Prokarium Achieves Milestone with First Patient Dosing in PARADIGM-1 Non-Muscle Invasive Bladder Cancer Clinical Trial

On February 7, 2024 Prokarium, a clinical-stage biopharmaceutical company bioengineering Living Cures through cutting-edge synthetic biology, reported the dosing of the first patient in their PARADIGM-1 Phase I/Ib clinical trial in non-muscle invasive bladder cancer (NMIBC) patients (NCT06181266) (Press release, Prokarium, FEB 7, 2024, View Source [SID1234639914]).

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!

The study is being conducted at multiple clinical sites across the United States and will initially evaluate the safety of a single intravesical administration of their investigational immunotherapy, ZH9, in NMIBC patients. The study will further investigate and characterize clinical efficacy of the ZH9 treatment regimen in pre-planned expansion cohorts under the same IND.

"Dosing our first patient in the clinical trial is an exciting milestone," said Dr Josefin-Beate (Josi) Holz, CMO of Prokarium. "It translates our scientific promises into practical advancements, as we strive to transform the treatment paradigm for patients with NMIBC."

Dr Daniel Zainfeld, Urology San Antonio commented, "We are excited to have dosed our first patient in Prokarium’s PARADIGM-1 study. The trial provides us the opportunity to explore an innovative treatment approach for our NMIBC patients who are facing significant gaps in available effective treatment options."

Kristen Albright, CEO of Prokarium, remarked, "We are grateful to the patients and urologists involved in the PARADIGM-1 clinical trial. This achievement represents a pivotal moment for Prokarium as we introduce Living Cures, a novel class of immunotherapies built with synthetic biology."

Neurocrine Biosciences Reports Fourth Quarter and Fiscal 2023 Financial Results and Provides Financial Expectations for 2024

On February 7, 2024 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the fourth quarter and full year ended December 31, 2023 and provided financial guidance for 2024 (Press release, Neurocrine Biosciences, FEB 7, 2024, View Source [SID1234639913]).

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!

"I’m exceptionally proud of the progress we made with INGREZZA last year, helping more patients than ever before treat their tardive dyskinesia. In addition, the positive Phase 3 crinecerfont results for the treatment of congenital adrenal hyperplasia opens the door for us to help patients living with a disabling neuroendocrine disorder," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With a strong foundation in INGREZZA, a potential second important growth driver in crinecerfont, and an advancing pipeline, Neurocrine Biosciences is making significant progress towards becoming a leading neuroscience focused company."

Financial Highlights

Three Months Ended

December 31,

Twelve Months Ended

December 31,

(unaudited, in millions, except per share data)

2023

2022

2023

2022

Revenues:

Net Product Sales

$ 507.2

$ 404.6

$ 1,860.6

$ 1,440.9

Collaboration Revenue

8.0

7.4

26.5

47.8

Total Revenues

$ 515.2

$ 412.0

$ 1,887.1

$ 1,488.7

GAAP Research and Development (R&D)

$ 137.5

$ 118.0

$ 565.0

$ 463.8

Non-GAAP R&D

$ 124.3

$ 103.9

$ 497.0

$ 406.1

GAAP Selling, General and Administrative (SG&A)

$ 218.9

$ 182.9

$ 887.6

$ 752.7

Non-GAAP SG&A

$ 194.0

$ 151.8

$ 757.4

$ 635.6

GAAP Net Income

$ 147.7

$ 89.0

$ 249.7

$ 154.5

GAAP Earnings Per Share – Diluted

$ 1.44

$ 0.88

$ 2.47

$ 1.56

Non-GAAP Net Income

$ 157.7

$ 124.7

$ 390.0

$ 343.2

Non-GAAP Earnings Per Share – Diluted

$ 1.54

$ 1.24

$ 3.86

$ 3.47

(unaudited, in millions)

December 31,

2023

December 31,

2022

Total Cash, Cash Equivalents and Marketable Securities

$ 1,719.1

$ 1,288.7

INGREZZA Net Product Sales Highlights

INGREZZA fourth quarter and fiscal 2023 net product sales were $500 million and $1.84 billion, respectively
INGREZZA fourth quarter net product sales grew 25% compared with fourth quarter 2022, driven by strong underlying patient demand offset slightly by seasonal gross-to-net dynamics
Other Key Financial Highlights

Differences in fourth quarter 2023 GAAP and non-GAAP operating expenses compared with fourth quarter 2022 were driven by:
Increased R&D expense in support of an expanded and advancing clinical portfolio including preclinical investments in muscarinic compounds, gene therapy programs and second generation VMAT2 inhibitors
Increased SG&A expense primarily due to ongoing commercial initiatives supporting INGREZZA growth including the expanded indication to treat chorea associated with Huntington’s disease
Fourth quarter 2023 GAAP net income and earnings per share were $148 million and $1.44, respectively, compared with $89 million and $0.88, respectively, for fourth quarter 2022
Fourth quarter 2023 non-GAAP net income and earnings per share were $158 million and $1.54, respectively, compared with $125 million and $1.24, respectively, for fourth quarter 2022
At December 31, 2023, the Company had cash, cash equivalents and marketable securities totaling approximately $1.7 billion
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this news release.

Recent Developments

In November 2023, the Company announced that all patent litigation brought by Neurocrine Biosciences against the companies that filed Abbreviated New Drug Applications (ANDA) to the FDA seeking approval to market generic versions of INGREZZA prior to the expiration of the Orange Book listed patents have been resolved. Accordingly, such companies have the right to sell generic versions of INGREZZA in the U.S. beginning March 2038, or earlier under certain circumstances.
In December 2023, the Company announced crinecerfont received Breakthrough Therapy designation from the FDA for the treatment of Congenital Adrenal Hyperplasia in adults and pediatrics.
Full Year 2024 Financial Guidance

Range

(in millions)

Low

High

INGREZZA Net Product Sales 1

$ 2,100

$ 2,200

GAAP R&D Expense 2

$ 645

$ 675

Non-GAAP R&D Expense 3

$ 570

$ 600

GAAP SG&A Expense 4

$ 930

$ 950

Non-GAAP SG&A Expense 3, 4

$ 830

$ 850

1.

INGREZZA sales guidance reflects expected net product sales of INGREZZA in tardive dyskinesia and chorea associated with Huntington’s disease.

2.

GAAP R&D guidance includes expense for development milestones once determined achievement is deemed probable. Acquired in-process research and development expense is included in guidance once significant collaboration and licensing arrangements have been completed.

3.

Non-GAAP guidance adjusted primarily to exclude estimated non-cash stock-based compensation expense of $75 million in R&D and $100 million in SG&A.

4.

SG&A guidance range reflects expense for ongoing commercial initiatives supporting INGREZZA growth including the expanded indication to treat chorea associated with Huntington’s disease and pre-launch commercial activities for crinecerfont.

2024 Expected Pipeline Milestones and Key Activities

Program

Indication

Expected Milestones / Key Activities

Valbenazine*

(Selective VMAT2 Inhibitor)

Sprinkle Formulation for Tardive Dyskinesia
/ Chorea in Huntington’s Disease

PDUFA April 30, 2024

Crinecerfont

(CRF1 Receptor Antagonist)

Congenital Adrenal Hyperplasia

(Pediatric and Adult)

Submitting New Drug Application to the
FDA in Q2’24

Efmody

(Hydrocortisone Modified
Release Hard Capsules)

Adrenal Insufficiency

Top-Line Phase 2 Data in 1H’24

Efmody

(Hydrocortisone Modified
Release Hard Capsules)

Congenital Adrenal Hyperplasia

Top-Line Phase 2 Data in 1H’24

NBI-1065845**

(AMPA Potentiator)

Inadequate Response in Major Depressive
Disorder

Top-Line Phase 2 Data in 1H’24

NBI-1117568†

(M4 Agonist)

Schizophrenia

Top-Line Phase 2 Data in 2H’24

Luvadaxistat**

(DAAO Inhibitor)

Cognitive Impairment Associated with
Schizophrenia

Top-Line Phase 2 Data in 2H’24

NBI-1070770**

(NMDA NR2B NAM)

Major Depressive Disorder

Initiating Phase 2 Study

NBI-1117567†

(M1 Agonist)

CNS Indications

Initiating Phase 1 Study

NBI-1076986

(M4 Antagonist)

Movement Disorders

Initiating Phase 1 Study

NBI-1065890

(Selective VMAT2 Inhibitor)

CNS Indications

Initiating Phase 1 Study

Key: VMAT2 = Vesicular Monoamine Transporter 2; CFR1 = Corticotropin-Releasing Factor Type 1; AMPA = alpha-amino-3-hydroxy-5-methyl-4-isoxazole propionic acid; M4 = M4 Muscarinic Receptor; DAAO = d-amino acid oxidase; NMDA NR2B NAM = n-methyl-d-aspartate Receptor Subtype 2B Negative Allosteric Modulator; M1 = M1 Muscarinic Receptor

Neurocrine Biosciences Partners: * Mitsubishi Tanabe Pharma Corporation has commercialization rights in East Asia; ** Partnered with Takeda Pharmaceutical Company Limited; † In-Licensed from Sosei Group Corporation

Conference Call and Webcast Today at 8:00 AM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time). Participants can access the live conference call by dialing 800-225-9448 (US) or 203-518-9708 (International) using the conference ID: NBIX. The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

INOVIO to Present at the Oppenheimer 34th Annual Healthcare Life Sciences Conference

On February 7, 2024 INOVIO (NASDAQ:INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, reported that Dr. Jacqueline Shea, President and CEO will present at the Oppenheimer 34th Annual Healthcare Life Sciences Conference (Press release, Inovio, FEB 7, 2024, View Source [SID1234639912]).

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!

Oppenheimer 34th Annual Healthcare Life Sciences Conference
Date: Wednesday, February 14, 2024
Time: 12:40 PM ET
Format: Fireside Chat

During the conference, Dr. Shea and members of INOVIO’s management team will conduct one-on-one meetings with registered investors.

A webcast of the presentation will be available on the INOVIO Investor Relations Events page at View Source A replay of the webcast will be available for 90 days after the date of the presentation.

Inhibikase Therapeutics Announces Preliminary Outcomes of its Pre-NDA Meeting with the FDA on the Pathway for Approval for IkT-001Pro in Blood and Gastrointestinal Cancers

On February 7, 2024 Inhibikase Therapeutics, Inc. (Nasdaq: IKT) ("Inhibikase" or "Company"), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson’s disease, Parkinson’s-related disorders and other diseases of the Abelson Tyrosine Kinases, reported preliminary outcomes of the Company’s discussion with the U.S. Food and Drug Administration (FDA) on the path to approval of IkT-001Pro in blood cancers, the Company’s prodrug of the anticancer agent imatinib mesylate (Press release, Inhibikase Therapeutics, FEB 7, 2024, View Source [SID1234639911]).

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 were pleased with the discussion we had with the FDA as we begin the process of building our first NDA package needed for approval," said Dr. Milton Werner, President and Chief Executive Officer of Inhibikase. "Our bioequivalence studies were presented to the FDA and we were given specific guidance on the manufacturing requirements necessary to complete the NDA. The FDA acknowledged that the appropriate approval path appears to be 505(b)(2) and we plan to seek all 11 indications for which imatinib mesylate has been approved, including its use in children. There is significant work ahead of us as we discuss these details with potential commercialization partners and carry out the work needed for the NDA submission," noted Dr. Werner.

On January 19, 2024, members of the Company along with its medical oncology consultants met with the FDA Review Team from the Division of Hematologic Malignancies to discuss the Company’s bioequivalence studies of IkT-001Pro. During the meeting Inhibikase inquired whether additional clinical studies may be needed to seek approval and discussed manufacturing and quality control requirements for approval. The Review Team acknowledged that the 505(b)(2) pathway appears to be the appropriate pathway for approval of IkT-001Pro and indicated that, pending formal review of the Company’s clinical data, clinical studies completed to date indicate that 600 mg and 800 mg IkT-001Pro provides similar exposures to 400 mg and 600 mg imatinib mesylate, respectively. These preliminary outcomes from the meeting are subject to formal review of the NDA package. In addition, given that imatinib mesylate is approved for use between 300 mg and 800 mg once daily for a variety of blood and gastrointestinal cancers, the Review Team advised that if the Company intends to seek approval across all currently approved indications, Inhibikase should evaluate additional doses as needed to measure the safety, tolerability and bioequivalent dose of IkT-001Pro that would deliver up to 800 mg, the highest approved dose of imatinib mesylate. The Review Team also discussed the possible difference between IkT-001Pro and imatinib mesylate absorption in the gut and recommended the Company evaluate whether IkT-001Pro and imatinib mesylate behave differently with respect to certain gut transporters that regulate absorption. Inhibikase is in alignment with the FDA and is initiating the necessary pre-clinical tests to evaluate this further to ensure that delivery of imatinib by IkT-001Pro mimics imatinib mesylate in all respects. Finally, a number of recommendations were discussed to prevent the mix-up between 001Pro and imatinib mesylate either at the pharmacy or by patients for two drugs delivering the same active ingredient. A comprehensive use-related risk analysis will be conducted as part of the manufacturing and quality control development program to identify ways to discriminate the two drugs by appearance, pill size and dosage. The Company will request milestone-based meetings as it completes the manufacturing and quality control processes to ensure are it is meeting the manufacturing requirements for approval.

The Company has continued to make progress in its evaluation of risvodetinib in the 201 Trial in Untreated Parkinson’s disease. As of February 7, 2024, 32 sites are open and actively evaluating prospective trial participants. 51 participants have been enrolled, 19 prospective participants are in medical screening and 46 potential participants are being evaluated for suitability to initiate medical screening. Twenty-three participants have completed the 12 week dosing period. Nine mild and one moderate treatment-related adverse events have been reported across all enrolled participants taking risvodetinib.

About IkT-001Pro
IkT-001Pro is a prodrug formulation of imatinib mesylate and has been developed to improve the safety of the first FDA-approved Abelson (Abl) kinase inhibitor, imatinib (marketed as Gleevec). Imatinib is commonly taken for hematological and gastrointestinal cancers that arise from Abl kinase mutations found in the bone marrow or for gastrointestinal cancers that arise from c-Kit and/or PDGFRa/b mutations in the stomach; c-Kit, PDGFRa/b and Abl are all members of the Abelson Tyrosine Kinase protein family. IkT-001Pro has the potential to be a safer alternative for patients and may improve the number of patients that reach and sustain major and/or complete cytogenetic responses in Stable-Phase Chronic Myelogenous Leukemia ("Stable-Phase CML") and/or reduce the relapse rate for these patients. In preclinical studies, IkT-001Pro was shown to be as much as 3.4 times safer than imatinib in primates, reducing burdensome gastrointestinal side effects that occur following oral administration. Imatinib delivered as IkT-001Pro was granted Orphan Drug Designation for Stable-Phase CML in September, 2018.

HUTCHMED Highlights Presentation of Phase III Data on Fruquintinib in Second-Line Gastric Cancer at ASCO Plenary Series Session

On February 7, 2024 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:​HCM; HKEX:​13) reported that data from FRUTIGA, HUTCHMED’s Phase III trial of fruquintinib in combination with paclitaxel for the treatment of second-line advanced gastric cancer in China, were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Plenary Series Session on February 6, 2024 (Press release, Hutchison China MediTech, FEB 7, 2024, View Source [SID1234639910]). The full presentation can be found here. Fruquintinib is a selective oral inhibitor of vascular endothelial growth factor receptors ("VEGFR") -1, -2 and -3, which play a pivotal role in blocking tumor angiogenesis.

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!

The ASCO (Free ASCO Whitepaper) Plenary Series was established to feature practice-changing and clinically relevant studies on the latest advances in cancer care.1

The FRUTIGA trial (clinicaltrials.gov identifier NCT03223376) was a 1:1 randomized, double-blind, Phase III study conducted at 35 sites in China to evaluate fruquintinib combined with chemotherapy, paclitaxel, compared with paclitaxel monotherapy for second-line treatment of 703 patients with advanced gastric or gastro­esophageal junction adenocarcinoma. The dual primary endpoints were progression-free survival ("PFS") and overall survival ("OS"), and the study was declared positive as the PFS endpoint met statistical significance at a pre-defined alpha level.

Median PFS for patients who received fruquintinib plus paclitaxel was 5.6 months, compared to 2.7 months for those who received paclitaxel monotherapy, a statistically significant improvement (stratified hazard ratio ["HR"] = 0.569; p < 0.0001). The objective response rate ("ORR") was significantly higher in the fruquintinib combination group (42.5% vs. 22.4%).

There was an improvement in OS with median OS of 9.6 months vs. 8.4 months, however this was not statistically significant. There was an imbalance of patients receiving subsequent antitumor therapies across the two groups, with 52.7% in the fruquintinib plus paclitaxel group vs. 72.2% in the paclitaxel monotherapy group. Pre-specified sensitivity analyses showed that in patients without these subsequent antitumor therapies, OS improvement was statistically significant. Median OS for patients who received the combination therapy was 6.9 months compared to 4.8 months for those receiving the placebo, with a HR of 0.72 (p = 0.0422).

Fruquintinib demonstrated a statistically significant improvement in multiple other endpoints, including disease control rate ("DCR") at 77.2% vs. 56.3%, and duration of response ("DoR") at 5.5 vs. 3.7 months. The most common (at least 5%) grade 3 or above treatment-emergent adverse events were neutropenia (60.0% vs. 36.4%), leukopenia (42.9% vs. 23.5%), anemia (11.7% vs. 10.6%) and palmar-plantar erythrodysesthesia syndrome (8.9% vs. 4.9%). As such, fruquintinib plus paclitaxel was well-tolerated with a safety profile consistent with expectations.

The presentation concludes that fruquintinib plus paclitaxel could be a promising second-line treatment option for patients with advanced gastric or gastro­esophageal adenocarcinoma who have failed fluoropyrimidine- or platinum-containing chemotherapy.

Presentation title

Presenter & Lead author

Presentation details

Fruquintinib plus paclitaxel versus paclitaxel as second-line therapy for patients with advanced gastric or gastroesophageal junction adenocarcinoma (FRUTIGA): a randomized, multicenter, double-blind, placebo-controlled, phase 3 study

Rui-Hua Xu, MD, PhD

February ASCO (Free ASCO Whitepaper) Plenary Series Session

Abstract 438730

Tuesday, February 6, 2024
3 pm ET (8pm GMT, 4am HKT)

The New Drug Application ("NDA") for fruquintinib in combination with paclitaxel for the treatment of second-line advanced gastric or gastroesophageal junction adenocarcinoma in China was accepted for review by the China National Medical Products Administration in April 2023. Fruquintinib is approved in China and the United States for the treatment of certain patients with metastatic colorectal cancer ("mCRC").

About the ASCO (Free ASCO Whitepaper) Plenary Series

According to ASCO (Free ASCO Whitepaper), the ASCO (Free ASCO Whitepaper) Plenary Series was established to feature practice-changing and clinically relevant studies on the latest advances in cancer care. Up to two abstracts are presented in each session, and accompanied by a discussant presentation and a live question and answer session. It was developed by ASCO (Free ASCO Whitepaper) so that researchers and clinicians can stay current on cutting-edge research in oncology in between meetings, providing faster dissemination of practice-changing science to better help clinicians deliver the most up-to-date care and treatments to patients.

Abstracts at the ASCO (Free ASCO Whitepaper) Plenary Series are expected to address novel scientific questions, detail clinical observations, and contain primary scientific data in the form of a randomized phase II and III trial, or be original research studies that highlight novel and high-impact research with practice-changing implications. Presented studies are also expected to be placed in an oral presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting.

About the Phase III FRUTIGA Trial

FRUTIGA is a randomized, double-blind, Phase III study in China to evaluate fruquintinib combined with paclitaxel compared with paclitaxel monotherapy, for second-line treatment of advanced gastric cancer. The study enrolled 703 patients. Its dual-primary endpoints were PFS and OS. The trial met the PFS endpoint at a statistically and clinically meaningful level. While there was an improvement in median OS, the OS endpoint was not statistically significant per the pre-specified statistical plan. Fruquintinib also demonstrated a statistically significant improvement in secondary endpoints including objective response rate (ORR), DCR and DoR. The safety profile of fruquintinib in FRUTIGA was consistent with previously reported studies. Additional details may be found at clinicaltrials.gov, using identifier NCT03223376.

About Gastric Cancer

Gastric cancer is a cancer that starts in the stomach. It is the fifth most common cancer worldwide in 2020. It was estimated to have caused approximately 770,000 deaths worldwide.2 In China, it was estimated that over 478,000 people were diagnosed with gastric cancer, and approximately 374,000 people died from gastric cancer.3

About Fruquintinib

Fruquintinib is a selective oral inhibitor of VEGFR-1, -2 and -3. VEGFR inhibitors play a pivotal role in inhibiting tumor angiogenesis. Fruquintinib was designed to have enhanced selectivity that limits off-target kinase activity, allowing for high drug exposure, sustained target inhibition, and flexibility for the potential use as part of combination therapy. Fruquintinib has demonstrated a manageable safety profile and is being investigated in combinations with other anti-cancer therapies.

About Fruquintinib Approval in China

Fruquintinib is approved for marketing in China, where it is co-marketed by HUTCHMED and Lilly under the brand name ELUNATE. It was included in the China National Reimbursement Drug List (NRDL) in January 2020. The approval was based on data from the FRESCO study, a Phase III pivotal registration trial of fruquintinib in 416 patients with mCRC in China, which were published in The Journal of the American Medical Association, JAMA. Since its launch in China, fruquintinib has benefited more than 80,000 colorectal cancer patients as of mid-2023.

About Fruquintinib Approval in the United States

Fruquintinib received approval in the United States in November 2023, where it is marketed by Takeda under the brand name FRUZAQLA. The approval was based on data from two large Phase III trials: the multi-regional FRESCO-2 trial, data from which were published in The Lancet, along with the FRESCO trial conducted in China. The trials investigated fruquintinib plus best supportive care versus placebo plus best supportive care in patients with previously treated mCRC. Both FRESCO and FRESCO-2 met their primary and key secondary efficacy endpoints and showed consistent benefit among a total of 734 patients treated with fruquintinib. Safety profiles were consistent across trials.