Sensei Biotherapeutics Signs Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to Expand Development Plan for SNS-101, a Conditionally Active VISTA-blocking Antibody

On February 6, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), an immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported that it has signed a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), to expand the development of SNS-101, a conditionally active, pH-selective VISTA-blocking antibody (Press release, Sensei Biotherapeutics, FEB 6, 2023, View Source [SID1234626884]).

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Under the terms of the CRADA, Sensei will collaborate with the NCI Center for Immuno-Oncology Co-Directors, Jeffrey Schlom, Ph.D., and James Gulley, M.D., Ph.D., and conduct preclinical studies to assess the mechanism of action of SNS-101 in combination with novel therapeutic modalities. The goal of this collaborative effort between Sensei and the NCI is to further elucidate the role of VISTA in immune checkpoint resistance and expand the potential of SNS-101 as a combination therapy beyond anti-PD-1. Sensei expects to submit an Investigational New Drug (IND) application for SNS-101 in or before April 2023 and the trial is expected to commence thereafter in 2023, pending regulatory clearance.

"We’re excited to collaborate with Dr. Schlom and Dr. Gulley, who have pioneered the use of novel immunotherapies for a range of human cancers and are recognized for their expertise in translating preclinical findings into first-in-human studies for multiple immunotherapy agents," said Edward van der Horst, Ph.D., Chief Scientific Officer of Sensei Biotherapeutics. "We believe this collaboration has potential to increase the body of evidence that pH-selective VISTA inhibition can achieve a potentially differentiated efficacy and safety profile, expand the rationale for combining inhibition of VISTA with mechanisms beyond PD-1 inhibition, and help us build upon the current clinical development plan for SNS-101."

PDS Biotech Announces Abstract Accepted for Presentation at ESMO Targeted Anticancer Therapies Congress

On February 6, 2023 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, reported that an abstract investigating the ability of PDS0101 in combination with KEYTRUDA (pembrolizumab) to induce HPV16-specific CD4 and CD8 T cell activation and functionality has been accepted for presentation at the ESMO (Free ESMO Whitepaper) Targeted Anticancer Therapies Congress 2023 (ESMO TAT) in Paris, March 6-8, 2023 (Press release, PDS Biotechnology, FEB 6, 2023, View Source [SID1234626883]).

PDS Biotech’s HPV-targeted immunotherapy, PDS0101, is being studied in combination with KEYTRUDA, Merck’s anti-PD-1 therapy, in the Phase 2, VERSATILE-002 clinical trial (NCT04260126) in patients with advanced recurrent or metastatic HPV16-positive head and neck cancer. Measurement of antigen-specific activation of endogenous T cells is critical to understanding drug-induced, T cell based immunity and its association with observed clinical outcomes.

Abstract Number: 246
Abstract Title: HPV16-specific CD4 and CD8 T-cell activation and functionality in patients receiving combination PDS0101 immunotherapy
Authors: Dr. Lauren V. Wood, Dr. David Schaaf, Nathalie Riebel, and Sally Jones from PDS Biotech and Stephen McCarthy, Dr. Adam Cotty and Dr. Julie Bick from FlowMetric

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Neurocrine Biosciences Reports Fourth Quarter and Fiscal 2022 Financial Results and Provides Financial Expectations for 2023

On February 6, 2023 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the fourth quarter and fiscal year ended December 31, 2022 and provided financial guidance for 2023 (Press release, Neurocrine Biosciences, FEB 6, 2023, View Source [SID1234626882]).

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"We enter this year with tremendous momentum aiming to help the 7 out of 10 patients living with tardive dyskinesia in the U.S. who have not received a proper diagnosis for their uncontrollable movement disorder," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "Our capital allocation priorities remain: Invest in INGREZZA and in our pipeline. Enrollment is now complete in both the adult and pediatric registrational studies of crinecerfont for the treatment of congenital adrenal hyperplasia and we look forward to sharing the top-line data this year. With INGREZZA growing and our pipeline advancing, the fundamentals of our business are strong as we advance on our mission to be a leading neuroscience company."

Three Months Ended

December 31,

Twelve Months Ended

December 31,

(unaudited, in millions, except per share data)

2022

2021

2022

2021

Revenues:

Product sales, net

$ 404.6

$ 303.5

$ 1,440.9

$ 1,090.1

Collaboration revenue

7.4

8.5

47.8

43.4

Total revenues

$ 412.0

$ 312.0

$ 1,488.7

$ 1,133.5

GAAP Research and Development (R&D)

$ 118.0

$ 87.4

$ 463.8

$ 328.1

Non-GAAP R&D

$ 103.9

$ 75.2

$ 406.1

$ 279.7

GAAP Selling, General and Administrative (SG&A)

$ 182.9

$ 156.5

$ 752.7

$ 583.3

Non-GAAP SG&A

$ 151.8

$ 133.1

$ 635.6

$ 497.5

GAAP net income (loss)

$ 89.0

$ (7.3)

$ 154.5

$ 89.6

GAAP earnings (loss) per share – diluted

$ 0.88

$ (0.08)

$ 1.56

$ 0.92

Non-GAAP net income

$ 124.7

$ 4.3

$ 343.2

$ 185.8

Non-GAAP earnings per share – diluted

$ 1.24

$ 0.04

$ 3.47

$ 1.90

(unaudited, in millions)

December 31,

2022

December 31,

2021

Total cash, cash equivalents and marketable securities

$ 1,288.7

$ 1,272.0

Fourth Quarter and Fiscal 2022 INGREZZA Net Product Sales and Commercial Highlights:

INGREZZA fourth quarter and fiscal 2022 net product sales were $399 million and $1.43 billion, respectively
INGREZZA fourth quarter net product sales and total prescriptions grew 33% and 29%, respectively, vs. fourth quarter of 2021
Sequential growth driven by record new patients and continued strength in existing patients’ refill rates
Fourth Quarter Financial Highlights:

Fourth quarter 2022 GAAP net income and diluted earnings per share of $89 million and $0.88, respectively, compared with GAAP net loss and earnings per share of $7 million and $0.08, respectively, for fourth quarter 2021.
Fourth quarter 2022 non-GAAP net income and diluted earnings per share of $125 million and $1.24, respectively, compared with $4 million and $0.04, respectively, for fourth quarter 2021.
Differences in fourth quarter 2022 GAAP and non-GAAP operating expenses compared with fourth quarter 2021 driven by:
In-Process Research and Development (IPR&D) in fourth quarter 2021 associated with a $100 million upfront fee paid to Sosei Heptares pursuant to our exclusive license agreement
Increased R&D expense in support of an expanded and advancing clinical portfolio
Increased SG&A expense primarily due to ongoing commercial initiatives, including the deployment of the expanded salesforce in April 2022
At December 31, 2022, the Company had cash, cash equivalents and marketable securities of approximately $1.3 billion.
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.

Recent Events:

On November 1, 2022, we acquired Diurnal Group plc in an all-cash transaction, for an aggregate value of approximately $55 million. We believe the transaction presents an opportunity to accelerate the establishment of our clinical development and commercial capabilities in the United Kingdom to the benefit of patient communities and other stakeholders.
On December 22, 2022, we announced that the U.S. Food and Drug Administration accepted the supplemental New Drug Application for valbenazine as a treatment for chorea associated with Huntington Disease. The agency set a Prescription Drug User Fee Act target action date of August 20, 2023.
On January 8, 2023, we entered into a new strategic collaboration with Voyager Therapeutics, Inc., or Voyager, to acquire the worldwide rights to Voyager’s GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases and three gene therapy programs directed to rare central nervous system targets. This transaction is anticipated to close in the first quarter subject to certain conditions including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and other customary closing conditions.
On February 6, 2023, we announced enrollment is complete in both the Adult and Pediatric Registrational Studies of crinecerfont for Congenital Adrenal Hyperplasia. We anticipate top-line data for both studies to read-out in the second half of 2023.
Fiscal 2023 INGREZZA Sales and Operating Expense Guidance:

Range

(in millions)

Low

High

INGREZZA (Tardive Dyskinesia Only) Net Product Sales 1

$ 1,670

$ 1,770

GAAP R&D expense 2

$ 550

$ 580

Non-GAAP R&D expense 3

$ 495

$ 525

GAAP SG&A expense

$ 850

$ 870

Non-GAAP SG&A expense 3

$ 730

$ 750

INGREZZA sales guidance for fiscal 2023 reflects expected sales of INGREZZA in tardive dyskinesia only. The guidance range is based upon recent trends and underlying business initiatives underway to help advance the development of the tardive dyskinesia market.
GAAP R&D guidance reflects the progression of the Company’s pipeline including 12 mid-to-late-stage clinical studies, meaningful investments in the muscarinic portfolio and expanded pre-clinical research efforts. GAAP R&D guidance includes (i) amounts for milestones that are probable of achievement or have been achieved and (ii) amounts for in-process research and development once significant collaboration and licensing arrangements have been completed.
GAAP SG&A guidance reflects the continued investment in the expanded commercial organization to support INGREZZA as well as the Company’s direct-to-consumer advertising campaign. In addition, guidance includes an investment to support the anticipated approval for valbenazine to treat patients with chorea associated with Huntington disease. Non-GAAP guidance adjusted to exclude estimated non-cash stock-based compensation expense of $55 million in R&D and $120 million in SG&A.
2023 Expected Pipeline Milestones and Key Activities

Program

Indication

2023 Milestones / Key Activities

Valbenazine*

(Selective VMAT2 Inhibitor)

Chorea in Huntington Disease

PDUFA Aug. 20, 2023

Crinecerfont

(CRF1 Receptor Antagonist)

Congenital Adrenal Hyperplasia (Adult)

Top-Line Registrational Data in Second Half of 2023

Congenital Adrenal Hyperplasia (Pediatric)

Top-Line Registrational Data in Second Half of 2023

NBI-921352**

(Selective NaV1.6 Channel Blocker)

Focal Onset Seizure in Adults

Top-Line Phase 2 Data in Second Half of 2023

NBI-1065846†

(GPR-139 Agonist)

Anhedonia in Major Depressive Disorder

Top-Line Phase 2 Data in Second Half of 2023

NBI-1117570‡

(Dual M1/ M4 Agonist)

Treatment of Schizophrenia

Initiate Phase 1 Study

New Chemical Entity
or Entities

Indication(s) TBD

Initiate at Least One Phase 1 Study

Key: VMAT2 = Vesicular Monoamine Transporter 2; CFR1 = Corticotropin-Releasing Factor Type 1; NaV1.6 = Sodium Channel, Voltage-Gated; M1 / M4 = M1 / M4 Muscarinic Receptor; GPR = Orphan G Protein Coupled Receptor

Neurocrine Biosciences Partners: * Mitsubishi Tanabe Pharma Corporation has commercialization rights in East Asia; ** In-Licensed from Xenon Pharmaceuticals; † 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 866-952-8559 (US) or 785-424-1743 (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.

MacroGenics to Participate in Upcoming Investor Conferences

On February 6, 2023 MacroGenics, Inc. (Nasdaq: MGNX), a biopharmaceutical company focused on developing and commercializing innovative antibody-based therapeutics for the treatment of cancer, reported that the Company’s management will participate in the following investor conferences in February 2023 (Press release, MacroGenics, FEB 6, 2023, View Source [SID1234626881]):

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Guggenheim Healthcare Talks | Oncology Day (New York). MacroGenics’ President & Chief Executive Officer, Scott Koenig, M.D., Ph.D., will participate in a fireside chat on Thursday, February 9, 2023, at 2:45 pm ET. MacroGenics’ management will also participate in one-on-one meetings.
SVB Securities Global Biopharma Conference (Virtual). Scott Koenig, M.D., Ph.D., will participate in a formal presentation session with a SVB Securities research analyst on Tuesday, February 14, 2023, at 3:00 pm ET. MacroGenics’ management will also participate in one-on-one meetings.
A webcast of the above presentations may be accessed under "Events & Presentations" in the Investor Relations section of MacroGenics’ website at View Source The Company will maintain an archived replay of this webcast on its website for 30 days.

Ligand to Report Fourth Quarter Financial Results on February 22

On February 6, 2023 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) will report fourth quarter 2022 financial results after the close of the U.S. financial markets on Wednesday, February 22, 2023 and will hold a conference call that same day beginning at 4:30 p.m. Eastern time (Press release, Ligand, FEB 6, 2023, View Source [SID1234626880]). Speakers on the call will include Ligand’s CEO Todd Davis, President and COO Matt Korenberg and CFO Tavo Espinoza.

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Conference Call and Webcast Information

What:

Ligand conference call to discuss financial results and provide general business updates

Date:

Wednesday, February 22, 2023

Time:

4:30 p.m. Eastern time (1:30 p.m. Pacific time)

Conference Call:

Dial (888) 350-3452

Conference ID is 6501694

Webcast:

Live and replay webcast of the call are available here.