venetoclax, and demonstrated even greater synergy when targeted against 17p deleted CLL cells which were not sensitive to either agent alone.

·CYC140 140-101 Phase 1/2 study in solid tumors and lymphomas. This registration-directed study opened in April at City of Hope and MD Anderson Cancer Center and is enrolling patients in the Phase 1 dose escalation stage. The study uses a streamlined design and will initially determine RP2D for single-agent oral CYC140. Following RP2D, the trial will immediately enter into proof-of-concept, cohort stage, using a Simon 2-stage design. In this stage CYC140 will be administered to patients in up to seven mechanistically relevant cohorts including patients with bladder, breast, colorectal (including KRAS mutant), hepatocellular and biliary tract, and lung cancers (both small cell and non-small cell), as well as lymphomas plus an eighth basket cohort which will enroll patients with biomarkers relevant to the drug’s mechanism.

More information on our clinical trials can be found here.

Financial Highlights

As of March 31, 2022, cash and cash equivalents totaled $29.6 million, compared to $36.6 million as of December 31, 2021. Subsequent to the end of the first quarter, the Company received $3.6 million of United Kingdom research & development tax credits and $1.3 million in royalty receipts providing pro forma March 31, 2022, cash and cash equivalents of $34.5 million. The Company estimates that its available cash will fund currently planned programs through June 2023.

Research and development (R&D) expenses were $5.0 million for the three months ended March 31, 2022, as compared to $2.6 million for the same period in 2021. R&D expenses relating to fadraciclib were $3.6 million for the three months ended March 31, 2022, as compared to $1.7 million for the same period in 2021 due to increase in clinical trial costs associated with ongoing clinical trials evaluating fadraciclib in Phase 1/2 studies along with an increase in non-clinical expenditures. R&D expenses related to CYC140 were $1.1 million for the three months ended March 31, 2022, as compared to $0.7 million for the same period in 2021 due to clinical trial costs associated with the opening of clinical sites for CYC140 Phase 1/2 studies.

General and administrative expenses for the three months ended March 31, 2022, were $1.6 million, compared to $1.7 million for the same period of the previous year due to a decrease in professional and recruitment costs.

Total other income, net, for the three months ended March 31, 2022, was $1.3 million, compared to $0.1 million for the same period of the previous year. The increase of $1.2 million for the three months ended March 31, 2022, is primarily related to royalty income received from Thermo Fisher Scientific Corporation.

United Kingdom research & development tax credits were $1.1 million for the three months ended March 31, 2022, as compared to $0.7 million for the same period in 2021 as a direct consequence of increased qualifying research and development expenditure. Tax credit receipts of $3.6 million in respect of the financial year ended December 31, 2021, were received in May 2022.

Net loss for the three months ended March 31, 2022, was $4.1 million, compared to $3.5 million for the same period in 2021.

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.

On May 11, 2022 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) ("Aeterna" or the "Company"), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, reported its financial and operating results for the first quarter ended March 31, 2022 (Press release, AEterna Zentaris, MAY 11, 2022, View Source;b=2533&ID=108323&m=rl&g=1592 [SID1234614236]). The Company also provided an update on its pre-clinical and clinical development programs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We are beginning to see tangible progress related to the development programs we in-licensed throughout 2021. In particular, we are very pleased to report data with our pre-clinical program for AIM biologicals, which have recently been accepted for presentation at two scientific conferences. We remain focused advancing our pipeline, executing on our strategic priorities and unlocking the full potential of our pipeline and value for all stakeholders," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna.

Recent Highlights

Presented results from pre-clinical studies of Aeterna’s AIM Biologicals (Autoimmunity Modifying Biologicals) for the potential treatment of Parkinson’s Disease ("PD") at IMMUNOLOGY2022, the Annual Event of the American Association of Immunologists, held May 6-10, 2022.
Secured new European patent providing intellectual property protection of macimorelin in 27 countries within the European Union as well as additional European non-EU countries, such as the UK and Turkey, for macimorelin (Ghryvelin; Macrilen) for use to diagnose growth hormone deficiency (GHD) in adults.
Announced that results from pre-clinical studies of Aeterna’s AIM Biologicals for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") were accepted for presentation at the 13th International Congress on Autoimmunity to be held June 10-13, 2022 in Athens, Greece.
Pre-Clinical and Clinical Programs Update:

Therapeutics Development Pipeline

AIM Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") and Parkinson’s disease (PD)

AIM Biologicals utilize a novel mechanism which is believed to demonstrate that peptide antigens presented on immunosuppressive MHC class I molecules can selectively and efficiently induce antigen-specific tolerance. Based on this mechanism, the targeted immunomodulating therapeutics are being designed as optimized soluble molecules with the goal that they may be adapted to selectively induce tolerance to various autoantigens. With AIM Biologicals, the Company aims to restore the tolerance against such proteins to treat autoimmune diseases.

Pre-clinical studies conducted by the University of Wuerzburg, Germany thus far indicate that tolerance induction appears to be achieved via selective elimination of antigen-specific immune effector cells and via induction of antigen-specific regulatory T cells from naïve T cells. AIM Biologicals thus have the potential to become highly specific and effective yet not personalized treatments of NMOSD. Data from the pre-clinical studies will be presented at the 13th International Congress on Autoimmunity to be held June 10-13, 2022 in Athens, Greece.

For the treatment of NMOSD, it is believed that the AIM Biologicals will present a specific antigen derived from the water channel protein aquaporin-4 (AQP4) loaded to soluble immunoregulatory HLA-G protein to selectively induce immunological tolerance in the central nervous system.

For the development of AIM Biologicals as potential PD therapeutics, Aeterna plans to utilize, among others, an innovative animal model on neurodegeneration by α-synuclein-specific T cells in AAV-A53T-α-synuclein Parkinson’s disease mice, which has recently been published by University of Wuerzburg researchers. Additionally, the Company recently presented pre-clinical data demonstrating that corresponding AIM Biologicals prevented mobility impairments and postmortem histopathological assessment confirmed the induction of favorable in-situ immune cell composition and the rescue of substantia nigra neurons. The pre-clinical data confirmed that the translation potential of the approach deserves further exploration.

The University of Wuerzburg continues to bolster its intellectual property (IP) protection and has filed new IP on AIM-Biologicals related to both NMOSD and PD.

Next Steps – NMOSD

Conduct in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate.
Manufacturing process development for a selected candidate.
Next Steps – Parkinson’s Disease

Design and produce antigen-specific AIM Biologics molecules for the potential treatment of Parkinson’s disease.
Conduct in-vitro and in-vivo assessments in relevant Parkinson’s disease models.
Delayed Clearance Parathyroid Hormone ("DC-PTH") Fusion Polypeptides: Potential treatment for primary hypoparathyroidism

In March 2021, Aeterna entered into an exclusive patent and know-how license agreement and research agreement with The University of Sheffield, United Kingdom, for the intellectual property relating to DC-PTH fusion polypeptides with delayed clearance for all human uses. In consultation with The University of Sheffield, Aeterna has selected AEZS-150 as the lead candidate in its DC-PTH program. AEZS-150 is being developed with the goal of providing a potential new treatment option for primary hypoparathyroidism in adults.

The Company has selected a contract manufacturing organization for the development of its manufacturing for AEZS-150.

Next Steps

Work with The University of Sheffield to conduct in depth characterization of development candidate (in-vitro and in-vivo).
Ongoing development of manufacturing process.
Formalize pre-clinical development of AEZS-150 in preparation for a potential IND filing for conducting the first in-human clinical study.
Macimorelin Therapeutic: Ghrelin agonist in development for the treatment of ALS (Lou Gehrig’s disease)

In January 2021, the Company entered into a material transfer agreement with the University of Queensland, Australia, to provide macimorelin for the conduct of pre-clinical and subsequent clinical studies evaluating macimorelin as a potential therapeutic for the treatment of ALS (Lou Gehrig’s disease). The University of Queensland researchers have filed for supportive grants and aim to conduct pre-clinical studies in multiple pre-clinical models to demonstrate the therapeutic potential of macimorelin to slow disease progression and disease-specific pathology.

Macimorelin, a potent ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland. Acting via this mechanism, which was established during the development as a diagnostic test for growth hormone deficiency, it is believed that macimorelin may slow the progression of certain neurodegenerative diseases like ALS.

Apart from already available pre-clinical and clinical data on macimorelin for the development as a diagnostic, Aeterna may utilize the established supply chain to support this development. Alternative formulations are currently also under development, as a further option in addition to the existing oral solution already approved for the diagnostic use in adult growth hormone deficiency (AGHD).

Next Steps

Continue investigating macimorelin efficacy in an ALS specific SOD1 mouse model.
Assess alternative formulations.
Formalize pre-clinical development plan.
Diagnostics Development Pipeline

Macimorelin Diagnostic: Ghrelin agonist in development for diagnostic use in childhood-onset growth hormone deficiency ("CGHD")

Aeterna is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of CGHD.

Children and adolescents from two to less than 18 years of age with suspected growth hormone deficiency are to be included. The study is expected to include approximately 100 subjects in Europe and North America, with at least 40 subjects in pre-pubertal and 40 subjects in pubertal status. Macimorelin growth hormone stimulation test ("GHST") will be performed twice for repeatability data and two standard GHSTs will be used as controls: arginine (i.v.) and clonidine (p.o.).

On April 22, 2021, the U.S. FDA Investigational New Drug Application associated with this clinical trial became active.

The first clinical sites in the U.S. and in Europe are open for patient recruitment. In Europe, national clinical trial approval procedures and site initiation activities are ongoing. Site activation and patient enrollment continues to be impacted by the ongoing COVID-19 pandemic. The Company is actively monitoring delays to mitigate potential impact of COVID-19 on estimated trial completion dates. Additionally, clinical trial sites originally planned in the Ukraine and Russia are being halted due to the conflict in Ukraine intensifying following the Russian invasion. As a result, further delays with enrollment are expected as the DETECT-trial planned to recruit at least 25% (25 subjects) within those countries. Due to these circumstances and the resulting feasibility data from the Company’s CRO on potential options, Aeterna believes recruitment for the DETECT-trial may now continue until later into 2023.

The Company continues to advance its ongoing business development discussions to secure commercialization partners for macimorelin in additional markets. In addition to its previously established agreements, Aeterna recently entered into a license agreement with NK Meditech Ltd., for the development and commercialization of macimorelin in the Republic of Korea, and a distribution agreement with Er-Kim Pharmaceuticals Bulgaria EOOD for the commercialization of macimorelin in Turkey and some Balkan countries.

Vaccine Development Pipeline

Bacterial Vaccine Platform: Orally active, live-attenuated bacterial vaccine platform with potential application against viruses and bacteria, such as coronavirus types, including COVID-19 (SARS-CoV-2) and Chlamydia

In February 2021, Aeterna entered into an exclusive option agreement with the University of Wuerzburg to evaluate a pre-clinical, potential COVID-19 vaccine developed at the University of Wuerzburg. In March 2021, the Company exercised its option and entered into a license agreement where the Company was granted an exclusive, world-wide, license to certain patent applications and know-how owned by the University of Wuerzburg to research and develop, manufacture, and sell a potential COVID-19 vaccine. The Company’s vaccine platform is currently undergoing pre-clinical studies for the prevention of coronavirus diseases, including COVID-19 (SARS-CoV-2) with the planned start of clinical development targeted for H1 2023.

In September 2021, the Company exercised its option under the agreement with the University of Wuerzburg on a then undisclosed field, now known to be Chlamydia. Chlamydia trachomatis is a sexually transmitted bacterium infecting over 130 million subjects annually. Asymptomatic disease can spread to the reproductive tract eventually inducing infertility, miscarriage, or ectopic pregnancy, which is a life-threatening condition. Ocular infections can lead to inclusion conjunctivitis or trachoma, which is the primary source of visual impairment or infectious blindness. Additionally, Prof. Dr. Thomas Rudel of the University of Wuerzburg was engaged by the Company in September 2021 as a scientific consultant to support development of the salmonella-based vaccine platform for the coronavirus and Chlamydia vaccines.

Recently, the Company expanded its research agreement with the University of Wuerzburg to conduct supplementary research activities and pre-clinical development studies on the potential vaccines, the results of which are covered within the scope of the license agreements. Under the expanded research program, the University of Wuerzburg will validate and utilize innovative human 3D intestinal tissue models to study the infection biology of Salmonella strains towards clinical development.

Next Steps – Coronavirus Vaccine

Evaluate administration route, dose and immunization scheme.
Initiate in-vivo immunology experiments with antigen variant candidates in relevant mice models.
Conduct virus challenge experiments in immunized transgenic animals.
Start manufacturing process assessment / development.
Conduct pre-clinical safety and toxicology assessment.
Next Steps – Chlamydia Vaccine

Design and prepare candidate vaccine strains.
Evaluate administration route, dose and immunization scheme.
Conduct In-vivo immunology experiments with candidate strains in relevant mouse models.
Summary of First Quarter 2022 Financial Results

All amounts in this press release are in U.S. dollars unless otherwise noted.

Results of operations for the three-month period ended March 31, 2022

For the three-month period ended March 31, 2022, we reported a consolidated net loss of ($2.6 million), or ($0.02) net loss per common share (basic), as compared with a consolidated net loss of ($1.5) million, or ($0.02) net income per common share (basic) for the three-month period ended March 31, 2021. The $1.1 million increase in net loss is primarily due to an increase of $1.3 million in total operating costs, $0.2 million decline in total revenues and offset by favorable foreign currency exchange rates of $0.4 million

Revenues

Our total revenue for the three-month period ended March 31, 2022 was $1.5 million as compared with $1.7 million for the same period in 2021, representing a decline of $0.2 million. The 2022 revenue was comprised of $0.43 million in licensing revenue (2021 – $0.52 million), $1.0 million in development revenue (2021 – $1.1), $0.04 million in supply chain revenue (2021 – $0.04 million), $0.02 million in royalty income (2021 – $0.01 million). and $0.06 in product sales (2021 – $nil)
Operating expenses

Our total operating expense for the three-month period ended March 31, 2022 was $4.3 million as compared with $3.0 million for the same period in 2021, representing an increase of $1.3 million. This increase arose primarily from a $0.9 increase research and development, $0.3 million increase in general and administrative expenses and an increase of $0.1 million in selling expenses.
Net finance (costs) income

Our net finance (costs) for the three-month period ended March 31, 2022 was $0.2 million as compared with net finance cost of $(0.3) million for the same period in 2021, representing an increase in net finance income of $0.5 million.
The Company had $63.6 million cash and cash equivalents at March 31, 2022 (December 31, 2021 – 65.3 million).

Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the Management’s Discussion and Analysis of Financial Condition and Results of Operations for the fourth quarter and full year 2021, as well as the Company’s unaudited consolidated interim financial statements as of December 31, 2021, will be available on the Company’s website (www.zentaris.com) in the Investors section or at the Company’s profile at www.sedar.com and www.sec.gov.

On May 10, 2022 Iktos, a company specialized in Artificial Intelligence (AI) for new drug design and Sygnature Discovery, a leading integrated drug discovery CRO headquartered in Nottingham, UK, with expertise across a range of therapeutic and biological target classes reported a collaboration agreement in AI for new drug design (Press release, Iktos, MAY 10, 2022, View Source [SID1234614046]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Under the 3-year agreement, Sygnature will deploy Iktos’ de novo generative design software Makya, which will be used by Sygnature scientists to facilitate rapid and efficient design of novel compounds and accelerate hit-to-lead/lead optimisation, reinforcing Sygnature Discovery’s drug design expertise delivering its integrated drug discovery solutions to its world-wide customer base.

In the recent years, Iktos has emerged as one of the world leaders in AI for drug design, establishing multiple collaborations with renowned pharmaceutical companies and successfully developing the AI software platforms Makya for new drug design and Spaya for synthesis planning.

Makya, a generative AI-driven de novo design software for Multi-Parametric Optimization (MPO), is available either as a SaaS platform or for implementation on customer premises or in the customer’s Virtual Private Cloud (VPC). Makya’s user-friendly interface enables it to be used by medicinal or computational chemists, and Makya can also be operated as a Python package through a Jupyter notebook interface. Makya is based on Iktos’ generative AI technology, which helps bring speed and efficiency to the drug discovery process by automatically designing virtual novel molecules that have desired activities for treating a given disease. It is a novel solution, validated through many collaborations, to one of the key challenges in drug design: the rapid identification of molecules that simultaneously satisfy multiple parameters, such as potency, selectivity, safety, and project-specific properties.

Sygnature Discovery serves its growing customer base with fully integrated drug discovery services from target validation through to preclinical candidate selection. The company has a relentless focus on quality and continually looks to improve on its ability to deliver novel therapeutics to the clinic.

Colin Sambrook-Smith, Director of Computational Sciences at Sygnature Discovery commented:

"Late stage lead optimisation projects routinely generate substantial data sets which are ideally placed for exploitation by AI and Machine Learning technologies. Our experience with the Iktos Makya AI/ML technology demonstrates that it generates high quality compound ideas, the QSAR models can be rapidly updated, and the interface allows us to distribute the software broadly and quickly. We believe that Makya will allow us to blend our proven medicinal and computational chemistry expertise with the benefit of AI/ML technologies to impact compound design. This is why we have selected Makya to enhance how we help our customers with their lead optimisation projects, with a view to reducing the number of compounds required to identify pre-clinical candidates and so keep overall costs and timelines down."

"We are very pleased to collaborate with Sygnature Discovery, they truly are a leading integrated drug discovery CRO and with a great track record. We are excited and proud to announce our first multi-year collaboration deal in the dynamic CRO sector and to have Sygnature’s scientists use our software to speed up their customers’ discovery programs," said Yann Gaston-Mathé, Co-founder and CEO of Iktos. "We are more than ever committed to make our technology available to biopharma companies around the world and maximize the impact of AI on the productivity of drug discovery."

On May 10, 2022 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal second quarter ended March 31, 2022 (Press release, Arrowhead Research Corporation, MAY 10, 2022, View Source [SID1234614078]). The company is hosting a conference call today, May 10, 2022, at 4:30 p.m. ET to discuss the results.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

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 3791265.

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 3791265.

Selected Recent Events

Initiated the PALISADE Phase 3 clinical study to evaluate the efficacy and safety of ARO-APOC3, Arrowhead’s investigational RNA interference (RNAi) therapeutic designed to inhibit the production of apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism, in adults with familial chylomicronemia syndrome
Completed enrollment of 204 patients in the Phase 2b ARCHES-2 clinical study of ARO-ANG3, our investigational medicine designed to reduce production of angiopoietin-like protein 3 ANGPTL3 as a potential treatment for patients with mixed dyslipidemia
ARCHES-2 is expected to be complete around the end of 2022 and topline data are anticipated to be available in the first half of 2023
Initiated the Phase 2 GATEWAY clinical study of ARO-ANG3, Arrowhead’s investigational medicine designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3), in patients with homozygous familial hypercholesterolemia (HoFH)
Initiated a Phase 1/2 study of ARO-C3, Arrowhead’s investigational medicine designed to reduce production of complement component 3 (C3) as a potential therapy for various complement mediated diseases, in up to 24 adult healthy volunteers, up to 24 adult patients with paroxysmal nocturnal hemoglobinuria (PNH), and up to 14 adult patients with complement-mediated renal disease
Filed Clinical Trial Applications (CTA) requesting regulatory clearance to begin clinical studies for two new investigational medicines designed to treat various muco-obstructive and inflammatory pulmonary conditions
ARO-MUC5AC, an investigational RNAi therapeutic designed to inhibit the production of mucin 5AC (MUC5AC)
ARO-RAGE, an investigational RNAi therapeutic designed to inhibit the production of Receptor for Advanced Glycation End products (RAGE)
Formed a joint venture, Visirna Therapeutics, with Vivo Capital to expand the reach of innovative medicines in Greater China
Broke ground on construction of a new drug manufacturing facility and announced awards of up to $18.5 million in tax incentives from the city of Verona and the Wisconsin Economic Development Corporation

On May 10, 2022 Bio-Techne Corporation (NASDAQ:TECH) reported that Exosome Diagnostics, a Bio-Techne brand, will have a major scientific presence at the annual American Urological Association Conference (AUA) taking place May 13-16 in New Orleans, LA (Press release, Bio-Techne, MAY 10, 2022, View Source [SID1234614094]). Exosome Diagnostics will have two poster presentations, including a podium presentation, six in-booth scientific presentations, and a live symposium with American icon, National Baseball Hall of Famer, and former Baltimore Orioles player "Iron Man" Cal Ripken, Jr. with leading urologist, Dr. Ronald Tutrone of Medical Director of Clinical Research at Chesapeake Urology in Baltimore, Maryland.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Scientific presentations include:

A Combined Biomarker/mpMRI Approach Provides Enhanced Clinical Information Prior to Prostate Biopsy by Dr. Alexander Kretschmer et al. on Friday, May 13, 2022, 1:00 pm – 3:00 pm, Room 245, Ernest N. Morial Convention Center

Development of Exosome-based Plasma RNA Biomarkers for High-Risk Prostate Cancer by Dr. Sandra Gaston, et al. on Saturday, May 14, 2022, 10:30 am – 11:45 am, Room 222, Ernest N. Morial Convention Center

Validation of a urinary exosome mRNA signature for the diagnosis of human kidney transplant rejection by Dr. Rania El Fakih on Sunday, May 15, 2022, 7:00 am – 8:15 am, Room 228, Ernest N. Morial Convention Center

Exosome’s Chief Scientific Officer, Dr. Johan Skog, will lead the in-booth scientific presentation series with recently published results of the association of the ExoDx Prostate (EPI) test with post-prostatectomy outcome. Additional presentations include results of the ExoDx Prostate test clinical utility study as well as a 2.5 year follow-up and interim analysis of subjects who participated in the study and will conclude with the combined performance of the ExoDx Prostate test and MRI in the early detection of prostate cancer.

Cal Ripken, Jr. returns to partner with Exosome Diagnostics and Bio-Techne as an ambassador and advocate of the ExoDx Prostate test, which became part of Cal’s own prostate cancer journey. During his live appearances at the AUA, Cal will use his high-profile platform and strong reputation among men in his age group to raise awareness of the importance of early detection of prostate cancer.

"We are so pleased to have a robust scientific presence at the AUA, and excited to continue our campaign with an American baseball legend, Cal Ripken, Jr.," stated Kim Kelderman, President of Diagnostics and Genomics Segment of Bio-Techne. "Cal is a great example of the powerful role our ExoDx Prostate test plays in the prostate cancer journey. We look forward to continuing to provide this important tool to the millions of men and their families who are facing difficult choices in the management of this disease."

One in nine American men will be diagnosed with prostate cancer during his lifetime. Prostate cancer (PCa) is the second leading cause of cancer death among American men and is the most commonly diagnosed cancer. The American Cancer Society estimates that 268,000 men will be diagnosed with prostate cancer in 2022 and projects that ~34,000 men will die from the disease. Currently, nearly 3.6 million American men are living with the disease – only slightly less than the population of Los Angeles.

The EPI test is a urine-based genomic test that helps inform the prostate biopsy decision. This liquid biopsy test has a positive coverage decision from Medicare and is included in the National Comprehensive Cancer Network (NCCN) guidelines for early detection of prostate cancer in men for both initial and repeat biopsy. It is performed by Exosome Diagnostics in its CLIA, ISO, NY certified and CAP-accredited laboratory located in Waltham, Massachusetts. The EPI test is a risk assessment tool that assists physicians and their patients with determining if a prostate biopsy is needed when presented with an ambiguous PSA test result, thereby reducing complications from unnecessary and invasive procedures. The ExoDx Prostate is the leading test for at-home collection, enabling thousands of patients unable to travel to their healthcare professional for a routine office visit to assess prostate cancer risk.