Quince Therapeutics to Present at Oppenheimer 34th Annual Healthcare Life Sciences Conference

On February 7, 2024 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology that leverages a patient’s own biology to deliver rare disease therapeutics, reported that Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, will present at the Oppenheimer 34th Annual Healthcare Life Sciences Conference, a virtual event, on Wednesday, February 14, 2024 beginning at 10:40 a.m. Eastern Time (Press release, Quince Therapeutics, FEB 7, 2024, View Source [SID1234639915]).

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A live webcast of the presentation will be accessible on the Events page under the News & Events heading of Quince’s Investor Relations website at ir.quincetx.com. An archive of the webcast will be available shortly following the end of the live event.

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]).

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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]).

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"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]).

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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]).

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"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.