Sana Biotechnology Announces Proposed Public Offering of Common Stock

On February 7, 2024 Sana Biotechnology, Inc. (Nasdaq: SANA), a company focused on changing the possible for patients through engineered cells, reported that it has commenced an underwritten public offering of $125.0 million of shares of its common stock (Press release, Sana Biotechnology, FEB 7, 2024, View Source [SID1234639917]). In addition, Sana intends to grant the underwriters a 30-day option to purchase up to an additional $18.75 million of shares of its common stock. All of the shares to be sold in this offering will be sold by Sana. The proposed offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the proposed offering.

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!

Morgan Stanley, J.P. Morgan, Goldman Sachs & Co. LLC, and BofA Securities are acting as joint book-running managers for the proposed offering.

The proposed offering is being made pursuant to a Registration Statement on Form S-3, including a base prospectus, previously filed with and declared effective by the SEC, and Sana will file a preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the proposed offering, copies of which can be accessed for free through the SEC’s website at www.sec.gov. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to the proposed offering may also be obtained from: Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, NY 10014 or by email at [email protected]; J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at [email protected]; Goldman Sachs & Co. LLC, Attn: Prospectus Department, at 200 West Street, New York, NY 10282, by telephone at (866) 471-2526 or by email at [email protected]; or BofA Securities, Attn: Prospectus Department, NC1-022-02-25, 201 North Tryon Street, Charlotte, NC 28255-0001 or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor will there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful before registration or qualification under the securities laws of any such state or jurisdiction.

Redx Announces Sale of KRAS Inhibitor Program

On February 7, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Redx Pharma plc (AIM: REDX) reported that the companies have signed a definitive agreement under which Jazz will acquire Redx’s KRAS (Kirsten rat sarcoma virus) inhibitor program (Press release, Redx Pharma, FEB 7, 2024, View Source [SID1234639916]). Jazz and Redx will collaborate to advance candidates through IND-enabling studies; Jazz will be responsible for all clinical development, regulatory, manufacturing and commercialization activities.

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!

"KRAS is a well-validated oncology target and there remains a high unmet need for innovation in this area based on challenges in developing molecules to target specific KRAS mutations. Redx has discovered a number of preclinical KRAS candidates and we plan to leverage our collective oncology development expertise to identify and advance the most promising molecules toward the clinic," said Robert Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "This transaction further expands our early-stage oncology pipeline, and we are excited to explore novel approaches to improving treatment options for cancer patients."

Lisa Anson, Chief Executive Officer of Redx, commented: "Once again, our distinguished expertise in medicinal chemistry has been recognized and we are pleased to secure another strategic transaction with Jazz, with whom we have a track record of collaboration. This agreement will allow us to collaborate on the advancement of novel KRAS inhibitors and we look forward to supporting Jazz in progressing these candidates through IND-enabling studies. Partnerships remain a key pillar of our corporate strategy, allowing us to advance what we believe are differentiated molecules in areas of high unmet need, while also creating long-term shareholder value through nondilutive funding, with the upfront milestone payment from this agreement extending our current cash runway into 2025."

Transaction Terms

Under the terms of the agreement, Jazz will make an upfront payment to Redx of $10 million for all rights, patents, title and interest relating to Redx’s proprietary KRAS inhibitor program, which includes G12D selective and panKRAS molecules. Redx is eligible to receive up to $870 million in development, regulatory and commercial milestone payments from Jazz, with the next milestone being an IND clearance from this program from the U.S. Food and Drug Administration. Redx is also eligible for tiered, mid-single digit percentage royalties based on any future net sales.

As part of a separate collaboration agreement, signed in parallel, Jazz will pay Redx to perform research and preclinical development activities with the goal of completing IND-enabling studies for both KRAS profiles.

About KRAS Inhibitors

Rat sarcoma virus (RAS) is the most frequently mutated oncogene across different cancer types, with KRAS mutations accounting for approximately 85% of these mutated oncogenes. Therefore, KRAS inhibitors targeting multiple commonly occurring mutations may offer a treatment option for large segments of colorectal, pancreatic and lung cancer patients who currently have limited treatment options. Developing orally bioavailable agents that generate optimized target coverage is a key opportunity for the next wave of KRAS-targeting agents that act beyond the G12C mutation.

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]).

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!

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]).

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.