On May 4, 2023 Intra-Cellular Therapies, Inc. (Nasdaq: ITCI), a biopharmaceutical company focused on the development and commercialization of therapeutics for central nervous system (CNS) disorders, reported its financial results for the first quarter ended March 31, 2023 and provided a corporate update (Press release, Intra-Cellular Therapies, MAY 4, 2023, View Source [SID1234631033]).

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"We are pleased with the progress we have made in the first quarter and we are confident in the continued growth of CAPLYTA," said Dr. Sharon Mates, Chairman and CEO of Intra-Cellular Therapies. "We are excited about our recent positive results in our clinical study in patients with mixed features in MDD and mixed features in bipolar depression. We are also encouraged by the progress we have made with our pipeline programs."

First Quarter Financial Highlights:

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Total revenues were $95.3 million for the first quarter of 2023, compared to $35.0 million for the same period in 2022. Net product sales of CAPLYTA were $94.7 million for the first quarter of 2023, compared to $34.8 million for the same period in 2022, representing a year-over-year increase of 173% and an 8% increase over the fourth quarter of 2022. First quarter CAPLYTA total prescriptions increased sequentially by 16% versus the fourth quarter of 2022.

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Net loss for the first quarter of 2023 was $44.1 million compared to a net loss of $72.1 million for the same period in 2022.

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Cost of product sales was $6.8 million in the first quarter of 2023 compared to $3.2 million for the same period in 2022.

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Selling, general and administrative (SG&A) expenses were $98.9 million for the first quarter of 2023, compared to $75.5 million for the same period in 2022. This increase is primarily due to an increase in marketing and advertising costs.

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Research and development (R&D) expenses were $38.0 million for the first quarter of 2023, compared to $29.0 million for the same period in 2022. This increase is primarily due to higher lumateperone project costs and non-lumateperone project costs.

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Cash, cash equivalents, restricted cash and investment securities totaled $540.5 million at March 31, 2023, compared to $593.7 million at December 31, 2022.

Reiterating Fiscal 2023 Financial Outlook:

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Reiterating our financial guidance for FY 2023 including CAPLYTA net product sales of $430 to $455 million.

COMMERCIAL HIGHLIGHTS

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CAPLYTA’s strong uptake continued in the first quarter of 2023 with total prescriptions increasing by 159% compared to the first quarter of 2022. First quarter CAPLYTA total prescriptions increased sequentially by 16% versus the fourth quarter of 2022.

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CAPLYTA maintained broad coverage in the Medicare Part D and Medicaid channels, with greater than 98% of lives covered and increased Commercial coverage to approximately 90% of lives covered by the end of the first quarter of 2023.

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Our LytaLink patient and prescriber support program continues to be very effective in supporting patient access to CAPLYTA.

CLINICAL HIGHLIGHTS

Lumateperone:

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Mixed Features program: Announced robust positive results from Study 403 evaluating lumateperone 42mg as monotherapy in the treatment of major depressive episodes in patients with MDD with mixed features and in patients with bipolar depression with mixed features.

In this study, lumateperone 42mg met the primary endpoint by demonstrating a statistically significant reduction in the Montgomery Asberg Depression Rating Scale (MADRS) total score compared to placebo at Week 6 in the combined patient population of MDD with mixed features and bipolar depression with mixed features (5.7 point reduction v. placebo; p<0.0001; Cohen’s d effect size (ES) of 0.64). Robust results were also seen in the individual patient population of MDD with mixed features (5.9 point reduction v. placebo; p<0.0001; ES= 0.67), and in the individual patient population of bipolar depression with mixed features (5.7 point reduction v. placebo; p<0.0001; ES= 0.64).

Lumateperone 42mg also met the key secondary endpoint in the study by demonstrating a statistically significant and clinically meaningful reduction in the clinician’s assessment of improvement in the overall severity on the Global Impression of Severity Scale (CGI-S) score compared to placebo at Week 6 in the combined patient population of MDD with mixed features and bipolar depression with mixed features (p<0.0001; ES= 0.59) and in the individual patient population of MDD with mixed features (p=0.0003; ES= 0.57), as well as the individual patient population of bipolar depression with mixed features p<0.0001; ES=0.61). Lumateperone was generally safe and well tolerated, with a side effect profile consistent with prior lumateperone trials.

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Adjunctive MDD program: Lumateperone is in Phase 3 clinical development as a novel treatment for MDD. Patient enrollment in Study 501 and Study 502, global Phase 3 clinical trials evaluating lumateperone 42 mg as an adjunctive therapy to antidepressants for the treatment of MDD, is ongoing. In addition, we recently initiated a third global Phase 3 trial, Study 505, also evaluating lumateperone 42 mg as an adjunctive therapy to antidepressants for the treatment of MDD. Subject to the results of Studies 501 and 502, we expect to file a supplemental New Drug Application with the FDA in 2024 for approval of lumateperone as an adjunctive therapy to antidepressants for the treatment of MDD.

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Lumateperone Long Acting Injectable (LAI) formulation: The goal of our program is to develop LAI formulations that are effective, safe and well-tolerated with treatment durations of one month or longer. We are undertaking Phase 1 single ascending dose studies with several formulations in 2023 and next year.

Other pipeline programs:

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ITI-1284-ODT-SL program: ITI-1284 is a deuterated form of lumateperone, a new chemical entity formulated as an oral disintegrating tablet for sublingual administration. We plan to initiate Phase 2 programs in agitation in patients with Alzheimer’s disease (AD), in psychosis in patients with AD and in generalized anxiety disorder in 2023.

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Phosphodiesterase type I inhibitor (PDE1) program: Our portfolio of PDE1 inhibitors is being developed to treat diseases in which PDE1 activity is over active.

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Lenrispodun (ITI-214) is our lead PDE1 inhibitor compound. Patient enrollment in a Phase 2 clinical trial in Parkinson’s disease (PD) commenced in the first quarter of 2023. The objective of this proof-of-concept study is to evaluate improvements in motor symptoms in patients with PD. Changes in cognition and inflammatory biomarkers will also be assessed.

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ITI-1020 is our second drug candidate in our PDE1 inhibitor program. We have an active Investigational New Drug application to evaluate ITI-1020 as a novel cancer immunotherapy. We have commenced a Phase 1 program in healthy volunteers.

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ITI-333 program: ITI-333, a 5-HT2A receptor antagonist and µ-opioid receptor partial agonist, provides potential utility in the treatment of opioid use disorder, pain and mood disorders. We have two ongoing studies: a multiple ascending dose study in healthy volunteers evaluating pharmacokinetics, safety and tolerability and a neuroimaging study.

Conference Call and Webcast Details

The Company will host a live conference call and webcast today at 8:30 AM Eastern Time to discuss the Company’s financial results and provide a corporate update. To attend the live conference call by phone please use this registration link (View Source). All participants must use the link to complete the online registration process in advance of the conference call.

The live and archived webcast can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.intracellulartherapies.com. Please log in approximately 5-10 minutes prior to the event to register and to download and install any necessary software.

CAPLYTA (lumateperone) is indicated in adults for the treatment of schizophrenia and depressive episodes associated with bipolar I or II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate.

Important Safety Information

Boxed Warnings:

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Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death. CAPLYTA is not approved for the treatment of patients with dementia-related psychosis.

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Antidepressants increased the risk of suicidal thoughts and behaviors in pediatric and young adults in short-term studies. All antidepressant-treated patients should be closely monitored for clinical worsening, and for emergence of suicidal thoughts and behaviors. The safety and effectiveness of CAPLYTA have not been established in pediatric patients.

Contraindications: CAPLYTA is contraindicated in patients with known hypersensitivity to lumateperone or any components of CAPLYTA. Reactions have included pruritus, rash (e.g., allergic dermatitis, papular rash, and generalized rash), and urticaria.

Warnings & Precautions: Antipsychotic drugs have been reported to cause:

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Cerebrovascular Adverse Reactions in Elderly Patients with Dementia-Related Psychosis, including stroke and transient ischemic attack. See Boxed Warning above.

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Neuroleptic Malignant Syndrome (NMS), which is a potentially fatal reaction. Signs and symptoms include: high fever, stiff muscles, confusion, changes in breathing, heart rate, and blood pressure, elevated creatinine phosphokinase, myoglobinuria (and/or rhabdomyolysis), and acute renal failure. Patients who experience signs and symptoms of NMS should immediately contact their doctor or go to the emergency room.

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Tardive Dyskinesia, a syndrome of uncontrolled body movements in the face, tongue, or other body parts, which may increase with duration of treatment and total cumulative dose. TD may not go away, even if CAPLYTA is discontinued. It can also occur after CAPLYTA is discontinued.

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Metabolic Changes, including hyperglycemia, diabetes mellitus, dyslipidemia, and weight gain. Hyperglycemia, in some cases extreme and associated with ketoacidosis, hyperosmolar coma or death, has been reported in patients treated with antipsychotics. Measure weight and assess fasting plasma glucose and lipids when initiating CAPLYTA and monitor periodically during long-term treatment.

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Leukopenia, Neutropenia, and Agranulocytosis (including fatal cases). Complete blood counts should be performed in patients with pre-existing low white blood cell count (WBC) or history of leukopenia or neutropenia. CAPLYTA should be discontinued if clinically significant decline in WBC occurs in absence of other causative factors.

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Decreased Blood Pressure & Dizziness. Patients may feel lightheaded, dizzy or faint when they rise too quickly from a sitting or lying position (orthostatic hypotension). Heart rate and blood pressure should be monitored and patients should be warned with known cardiovascular or cerebrovascular disease. Orthostatic vital signs should be monitored in patients who are vulnerable to hypotension.

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Falls. CAPLYTA may cause sleepiness or dizziness and can slow thinking and motor skills, which may lead to falls and, consequently, fractures and other injuries. Patients should be assessed for risk when using CAPLYTA.

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Seizures. CAPLYTA should be used cautiously in patients with a history of seizures or with conditions that lower seizure threshold.

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Potential for Cognitive and Motor Impairment. Patients should use caution when operating machinery or motor vehicles until they know how CAPLYTA affects them.

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Body Temperature Dysregulation. CAPLYTA should be used with caution in patients who may experience conditions that may increase core body temperature such as strenuous exercise, extreme heat, dehydration, or concomitant anticholinergics.

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Dysphagia. CAPLYTA should be used with caution in patients at risk for aspiration.

Drug Interactions: CAPLYTA should not be used with CYP3A4 inducers. Dose reduction is recommended for concomitant use with strong CYP3A4 inhibitors or moderate CYP3A4 inhibitors.

Special Populations: Newborn infants exposed to antipsychotic drugs during the third trimester of pregnancy are at risk for extrapyramidal and/or withdrawal symptoms following delivery. Breastfeeding is not recommended. Dose reduction is recommended for patients with moderate or severe hepatic impairment.

Adverse Reactions: The most common adverse reactions in clinical trials with CAPLYTA vs. placebo were somnolence/sedation, dizziness, nausea, and dry mouth.

CAPLYTA is available in 10.5 mg, 21 mg, and 42 mg capsules.

Please click here to see full Prescribing Information including Boxed Warning.

About CAPLYTA (lumateperone)

CAPLYTA 42 mg is an oral, once daily atypical antipsychotic approved in adults for the treatment of schizophrenia and depressive episodes associated with bipolar I or II disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate. While the mechanism of action of CAPLYTA is unknown, the efficacy of CAPLYTA could be mediated through a combination of antagonist activity at central serotonin 5-HT2A receptors and postsynaptic antagonist activity at central dopamine D2 receptors.

Lumateperone is being studied for the treatment of major depressive disorder, and other neuropsychiatric and neurological disorders. Lumateperone is not FDA-approved for these disorders.

On May 4, 2023 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, reported operational highlights and financial results for the first quarter ended March 31, 2023 (Press release, Intellia, MAY 4, 2023, View Source [SID1234631032]).

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"We’ve had a productive start to the year with the successful clearance of our first IND application for an investigational in vivo CRISPR-based therapy alongside broad pipeline and platform progress," said Intellia President and Chief Executive Officer John Leonard, M.D. "Today, we are pleased to announce dosing has begun in the Phase 2 study of NTLA-2002, and based on strong interest from investigators and patients, we expect to complete enrollment in the second half of this year. Our rapid progression of the clinical development of NTLA-2002 supports our goal to bring forth a potential functional cure for the treatment of hereditary angioedema. Simultaneously, we are working toward submitting our second in vivo IND application and initiating a global pivotal trial for NTLA-2001. We look forward to sharing new interim data from both the NTLA-2001 and NTLA-2002 first-in-human studies in the months to come."

First Quarter 2023 and Recent Operational Highlights

In Vivo Program Updates

Transthyretin (ATTR) Amyloidosis

NTLA-2001: NTLA-2001 is an in vivo, systemically delivered, investigational CRISPR-based therapy designed to inactivate the TTR gene in liver cells and thereby prevent the production of transthyretin (TTR) protein for the treatment of ATTR amyloidosis. NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, consistent and potentially lifelong reduction in TTR protein after a single dose. NTLA-2001 is subject to a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc.
ATTR Amyloidosis with Cardiomyopathy (ATTR-CM):
Intellia plans to submit a U.S. Investigational New Drug (IND) application in mid-2023. Subject to regulatory feedback, the Company anticipates initiating a global pivotal trial for ATTR-CM by year-end 2023.
The Company expects to present additional data from the ATTR-CM arm of the Phase 1 study in 2023, including longer-term safety and durability data, as well as emerging clinical endpoints.
Hereditary ATTR Amyloidosis with Polyneuropathy (ATTRv-PN):
As previously announced, during the first quarter of 2023, the planned enrollment of the dose-expansion portion of the ATTRv-PN arm in the Phase 1 study was completed to inform a pivotal study. The Company is actively preparing for a global pivotal study, which includes discussions with regulatory authorities.
Intellia recently began redosing patients in the 0.1 mg/kg cohort (n=3), the initial cohort and lowest dose tested in the dose-escalation portion of the Phase 1 study, with the 55 mg dose selected for the dose-expansion cohort.
The Company plans to present additional clinical data from the ATTRv-PN arm of the Phase 1 study in 2023.
Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 is a wholly owned, in vivo, systemically delivered investigational CRISPR-based therapy. NTLA-2002 is designed to knock out the KLKB1 gene in the liver, with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator of HAE. This investigational approach aims to prevent attacks for people living with HAE by providing continuous reduction of plasma kallikrein activity, following a single dose. It also aims to eliminate the significant treatment burden associated with currently available HAE therapies. NTLA-2002 is being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE.
Intellia announced today that the first patient has been dosed in the global Phase 2 portion of its Phase 1/2 clinical trial of NTLA-2002. Based on encouraging study interest from both investigators and patients, the Company expects to complete enrollment (n=25) in 2H 2023.
The Company announced in March that the U.S. Food and Drug Administration (FDA) cleared the NTLA-2002 Phase 2 IND application. Additionally, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for the treatment of HAE.
In January 2023, Intellia was awarded the Innovation Passport for NTLA-2002 by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). The Innovation Passport is the point of entry into the U.K.’s Innovative Licensing and Access Pathway (ILAP), which is designed to accelerate time to market and facilitate patient access to innovative medicines.
The Company plans to present additional clinical data from the Phase 1 portion of the first-in-human study in 2023. Data expected to be presented include updated safety, durability of pharmacodynamic effect and attack-rate measures from all three patient cohorts.
Alpha-1 Antitrypsin Deficiency (AATD)

NTLA-3001 for Associated Lung Disease: NTLA-3001 is a wholly owned, first-in-class CRISPR-mediated in vivo targeted gene insertion development candidate for the treatment of AATD-associated lung disease. It is designed to precisely insert a healthy copy of the SERPINA1 gene, which encodes the alpha-1 antitrypsin (A1AT) protein, with the potential to restore permanent expression of functional A1AT protein to therapeutic levels after a single dose. This approach seeks to improve patient outcomes, including eliminating the need for weekly intravenous infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting IND-enabling activities for NTLA-3001 and plans to submit an IND or IND-equivalent filing in 2H 2023.
NTLA-2003 for Associated Liver Disease: NTLA-2003 is a wholly owned, in vivo knockout development candidate for the treatment of AATD-associated liver disease. It is designed to inactivate the SERPINA1 gene responsible for the production of abnormal A1AT protein in the liver. This approach aims to halt the progression of liver disease and eliminate the need for liver transplant in severe cases.
Intellia is conducting IND-enabling activities for NTLA-2003, with the expectation of completing these activities by year-end 2023.
Ex Vivo Program Updates

Immuno-oncology and Autoimmune Diseases

Intellia is advancing multiple preclinical programs, wholly owned and in collaboration with partners, utilizing its allogeneic platform for the treatment of immuno-oncology and autoimmune diseases. The Company’s proprietary allogeneic cell engineering platform is designed to avoid both T cell- and NK cell-mediated rejection, a key unsolved challenge with other investigational allogeneic approaches.
Research and Corporate Updates

Modular Platform and Pipeline Expansion: Intellia is expanding its industry-leading genome editing platform and scientific leadership through editing, delivery and cell engineering innovations that may enable broader in vivo and ex vivo applications.
Board of Directors Update: In April, Intellia announced the appointment of Bill Chase to its board of directors. Mr. Chase will be a member of the audit committee and will succeed Caroline Dorsa as chair of the audit committee upon her retirement from the board on June 15, 2023.
Corporate Responsibility Report: In May, Intellia published its 2023 Corporate Responsibility Report. The report highlights the Company’s Environmental, Social and Governance (ESG) principles and practices as part of its objective to build a sustainable company while delivering on its commitments to patients, employees and shareholders.
Upcoming Events

The Company will participate in the following events during the second quarter of 2023:

Bank of America Securities 2023 Health Care Conference, May 9, Las Vegas
2023 RBC Capital Markets Global Healthcare Conference, May 16, New York City
Barclays Gene Editing & Therapy Summit, May 24, New York City
Stifel Genetic Medicines Day, May 30, virtual
Upcoming Milestones

The Company has set forth the following expected milestones for pipeline progression:

NTLA-2001 for ATTR amyloidosis:
Submit an IND application in mid-2023 to enable inclusion of U.S. sites in a pivotal study of NTLA-2001 for patients with ATTR-CM.
Present additional clinical data from the ongoing Phase 1 study of NTLA-2001 in 2023.
Initiate a global pivotal NTLA-2001 trial for ATTR-CM by year-end 2023, subject to regulatory feedback.
Prepare for a Phase 3 study of NTLA-2001 for the treatment of ATTRv-PN, including discussions with regulatory authorities.
NTLA-2002 for HAE:
Present additional clinical data from the ongoing first-in-human study of NTLA-2002 in 2023.
Complete enrollment in the Phase 2 portion of the Phase 1/2 study in 2H 2023.
AATD Franchise:
Submit an IND or IND-equivalent application for NTLA-3001 for AATD-associated lung disease in 2H 2023.
Complete IND-enabling activities for NTLA-2003 for AATD-associated liver disease by year-end 2023.
Platform Innovation:
Advance novel gene editing technologies, including DNA writing and delivery to other tissues outside of the liver.
First Quarter 2023 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1.2 billion as of March 31, 2023, compared to $1.3 billion as of December 31, 2022. The decrease was driven by cash used to fund operations of approximately $120.9 million. The decrease was offset in part by $12.0 million of interest income, $3.4 million of reimbursement from its collaborators, $1.5 million of net equity proceeds from the Company’s "At the Market" (ATM) program and $0.8 million in proceeds from employee-based stock plans.
Collaboration Revenue: Collaboration revenue increased by $1.3 million to $12.6 million during the first quarter of 2023, compared to $11.3 million during the first quarter of 2022.
R&D Expenses: Research and development expenses decreased by approximately $36.0 million to $97.1 million during the first quarter of 2023, compared to $133.1 million during the first quarter of 2022. This decrease was primarily driven by $56.0 million of expense related to the acquisition of Rewrite Therapeutics, Inc. during the first quarter of 2022. The decrease related to the acquisition of Rewrite Therapeutics, Inc. was offset by an increase in expenses of $20.0 million primarily driven by the advancement of its lead programs and personnel growth to support these programs. Stock-based compensation expense included in research and development expenses was $16.9 million for the first quarter of 2023.
G&A Expenses: General and administrative expenses increased by $5.0 million to $27.4 million during the first quarter of 2023, compared to $22.4 million during the first quarter of 2022. This increase was primarily related to an increase in stock-based compensation of $2.1 million. Stock-based compensation expense included in general and administrative expenses was $10.3 million for the first quarter of 2023.
Net Loss: The Company’s net loss was $103.1 million for the first quarter of 2023, compared to $146.9 million during the first quarter of 2022.
Conference Call to Discuss First Quarter 2023 Results

The Company will discuss these results on a conference call today, Thursday, May 4, at 8 a.m. ET.
To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at intelliatx.com, beginning on May 4, at 12 p.m. ET.

On May 4, 2023 INmune Bio Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported its financial results for the quarter ended March 31, 2023 and provides a business update (Press release, INmune Bio, MAY 4, 2023, View Source [SID1234631031]).

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Q1 2023 Corporate Highlights:

DN-TNF Platform Highlights (XPro and INB03):

● AD02 blinded randomized Phase 2 clinical trial in patients with early Alzheimer’s Disease (AD) continues to enroll in Australia and Canada. The company is actively pursuing other regulatory venues to expand the clinical trial footprint. The company announced consolidation of the mild ADi and Mild Cognitive Impairment Phase 2 programs into a single trial to improve the efficiency and align the program with the most probable Phase 3 design. Discussions with the FDA have provided a clear pathway to lifting the clinical hold before the end of year.

● Announced data in acute mdx model of Duchene’s Muscular Dystrophy (DMD) and formed DN02, Inc. a wholly owned subsidiary to facilitate partnering and development of DN-TNF in the treatment of DMD. Results from the long-term D2.mdx model are expected soon. Data presented to date suggests DN-TNF may improve treatment from boys with DMD by promoting muscle fiber regeneration and eliminating the need for corticosteroids. Corticosteroids, the current standard-of-care, is responsible for many of the metabolic and physical complications suffered by boys with DMD.

● Presented additional data on combination of INB03 DN-TNF with trastuzumab-deruxtecan (Enhertu, TDxd) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual scientific meeting in April showing that triple negative breast cancer (TNBC) often express MUC4. MUC4 expressing cancers, breast cancer, HER2+ breast cancer, TNBC, gastric and pancreatic cancer, are candidates for combination therapy with DN-TNF. In pre-clinical models, INB03 DN-TNF reduced MUC4 expression to decrease resistance to immunotherapy including tyrosine kinase inhibitors (TKIs), trastuzumab and TDxd. The Company is developing a novel DN-TNF-bioconjugate compound with distinct intellectual property protections and testing in a variety of animal models in preparation of launching a strategic partnering program in oncology.

● Announced pre-clinical, mouse model data that support the use of XPro as an early treatment following Traumatic Brain Injury (TBI) to prevent the development of amyloid pathology. Data shows neuroinflammation drives the development of amyloid pathology after TBI and can be prevented by neutralizing sTNF with XPro. These data implicated TBI as a contributing factor for the development of AD.

INKmune Platform:

● IND filed in April for the use of INKmune to treat patients with metastatic castration-resistant prostate cancer (mCRPC) in the US. Excluding skin cancer, prostate cancer is the most common cancer in men. The Phase I/II design will enroll patients at several medical centers in the US.

● Additional sites have opened to support the ongoing Phase 1 trial in high risk MDS/AML. A second site in the UK, The Royal Hallamshire Hospital at Sheffield University Medical School treated their first patient on March 9th. A third site in Europe, Attikon University Hospital in Athens, Greece, has opened for recruitment and INKmune is being imported into Greece in readiness for patient enrollment.

● Our CSO Mark Lowdell co-hosted a session at the Innate Killer Summit in La Jolla in March focused on the expanding world of memory-like NK cells in cancer immunotherapy and the ability of INKmune to generate these cells in vivo.

● On June 1st, Dr Lowdell will present the opening plenary presentation in the Presidential Session at the annual International Society of Cell & Gene Therapy (ISCT) where he will share the latest data on INKmune.

Upcoming Events and Milestones:

● Top-line results for the Phase 2 XPro trial for treatment of neuroinflammation as a cause of Alzheimer’s Disease is expected in 2H 2024.

● Initiate a Phase 2 trial of XPro in patients with Treatment Resistant Depression that is partially funded by a $2.9 million NIH grant upon resolution of the FDA manufacturing review.

● Additional open-label Phase 1 trial data of INKmune in high-risk MDS/AML in 2023.

● Opening of a Phase I/II trial studying INKmune in prostate cancer upon the acceptance of the IND by the FDA.

Financial Results for the Quarter Ended March 31, 2023:

Net loss attributable to common stockholders for the quarter ended March 31, 2023 was approximately $6.5 million, compared to approximately $6.9 million during the quarter ended March 31, 2022.

Research and development expense totaled approximately $4.1 million for the quarter ended March 31, 2023 compared to approximately $4.3 million during the quarter ended March 31, 2022.

General and administrative expenses were approximately $2.3 million for the quarter ended March 31, 2023 compared to approximately $2.3 million during the quarter ended March 31, 2022.

Other expense was approximately $0.1 million for the quarter ended March 31, 2023 compared to approximately $0.4 million during the quarter ended March 31, 2022.

In addition, the Company received research and development rebates from Australia and the United Kingdom during the three months ended March 31, 2023 that totaled approximately $6.5 million.

As of March 31, 2023, the Company had cash and cash equivalents of approximately $51.0 million.

As of May 3, 2023, the Company had approximately 17.9 million common shares outstanding.

Earnings Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call. Please ask for the INmune Bio First Quarter Conference Call when reaching an operator.

Date: May 3, 2023
Time: 4:30 PM Eastern Time
Participant Dial-in: 1-844-826-3035 Participant Dial-in (international): 1-412-317-5195
Conference ID: 10177985

A live audio webcast of the call can be accessed using this link or clicking here:
View Source

A transcript will follow approximately 24 hours from the scheduled call. A replay will also be available through May 10 by dialing 1-844-512-2921 or 1-412-317-6671 (international) and entering PIN no. 10177985.

About XPro

XPro is a next-generation inhibitor of tumor necrosis factor (TNF) that is currently in clinical trial and acts differently than currently available TNF inhibitors in that it neutralizes soluble TNF (sTNF), without affecting trans-membrane TNF (tmTNF) or TNF receptors. XPro could have potential substantial beneficial effects in patients with neurologic disease by decreasing neuroinflammation. For more information about the importance of targeting neuroinflammation in the brain to improve cognitive function and restore neuronal communication visit this section of the INmune Bio’s website.

About INKmune

INKmune is a pharmaceutical-grade, replication-incompetent human tumor cell line which conjugates to resting NK cells and delivers multiple, essential priming signals to convert the cancer patient’s resting NK cells into tumor killing memory-like NK cells. INKmune treatment’s effect on NK cells is akin to treatment with at least three cytokines in combination (IL-12, IL-15, IL-18) to form memory-like NK cells. In patients, INKmune primed tumor killing NK cells persist for more than 100 days and function in the in the hypoxic TME because due to upregulated nutrient and mitochondrial survival proteins. INKmune is a patient friendly therapy that can be easily transported, stored and delivered to the patient by a simple intravenous infusion without the need for patient conditioning or premedication. INKmune is tumor agnostic; it can be used to treat many types of NK-resistant tumors including leukemia, lymphoma, myeloma, lung, ovarian, breast, renal and nasopharyngeal cancer. INKmune is treating patients in an open label Phase I trial in high-MDS/AML in the UK and Europe. The company plans an open label Phase I/II trial in metastatic castration-resistant prostate cancer in the US this year.

On May 4, 2023 ImmunoGen Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported the pricing of an underwritten public offering of 26,000,000 shares of its common stock at a price of $12.50 per share, before underwriting discounts and commissions (Press release, ImmunoGen, MAY 4, 2023, View Source [SID1234631030]). The offering is expected to close on or about May 9, 2023, subject to satisfaction of customary closing conditions. ImmunoGen also granted the underwriters a 30-day option to purchase up to an additional 3,900,000 shares of its common stock at the public offering price, less underwriting discounts and commissions. All of the shares of common stock in the offering are to be sold by ImmunoGen.

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ImmunoGen anticipates the total gross proceeds from the offering (before deducting the underwriting discounts and estimated offering expenses) will be $325 million, excluding any exercise of the underwriters’ option to purchase additional shares.

ImmunoGen intends to use the net proceeds from this offering to fund its operations, including, but not limited to, global commercialization activities, supply of ELAHERE (mirvetuximab soravtansine-gynx) drug product, clinical trial activities, pipeline research and development activities, business development activities, and capital expenditures.

Jefferies, Goldman Sachs & Co. LLC, and Guggenheim Securities are acting as joint book-running managers for the proposed offering. Canaccord Genuity is acting as lead manager for the proposed offering.

The securities described above are being offered by ImmunoGen pursuant to a shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC) and became effective upon filing. This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in this offering, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. A preliminary prospectus supplement and accompanying prospectus relating to the offering was filed with the SEC and is available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by e-mail at [email protected] or by telephone at (877) 821-7388; Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by phone at (866) 471-2526, or by email at [email protected]; or Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, or by email at [email protected] or by telephone at (212) 518-9544.

On May 4, 2023 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio", "Company", "We" or "Us") reported that updated NXC-201 clinical data has been selected for presentation at the upcoming 26th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) to be held in Los Angeles May 16-20, 2023 (Press release, Immix Biopharma, MAY 4, 2023, View Source [SID1234631029]).

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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

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"We are delighted to present additional clinical data for what we believe is the only CAR-T in development in AL Amyloidosis at the upcoming 26th Annual Meeting of The American Society of Gene & Cell Therapy," said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator for the NEXICART-1 Phase 1b/2a clinical trial of NXC-201. "A potential one-time treatment such as CAR-T NXC-201 in AL amyloidosis would be a welcome option for this devastating disease."

Oral Presentation:

Title: "BCMA-Targeted CART (HBI0101), a Safe and Efficacious Novel Modality of Treatment for Light Chain Amyloidosis Patients"
Oral Presentation Date/Time: Friday May 19, 2023, 9:15am – 9:30am
Event: 26th Annual Meeting of The American Society of Gene and Cell Therapy, Los Angeles, CA
Session Title: Late-Breaking Abstracts 1
Session Date/Time: Friday May 19, 2023, 8:00am – 9:45am

About NEXICART-1

NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed or refractory multiple myeloma and AL amyloidosis.

The primary objective of the Phase 1b portion of the study is to characterize the safety and confirm the Maximally Tolerated Dose (MTD) and Phase 2 dose of NXC-201. The Phase 2 portion of the study will evaluate the efficacy and safety of NXC-201 with endpoints of overall survival, progression-free survival and response rates according to International Myeloma Working Group (IMWG) Uniform Response Criteria.

The Phase 1b portion of the ongoing Phase 1b/2 clinical trial has been successful in determining the recommended Phase 2 dose (RP2D) of 800 million CAR+T cells. Over the coming months, Nexcella (an Immix Biopharma subsidiary) plans to submit an IND application to the FDA for a Phase 1b/2 of NXC-201 in relapsed/refractory multiple myeloma and AL amyloidosis in order to expand the ongoing clinical trial to the U.S. The expected primary endpoint for the Phase 2 portion of the ongoing Phase 1b/2a clinical trial of NXC-201 in relapsed/refractory multiple myeloma is overall response rate and duration of response. Nexcella plans to submit data to the FDA in multiple myeloma once 100 patients are treated with NXC-201. The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate. Nexcella plans to submit data to the FDA in AL amyloidosis once 30-40 patients are treated with NXC-201.

About NXC-201

NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis.

About Multiple Myeloma

Multiple myeloma ("MM") is an incurable blood cancer of plasma cells that starts in the bone marrow and is characterized by an excessive proliferation of these cells. Despite initial remission, unfortunately, most patients are likely to relapse. There are 35,730 patients in the United States diagnosed with MM each year. Prognosis for patients who do not respond to or relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents remains poor. The $13.9 billion Multiple Myeloma market in 2017 is expected to reach $28.7 billion in 2027 according to Wilcock, et al. Nature Reviews.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage, and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S. The annual global incidence of AL Amyloidosis is ~15,000 patients.

The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research.