Arrowhead Pharmaceuticals Reports Fiscal 2023 First Quarter Results

On February 6, 2023 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal first quarter ended December 31, 2022 (Press release, Arrowhead Research Corporation, FEB 6, 2023, View Source [SID1234626876]). The company is hosting a conference call today, February 6, 2023, 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 A replay of the webcast will be available approximately two hours after the conclusion of the call.
For analysts that wish to participate in the conference call, please register at https://register.vevent.com/register/BI9b2661110f7a4b0ebc648bf42d2a403f. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.
Selected Recent Events
•Announced topline results with Takeda from the Phase 2 SEQUOIA clinical study of investigational fazirsiran for the treatment of liver disease associated with alpha-1 antitrypsin deficiency and provided an outline of a Phase 3 study that was co-developed by Takeda and Arrowhead and will be conducted by Takeda. Key results from SEQUOIA included the following:
◦Fibrosis regression observed in 50% of patients receiving fazirsiran
◦Median reductions of 94% of Z-AAT accumulation in the liver and mean reduction of 68% in histologic globule burden
◦Treatment emergent adverse events were generally well balanced between fazirsiran and placebo groups
◦Results consistent with AROAAT-2002 open-label study previously published in The New England Journal of Medicine
•Earned a $25 million milestone payment from Amgen after the first subject was enrolled in Amgen’s Phase 3 trial of olpasiran for the treatment of cardiovascular disease
•Earned a $15 million milestone payment from Horizon Therapeutics after the first subject was enrolled in Horizon’s Phase 1 study of HZN-457, formerly called ARO-XDH, for the treatment of gout
•Initiated dosing in AROMMP7-1001 (NCT05537025), a Phase 1/2a single ascending dose and multiple ascending dose clinical study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-MMP7, an investigational RNAi therapeutic designed to reduce expression of matrix metalloproteinase 7 (MMP7) as a potential treatment for idiopathic pulmonary fibrosis (IPF), in up to 56 healthy volunteers and in up to 21 patients with IPF

•Presented interim data on the cardiometabolic pipeline in three late-breaking oral presentations at the American Heart Association (AHA) Scientific Sessions 2022 and at a virtual analyst and investor event. Key results included the following:
◦ARO-APOC3, an investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3) being developed as a treatment for patients with cardiovascular disease, severe hypertriglyceridemia (SHTG), and familial chylomicronemia syndrome (FCS), decreased triglycerides by 86%, and non-HDL-C by 45% while increasing HDL-C by 99% in patients with severe hypertriglyceridemia
◦ARO-ANG3, an investigational RNAi therapeutic designed to silence the hepatic expression of angiopoietin-like protein 3 (ANGPTL3) being developed as a treatment for patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH), decreased triglycerides by 59%, LDL-C by 32%, and was associated with a relative reduction in liver fat fraction in patients with mixed dyslipidemia
◦Olpasiran, which was originally developed by Arrowhead using its proprietary Targeted RNAi Molecule (TRiM) platform and licensed to Amgen in 2016 and is designed to lower levels of lipoprotein(a) (Lp(a)), a genetically-determined independent risk factor for cardiovascular disease, reduced Lp(a) levels by more than 95% in patients with established atherosclerotic cardiovascular disease. These data were simultaneously published in the New England Journal of Medicine (NEJM)
•Strengthened the balance sheet with the sale of Arrowhead’s royalty interest in olpasiran to Royalty Pharma for:
◦$250 million in cash upfront
◦Up to $160 million in additional payments contingent on the achievement of certain clinical, regulatory, and sales milestones
◦Retained rights to $400 million in development, regulatory, and sales milestone payments potentially due from Amgen from the 2016 out-licensing agreement

Selected Fiscal 2022 Financial Results
ARROWHEAD PHARMACEUTICALS, INC.
CONSOLIDATED CONDENSED FINANCIAL INFORMATION
(in thousands, except per share amounts)


Year Ended December 31,
OPERATING SUMMARY 2022 2021
(unaudited)
Revenue $ 62,546 $ 27,439
Operating Expenses:
Research and development 83,695 65,765
General and administrative expenses 20,985 24,995
Total Operating Expenses 104,680 90,760
Operating loss (42,134) (63,321)
Other income, net 340 449
Loss before income tax expense and noncontrolling interest (41,794) (62,872)
Income tax expense 17 —
Net loss including noncontrolling interest (41,811) (62,872)
Net loss attributable to noncontrolling interest, net of tax (486) —
Net loss attributable to Arrowhead Pharmaceuticals, Inc. $ (41,325) $ (62,872)
Net loss per share attributable to Arrowhead Pharmaceuticals, Inc. – Diluted $ (0.39) $ (0.60)
Weighted-average shares used in calculating – Diluted 106,039 104,534

FINANCIAL POSITION SUMMARY December 31,
2022 September 30,
2022
(unaudited)
Cash, cash equivalents and restricted cash $ 202,249 $ 108,005
Short-term investments 299,582 268,391
Long-term investments 115,774 105,872
Total cash resources (cash and investments) 617,605 482,268
Other assets 273,884 209,671
Total Assets $ 891,489 $ 691,939
Current deferred revenue $ 66,281 $ 74,099
Long-term deferred revenue 40,789 55,950
Other liabilities 388,047 143,551
Total Liabilities $ 495,117 $ 273,600
Total Arrowhead Pharmaceuticals, Inc. Stockholders’ Equity $ 377,039 $ 398,520
Noncontrolling Interest 19,333 19,819
Total Noncontrolling Interest and Stockholders’ Equity 377,039 418,339
Total Liabilities, Noncontrolling Interest and Stockholders’ Equity $ 872,156 $ 691,939
Shares Outstanding 106,140 105,960

Dr. Tony Reid from EpicentRx Presents at 3rd TGF-B for Immuno-Oncology Drug Development Summit and Round Table

On February 6, 2023 EpicentRx, reported that 2023 has already been transforming for the company—as in transforming growth factor beta (TGF-ß), which is overexpressed in most tumors. TGF-ß is a cytokine, a small protein that cells use to communicate with each other, normally released in wound areas following tissue damage. In wounds, TGF-ß instructs tolerance and suppresses inflammation, which tumors, crafty and treacherous as they are, co-opt to turn down the anticancer immune response. Because of this adverse effect on the anticancer immune response, EpicentRx has engineered an oncolytic adenovirus, AdAPT-001, which carries and expresses a TGF-ß trap that binds to and neutralizes TGF-ß in the tumor microenvironment, which surrounds and supports the tumor. An adenovirus is an agent of the common cold, which usually causes mild symptoms like cough, sore throat and, occasionally, pink eye.

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For AdAPT-001, the adenovirus has been modified to only infect and replicate in cancer cells, not normal ones, so patients tend not to experience substantial side effects. AdAPT-001 is currently in a Phase 1/2 clinical trial called BETA PRIME (NCT04673942), and preliminary data indicates activity particularly in TGF-ß-driven tumors like sarcoma. In BETA PRIME, AdAPT-001 has been given as a single agent and in combination with checkpoint inhibitors (CIs), since high levels of TGF-ß predict resistance to CIs. In preclinical studies, the combination of AdAPT-001 with CIs makes CI-resistant tumors sensitive to them, as the graph below demonstrates.

Armed with this data, Dr. Tony R. Reid, CEO of EpicentRx, presented at the 3rd TGF-β for Immuno-Oncology Drug Development Summit in San Francisco and held forth at a TGF-β round table, where he discussed the potential advantages of AdAPT-001 over the several TGF-β inhibitor antibodies that are in the clinic. The TGF-β for Immuno-Oncology Drug Development Summit is a conference which aims to bring together all the major players in the TGF-β field. In attendance were Pliant, Scholar Rock, Corbus Pharmaceuticals, Genentech, Pfizer, Oncotelic, and Sanofi Genzyme.

Dr. Reid explained that unlike AdAPT-001, which directly kills cancer cells, TGF-β inhibitor antibodies only indirectly eliminate them. This is important because as cancer cells die, they release proteins, which is often sufficient to activate an immune response. Dr. Reid also believes that because of many barriers in the tumor, which impede drug delivery, the large size of the TGF-β inhibitor antibodies prevents adequate tumor penetration and distribution. Dr. Reid also discussed the favorable safety profile of AdAPT-001, which so far in 30 patients dosed, both alone and in combination, has been very well tolerated with no dose limiting toxicities or related serious adverse events.

Overall, the conference was very well organized and attended, with a robust, interactive crowd. Dr. Reid’s presentations drew interest and attention from academics, clinicians, and scientists, since AdAPT-001 is the only oncolytic virus ever in development to be armed with a TGF-β trap. So, in summary, 2023 is off to a transforming start—for EpicentRx certainly and, hopefully, also for cancer patients that receive treatment with AdAPT-001.

Emergent BioSolutions to Release Fourth Quarter 2022 Financial Results and Conduct Conference Call on February 27, 2023

On February 6, 2023 Emergent BioSolutions Inc. (NYSE: EBS) reported that it will host a conference call on Monday, February 27, 2023, at 5:00 pm eastern time to discuss the financial results for the fourth quarter of 2022, recent business developments, and financial outlook for full year 2023 (Press release, Emergent BioSolutions, FEB 6, 2023, View Source [SID1234626868]).

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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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Conference Call Information
Participants can access the conference call live via webcast from the Investors page of Emergent’s website. To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call.

A replay of the call can be accessed from the Investors page of Emergent’s website.

Domain Therapeutics to Participate in Upcoming Investor and Industry Conferences

On February 6, 2023 Domain Therapeutics ("Domain" or "the Company"), a drug discovery and development company focused on G Protein-Coupled Receptors (GPCRs) in immuno-oncology (IO), reported that investor and industry conferences that its Chief Executive Officer, Pascal Neuville, will attend or speak at in the first half of 2023 (Press release, Domain Therapeutics, FEB 6, 2023, View Source [SID1234626867]).

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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

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OBIO Investor Summit, World Trade Centre, Toronto, Canada – 8-9 February

Presentation slot: February 9th, 3:30 p.m., Room C/D
LSX World Congress, London, UK – 3-4 May

Bio€quity, Dublin, Ireland – 14-16 May

Presentation slot: to be announced at a later date

CymaBay Therapeutics to Present at the SVB Securities Global Biopharma Conference

On February 6, 2023 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that management will participate in the SVB Securities Global Biopharma Conference (Press release, CymaBay Therapeutics, FEB 6, 2023, View Source [SID1234626866]).

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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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SVB Securities Global Biopharma Conference
Date: Wednesday, February 15
Time: 3:40 pm Eastern Time
Location: Virtual
Format: Fireside Chat
Webcast: View Source