On February 6, 2024 Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, reported financial results for the fourth quarter and year ended December 31, 2023 and provided a corporate update (Press release, Deciphera Pharmaceuticals, FEB 6, 2024, View Source [SID1234639868]).

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"We are proud of the significant progress we made across our company throughout 2023, particularly in our late-stage programs as we continue on our path to becoming a self-sustaining, fully integrated biotechnology company. I am excited to announce another record quarter of QINLOCK revenue, demonstrating the proven commercial capabilities that position us well as we continue to evolve into a company with multiple approved medicines," said Steve Hoerter, President and Chief Executive Officer of Deciphera Pharmaceuticals. "Looking ahead, we plan to build upon this momentum in 2024 as we work to file regulatory submissions for vimseltinib, which has the potential to be a much-needed treatment option for patients with tenosynovial giant cell tumor, continue enrollment of our INSIGHT Phase 3 study of QINLOCK, and progress our early-stage pipeline of potential first- or best-in-class candidates."

Fourth Quarter 2023 and Upcoming Milestones

QINLOCK (ripretinib)

Recorded $46.7 million in QINLOCK net product revenue in the fourth quarter of 2023, including $35.3 million in U.S. net product revenue and $11.4 million in international net product revenue, an increase of 42% from net product revenue of $32.9 million in the fourth quarter of 2022.

Published results in Nature Medicine from an exploratory circulating tumor DNA (ctDNA) analysis of the INTRIGUE Phase 3 study demonstrating the substantial clinical benefit of QINLOCK in second line gastrointestinal stromal tumor (GIST) patients with mutations in KIT exon 11 and 17/18.

Presented final OS results from the INTRIGUE Phase 3 clinical study in second-line GIST patients at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers (ASCO GI) Symposium showing that the median OS was similar with QINLOCK (35.5 months) versus sunitinib (31.5 months) (HR 0.86; 95% CI, 0.65 to 1.13; nominal p= 0.275). Treatment with QINLOCK continued to show a favorable safety profile compared to treatment with sunitinib, with fewer patients experiencing Grade 3/4 drug-related treatment emergent adverse events with QINLOCK (27.4%) compared with sunitinib (57.9%). The results also showed that patient outcomes in the third line setting were comparable for patients that were treated with either QINLOCK or sunitinib in the second line. The presentation is available on the Company’s website at www.deciphera.com/presentations-publications.
Entered into a supply and distribution agreement with GENESIS Pharma, a leading regional biopharma company, in Central and Eastern Europe under which GENESIS Pharma will be the exclusive distributor of QINLOCK in 14 countries in the European Union with a combined population of 118 million including Czech Republic, Greece, Hungary, Romania, and Poland.

Continue to enroll the INSIGHT Phase 3 study comparing QINLOCK versus sunitinib in second-line GIST patients with mutations in KIT exon 11 and 17/18.
Vimseltinib

Expects to submit a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) in the second quarter of 2024 and a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA) in the third quarter of 2024.
Expects to present additional results from Part 1 of the MOTION pivotal Phase 3 study of vimseltinib at a medical meeting in the second quarter of 2024.
Expects to present updated results from the Phase 1/2 study of vimseltinib in TGCT in the second half of 2024.
Expects to initiate a Phase 2 proof-of-concept study of vimseltinib for the treatment of chronic graft versus host disease (cGVHD) in the fourth quarter of 2024.
DCC-3116

Expects to select a recommended Phase 2 dose for expansion cohort(s) for DCC-3116, an investigational switch-control kinase inhibitor of ULK1/2 designed to inhibit autophagy, in 2024.
DCC-3084

Expects to initiate a Phase 1 study for DCC-3084, a potential best-in-class pan-RAF inhibitor, in the first half of 2024.
DCC-3009

Expects to submit an Investigational New Drug (IND) application with the FDA for DCC-3009, a potential best-in-class pan-KIT inhibitor, in the first half of 2024 and initiate a Phase 1 study in the second half of 2024.
Fourth Quarter and Full Year 2023 Financial Results

Revenue: Total revenue for the fourth quarter of 2023 was $48.3 million, which includes $46.7 million of net product revenue of QINLOCK and $1.6 million of collaboration revenue compared to $36.3 million of total revenue, including $32.9 million of net product revenue of QINLOCK and $3.4 million of collaboration revenue, for the same period in 2022. Total revenue for the year ended December 31, 2023 was $163.4 million, which includes $159.1 million of net product revenue of QINLOCK and $4.3 million of collaboration revenue compared to $134.0 million of total revenue, including $125.5 million of net product revenue of QINLOCK and $8.5 million of collaboration revenue, for the same period in 2022.

Cost of Sales: Cost of sales were $1.8 million in the fourth quarter of 2023, which includes $0.9 million in cost of product sales, compared to cost of product sales of $0.7 million for the fourth quarter of 2022. For the year ended December 31, 2023, cost of sales were $3.7 million, including $2.0 million in cost of product sales, compared to cost of sales of $8.7 million in 2022, including cost of product sales of $2.7 million. In the third quarter of 2022, the Company completed the sales of zero cost inventories of QINLOCK that had been expensed prior to FDA approval.
R&D Expenses: Research and development expenses for the fourth quarter of 2023 were $58.6 million, compared to $48.1 million for the same period in 2022, and $234.1 million for the year ended December 31, 2023 compared to $187.8 million for the same period in 2022. The increase was primarily due to higher clinical study costs related to QINLOCK, an increase in clinical study costs related to the Phase 1/2 study of DCC-3116, and the Phase 3 study of vimseltinib. Non-cash, stock-based compensation was $21.8 million and $22.2 million for the year ended December 31, 2023 and 2022, respectively.

SG&A Expenses: Selling, general, and administrative expenses for the fourth quarter of 2023 were $39.1 million, compared to $32.2 million for the same period in 2022 and $136.5 million for the year ended December 31, 2023, compared to $120.2 million for the same period in 2022. The increase was primarily due to an increase in professional and consultant fees and personnel-related costs. Non-cash, stock-based compensation was $28.8 million and $29.7 million for the year ended December 31, 2023 and 2022, respectively.
Net Loss: For the fourth quarter of 2023, Deciphera reported a net loss of $47.2 million, or $0.54 per share, compared with a net loss of $45.9 million, or $0.60 per share, for the same period in 2022. Net loss for the year ended December 31, 2023 was $194.9 million, or $2.29 per share, compared with a net loss of $178.9 million, or $2.37 per share, for the year ended December 31, 2022.
Cash Position: As of December 31, 2023, cash, cash equivalents, and marketable securities were $352.9 million, compared to $339.0 million as of December 31, 2022. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product, royalty, and supply revenues, but excluding any potential future milestone payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into the second half of 2026.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, February 6, 2024, at 8:00 AM ET. The conference call may be accessed via this link: https://register.vevent.com/register/BI7a0bbbeb53864df9a854d959bbbae709. A live webcast of the conference call will be available in the "Events and Presentations" page in the "Investors & News" section of the Company’s website at View Source A replay will be available on the Company’s website approximately two hours after the conference call and will be available for 30 days following the call.

On February 6, 2024 BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported that on February 05, 2024, the compensation committee of BridgeBio’s board of directors granted thirty-five new employees restricted stock units for an aggregate of 123,838 shares of the Company’s common stock (Press release, BridgeBio, FEB 6, 2024, View Source [SID1234639867]). One-fourth of the shares underlying each employee’s restricted stock units will vest on February 16, 2025, with one-twelfth of the remaining shares underlying each such employee’s restricted stock units vesting on a quarterly basis thereafter, in each case, subject to each such employee’s continued employment with the Company or one of its subsidiaries on such vesting dates. All of the above-described awards were made under BridgeBio’s Amended and Restated 2019 Inducement Equity Plan (the "Plan").

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The above-described awards were each granted as an inducement material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4) and were granted pursuant to the terms of the Plan. The Plan was adopted by BridgeBio’s board of directors in November 2019, and amended and restated on February 10, 2023 and on December 13, 2023.

On February 6, 2024 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company dedicated to developing first-in-class and best-in-class targeted cancer therapies, reported that it entered into a definitive asset purchase agreement with Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, to acquire AL102 and related drug candidate AL101 from Ayala (Press release, Ayala Pharmaceuticals, FEB 6, 2024, View Source [SID1234639866]). Based on the terms of the agreement, Immunome will pay Ayala $20 million in cash and $30 million in Immunome common stock (valued at 30-day VWAP as of February 1, 2024) at the closing and will pay up to an additional $37.5 million in development and commercial milestone payments. Completion of the transaction is subject to customary conditions including Ayala obtaining the requisite stockholder approval.

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AL102 is an investigational small molecule gamma secretase inhibitor currently being evaluated in the randomized Phase 3 RINGSIDE international trial for the treatment of desmoid tumors – a debilitating soft tissue malignancy. AL102 is a potential once-daily oral treatment for desmoid tumors. Data from clinical trials have shown AL102 may be more effective in treating desmoid tumors than OGSIVEO (nirogacestat), which recently became the first treatment approved for desmoid tumors by the U.S. Food and Drug Administration (FDA) in November 2023.

The Phase 2 portion of the RINGSIDE study demonstrated clinically meaningful anti-tumor activity across multiple parameters. The data showed high objective response rates, including 75% of evaluable patients (and 64% of intent-to-treat patients) in the 1.2 mg once daily arm, the dose being evaluated in the current Phase 3 trial. Furthermore, patients receiving the 1.2 mg daily dose experienced a median best reduction in tumor volume of 88% as measured by serial MRI exams, as of the previously reported July 5, 2023 data cut-off, and also demonstrated improvement in other radiographic parameters.

"Immunome is committed to advancing therapies with best-in-class potential. We are especially optimistic about the rapidity and depth of the tumor responses observed in the Phase 2 portion of RINGSIDE," said Clay B. Siegall, Ph.D., President and Chief Executive Officer of Immunome. "AL102 will complement our existing portfolio of targeted cancer agents that are approaching Phase 1 trials. As we complete the work required to advance AL102 to NDA submission, our goal is to bring clinical benefit to an underserved patient population while generating substantial value for stockholders. We also plan to investigate other populations of cancer patients that could benefit from treatment with AL102."

Ken Berlin, CEO of Ayala Pharmaceuticals, stated, "We are pleased that this exciting molecule will be advanced by Immunome and its team of highly-experienced drug developers which, we believe, maximizes the ability to conclude the RINGSIDE study and bring our lead asset to commercialization. I am proud of the work Ayala has done over the last several years to assemble compelling clinical data and reach this point."

It is estimated that as many as 1,650 people in the U.S. are diagnosed annually with desmoid tumors, also known as aggressive fibromatosis, and the population prevalence is much higher. Desmoid tumors, which occur predominantly in young adults, can cause significant pain and disability. Available non-pharmacologic therapies such as surgery or radiation are often ineffective and can lead to long term complications, and unapproved targeted therapies lack long-term potency. In November 2023, AL102 was granted Orphan Drug Designation by the FDA.

A.G.P./Alliance Global Partners is acting as strategic advisor to Ayala Pharmaceuticals, Inc. in connection with the transaction.

On February 6, 2024 Arvinas, Inc. (Nasdaq: ARVN) and Pfizer Inc. (NYSE: PFE) reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the investigation of vepdegestrant (ARV-471) for monotherapy in the treatment of adults with estrogen receptor (ER) positive/human growth epidermal growth factor 2 (HER2) negative (ER+/HER2-) locally advanced or metastatic breast cancer previously treated with endocrine-based therapy (Press release, Arvinas, FEB 6, 2024, View Source [SID1234639865]). Vepdegestrant is a novel oral PROteolysis Targeting Chimera (PROTAC) ER degrader that is being jointly developed by Arvinas and Pfizer.

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As described by the FDA, Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to patients earlier. Vepdegestrant as a monotherapy is being studied in the ongoing Phase 3 VERITAC-2 clinical trial, which is evaluating vepdegestrant or fulvestrant in patients with locally advanced or metastatic ER+/HER2- breast cancer who have been previously treated with an endocrine-based therapy.

"We are focused on the persisting unmet needs of people with ER+/HER2- breast cancer and doing all that we can to expedite the development of vepdegestrant as a novel, oral ER-targeted potential therapy for this patient community," said John Houston, Ph.D., Arvinas Chairperson, Chief Executive Officer, and President. "We are pleased the FDA has granted Fast Track designation for vepdegestrant, and we continue to believe this investigational drug has the potential to harness the body’s own natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins."

"The receipt of Fast Track designation reinforces the potential of vepdegestrant to provide an important new therapeutic option for people with ER+/HER2- breast cancer whose disease has progressed," said Roger Dansey, M.D., Chief Development Officer, Oncology, Pfizer. "We are proud to continue our legacy of developing innovative treatment options for people impacted by metastatic breast cancer and look forward to working with the FDA as we advance our development program for vepdegestrant."

About Vepdegestrant (ARV-471) and its Clinical Trials
Vepdegestrant is an investigational, orally bioavailable PROTAC protein degrader designed to specifically target and degrade the estrogen receptor (ER) for the treatment of patients with ER positive (ER+)/human epidermal growth factor receptor 2 (HER2) negative (ER+/HER2-) breast cancer.

In preclinical studies, vepdegestrant demonstrated up to 97% ER degradation in tumor cells, induced robust tumor shrinkage when dosed as a single agent in multiple ER-driven xenograft models, and showed increased anti-tumor activity when compared to a standard of care agent, fulvestrant, both as a single agent and in combination with a CDK4/6 inhibitor. In July 2021, Arvinas announced a global collaboration with Pfizer for the co-development and co-commercialization of vepdegestrant; Arvinas and Pfizer will equally share worldwide development costs, commercialization expenses, and profits. Ongoing and planned clinical trials will continue to monitor and evaluate the safety and anti-tumor activity of vepdegestrant.

Vepdegestrant is currently being evaluated as a monotherapy in the second-line setting in the ongoing Phase 3 VERITAC-2 clinical trial and in the first-line setting in combination with palbociclib in the ongoing study lead-in cohort of the Phase 3 VERITAC-3 clinical trial. Vepdegestrant is also being evaluated for potential combination therapy with abemaciclib, ribociclib, samuraciclib, everolimus, and with Pfizer’s investigational novel CDK4 inhibitor, PF-07220060.

On February 6, 2024 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2024 first quarter ended December 31, 2023 (Press release, Arrowhead Research Corporation, FEB 6, 2024, View Source [SID1234639864]). The company is hosting a conference call today, February 6, 2024, at 4:30 p.m. ET to discuss the results.

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Webcast and Conference Call and 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/BId9fba8b41eb34305a8f07de301a9d42c. 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
•Continued to build the cardiometabolic vertical to include two new additional complementary medicines designed to treat obesity and metabolic disease, both of which are on schedule for clinical trial application (CTA) filings before the end of 2024
◦ARO-INHBE utilizes the liver targeted TRiMTM platform and targets the INHBE gene. INHBE expression is increased in obesity and INHBE loss-of-function variants identified in human genetic databases are protective of type 2 diabetes and are associated with reduced visceral fat and a reduced waste to hip ratio
◦The second is based on Arrowhead’s adipose tissue targeting TRiMTM platform and addresses an undisclosed gene target
•Performed a portfolio review and R&D organization restructuring resulting in key changes, including:
◦Arrowhead will focus investment in late-stage clinical development and build out commercial infrastructure to support sales and marketing of medicines primarily in the cardiometabolic and pulmonary verticals
◦Engaged Bruce Given, M.D., Arrowhead’s former chief operating officer and head of R&D for Arrowhead for several years before his retirement in 2020 after a more than 30-year career in biotechnology and pharmaceutical research, development, and sales and marketing, on an interim basis to serve as chief medical scientist to help with restructuring efforts and the process to recruit new therapeutic area experts to decentralize the chief medical officer function
◦Identified opportunities to reduce forward operating cash burn guidance by up to $100 million, approximately $30 million per quarter, while retaining full funding and investment in core cardiometabolic and pulmonary programs
◦Focused R&D spending for non-core programs on development activities that advance candidates to an attractive stage for potential partnerships

◦Terminated development of ARO-SOD1 and HZN-457
•Strengthened the balance sheet through an underwritten registered offering of common stock gross proceeds of approximately $450 million, before deducting underwriting discounts, commissions, and other offering expenses payable by the company
•Presented data on cardiometabolic pipeline at the American Heart Association Scientific Sessions in November 2023
◦Presented new Phase 2 clinical data from the ongoing SHASTA-2 and MUIR studies of plozasiran (ARO-APOC3) and the ARCHES-2 study of zodasiran (ARO-ANG3)
◦Hosted a webinar to discuss plozasiran featuring experts in the treatment and management of lipid and lipoprotein disorders
•Completed chronic GLP toxicology for pulmonary candidates ARO-RAGE, designed to reduce expression of the receptor for advanced glycation end products as a potential treatment for inflammatory pulmonary diseases, and ARO-MMP7, designed to reduce expression of matrix metalloproteinase 7 as a potential treatment for idiopathic pulmonary fibrosis
◦Results were highly encouraging and suggest sufficient safety margins to proceed to Phase 2 studies
•Filed for regulatory clearance to initiate a Phase 1/2a study of ARO-CFB, Arrowhead’s RNAi therapeutic candidate for patients with complement renal disease such as immunoglobulin A nephropathy (IgAN), which is the world’s most common glomerular disease
•Filed for regulatory clearance to initiate a Phase 1/2a study of Arrowhead’s second RNAi therapeutic candidate targeting skeletal muscle, ARO-DM1, for patients with type 1 myotonic dystrophy

Selected Fiscal 2024 Financial Results
ARROWHEAD PHARMACEUTICALS, INC.
CONSOLIDATED CONDENSED FINANCIAL INFORMATION
(in thousands, except per share amounts)

Year Ended
December 31,
OPERATING SUMMARY 2023 2022
(Unaudited)
Revenue $ 3,551 $ 62,546
Operating Expenses:
Research and development 116,491 83,695
General and administrative expenses 23,605 20,985
Total Operating Expenses 140,096 104,680
Operating loss (136,545) (42,134)
Total other (expense) income (2,144) 340
Loss before income tax (benefit) expense and noncontrolling interest (138,689) (41,794)
Income tax (benefit) expense (3,313) 17
Net loss including noncontrolling interest (135,376) (41,811)
Net loss attributable to noncontrolling interest, net of tax (2,512) (486)
Net loss attributable to Arrowhead Pharmaceuticals, Inc. $ (132,864) $ (41,325)

Net loss per share attributable to Arrowhead Pharmaceuticals, Inc. – Diluted $ (1.24) $ (0.39)
Weighted-average shares used in calculating – Diluted 107,415 106,039

FINANCIAL POSITION SUMMARY December 31,
2023 September 30,
2023
(unaudited)
Cash, cash equivalents and restricted cash $ 58,215 $ 110,891
Investments 162,064 292,735
Total cash resources (cash and investments) 220,279 403,626
Other assets 406,007 361,926
Total Assets $ 626,286 $ 765,552
Current deferred revenue $ — $ 866
Other liabilities 452,572 477,524
Total Liabilities $ 452,572 $ 478,390
Total Arrowhead Pharmaceuticals, Inc. Stockholders’ Equity $ 160,407 $ 271,343
Noncontrolling Interest 13,307 15,819
Total Noncontrolling Interest and Stockholders’ Equity $ 173,714 $ 287,162
Total Liabilities, Noncontrolling Interest and Stockholders’ Equity $ 626,286 $ 765,552
Shares Outstanding 107,500 107,312