UroGen Pharma to Report Third Quarter 2022 Financial Results on Thursday, November 10, 2022

On November 3, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it will report third quarter 2022 financial results on Thursday, November 10, 2022, prior to the open of the market (Press release, UroGen Pharma, NOV 3, 2022, View Source [SID1234623005]). The announcement will be followed by a live audio webcast and conference call at 10:00 AM Eastern Time.

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A live public webcast of the earnings conference call can be accessed on UroGen’s Investor Relations website. Following the live webcast, a replay will be available on the site for approximately 30 days.

XOMA Reports Third Quarter 2022 Financial Results and Highlights Recent Operational Events

On November 3, 2022 XOMA Corporation (Nasdaq: XOMA), a biotech royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of advancing novel therapeutic candidates aimed at improving human health, reported its third quarter 2022 financial results and provided a recent operations update (Press release, Xoma, NOV 3, 2022, View Source [SID1234623004]).

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"The third quarter was a turning point for XOMA as a royalty and milestone aggregator. Less than one year after we acquired the 0.5% economic interest in VABYSMO sales, we have received our first commercial payment from Roche. The therapy is now available to retinal specialists and their patients in three important markets – the U.S., EU, and Japan," stated Jim Neal, Chairman and Chief Executive Officer of XOMA. "Separately, as a result of a monetization transaction we closed in 2021 with Kuros Biosciences associated with vidutolimod, which was being developed by Checkmate Pharmaceuticals, we received a $2.5 million payment as Regeneron completed its acquisition of Checkmate. We look forward to additional business and clinical milestone achievements from our partners as they advance their assets."

Financial Results

XOMA recorded total revenues of $0.5 million for the third quarter of 2022 and $0.9 million for the third quarter of 2021. The decrease for the three months ended September 30, 2022, as compared to the same period in 2021, was primarily due to $0.5 million of milestone revenue recognized in the third quarter of 2021 under XOMA’s license agreement with Compugen.

Research and development ("R&D") expenses were $29,000 and $30,000, respectively, for the third quarters of 2022 and 2021.

General and administrative ("G&A") expenses were $4.8 million for the third quarter of 2022, compared to $4.3 million for the third quarter of 2021. The additional $0.5 million during the third quarter of 2022 reflects increased consulting and legal expenses associated with deal costs.

In the third quarter of 2022, G&A expenses included $0.8 million in non-cash stock-based compensation expense, which was consistent with the third quarter of 2021. XOMA’s net cash used in operations in the third quarter of 2022 was $3.7 million, as compared with $3.1 million during the third quarter of 2021.

Other income, net was $0.2 million for the third quarter of 2022, compared to other expense, net of $1.1 million in the corresponding quarter of 2021. The fluctuation in other income (expense), net between the quarters ended September 30, 2022 and 2021, is primarily due to the change in the fair value of equity securities XOMA holds in Rezolute, Inc.

Net loss for the third quarter of 2022 was $4.2 million, compared to net loss of $4.4 million for the third quarter of 2021.

On September 30, 2022, XOMA had cash of $78.3 million. In August 2022, XOMA received a $0.5 million cash payment from Roche, representing the first commercial payment for XOMA’s 0.5% commercial interest in the sales of VABYSMO (faricimab-svoa). The payment was recorded in the Company’s condensed consolidated balance sheet as of September 30, 2022, as a reduction of long-term royalty and commercial payment receivables. On October 17, 2022, the Company paid cash dividends on the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) equal to $0.53906 per share and cash dividends on the 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO) equal to $0.52344 per depositary share. The Company ended December 31, 2021, with cash and restricted cash of $95.4 million. After paying its remaining debt obligations in the second quarter of 2021, XOMA has no debt on its balance sheet. The Company continues to believe its current cash position will be sufficient to fund XOMA’s operations for multiple years.

INTRA-CELLULAR THERAPIES REPORTS THIRD QUARTER 2022 FINANCIAL RESULTS AND PROVIDES CORPORATE UPDATE

On November 3, 2022 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 third quarter ended September 30, 2022 and provided a corporate update (Press release, Intra-Cellular Therapies, NOV 3, 2022, View Source [SID1234623003]).

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"The successful launch of CAPLYTA continues with another quarter of strong revenue growth. In addition to our efforts to fuel continued commercial success, we are making investments to broaden CAPLYTA’s indications and advance our pipeline," said Dr. Sharon Mates, Chairman and CEO of Intra-Cellular Therapies.

THIRD QUARTER FINANCIAL HIGHLIGHTS

Total revenues were $71.9 million for the third quarter of 2022, compared to $22.2 million for the third quarter of 2021. Net product revenues of CAPLYTA were $71.9 million for the third quarter of 2022, compared to $21.6 million for the same period in 2021, representing a year-over-year increase of 233% and a 30% increase over the second quarter of 2022.

Cost of product sales were $5.9 million in the third quarter of 2022, compared to $2.0 million for the third quarter of 2021.

Selling, general and administrative (SG&A) expenses were $88.4 million for the third quarter of 2022, compared to $70.5 million for the third quarter of 2021. This increase is primarily due to an increase in marketing and advertising expenses and labor related costs.

Research and development (R&D) expenses for the third quarter of 2022 were $33.3 million, compared to $27.0 million for the third quarter of 2021. This increase is due to higher lumateperone clinical trial and non-clinical related costs and an increase in non-lumateperone program costs.

Net loss for the quarter ended September 30, 2022 was $53.5 million, compared to a net loss of $76.9 million for the quarter ended September 30, 2021.

Cash, cash equivalents, restricted cash and investment securities totaled $630.5 million at September 30, 2022, compared to $413.7 million at December 31, 2021. In January 2022, the Company completed a $460.0 million public offering resulting in net proceeds to the Company of approximately $433.7 million.

COMMERCIAL HIGHLIGHTS

Following its initial approval in schizophrenia in 2019, CAPLYTA received U.S. Food and Drug Administration (FDA) approval for bipolar depression in December 2021, becoming the first medicine approved in the U.S. for the treatment of depressive episodes associated with bipolar I or II disorder (bipolar depression) in adults as monotherapy and as adjunctive therapy with lithium or valproate.


CAPLYTA’s successful launch in bipolar depression continues. Third quarter CAPLYTA total prescriptions increased by 220% versus the third quarter of 2021. Third quarter CAPLYTA total prescriptions increased by 26% versus the second quarter of 2022.

In the third quarter we launched two new dosage strengths of CAPLYTA, 10.5 mg and 21 mg. These dosage strengths expand the patient population for CAPLYTA by providing dosage recommendations for patients taking strong or moderate CYP3A4 inhibitors and patients with moderate or severe hepatic impairment.

CAPLYTA maintained broad coverage in the Medicare Part D and Medicaid channels, with greater than 98% of lives covered and approximately 85% of lives covered in the commercial channel. Our LytaLink patient and prescriber support programs are highly effective at addressing coverage and reimbursement processes and overall patient affordability where appropriate.

Adjunctive MDD program: Studies 501 and 502 are global, double-blind placebo-controlled studies evaluating lumateperone 42 mg as adjunctive treatment to antidepressants. The primary endpoint is change from baseline on the Montgomery-Asberg Depression Rating scale (MADRS) total score at week 6. Additionally, Study 503 is an open label roll-over study to assess long-term safety in this patient population. Patient enrollment in these studies is ongoing and we expect to file a supplemental New Drug Application (sNDA) with the FDA for lumateperone as an adjunctive therapy to antidepressants for the treatment of major depressive disorder (MDD) in 2024.

Mixed Features program: We have completed patient enrollment in Study 403 and expect to report topline results in the first quarter of 2023. Study 403 is a global clinical trial evaluating lumateperone 42 mg in patients with MDD and in patients with bipolar depression who exhibit mixed features. Efficacy is assessed at week 6 and there is a follow-up safety visit two weeks after the last medication dose. The primary endpoint is change from baseline versus placebo on the MADRS total score at week 6, and the key secondary endpoint is the Clinical Global Impression (CGI-S) scale.

Lumateperone Long Acting Injectable (LAI) formulation: We have completed a Phase 1 single ascending dose study with our initial formulation. This study evaluated the pharmacokinetics, safety and tolerability of lumateperone LAI in patients with stable symptoms of schizophrenia. We have also explored alternate sites of injection with this formulation and have been progressing other formulations. We are completing our analyses of these studies which will enable us to define the next steps in this program. The goal of our program is to develop LAI formulations that are effective, safe and well-tolerated with treatment durations of one month and longer.

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 have completed a food intake study showing food had no significant impact on the pharmacokinetic (PK) profile. We have also completed Phase 1 safety studies demonstrating ITI-1284 is safe and generally well tolerated in normal healthy volunteers and normal healthy elderly volunteers. Other Phase 1 studies are ongoing or planned and include drug-drug interaction studies and mass balance studies. We also continue to progress our toxicology program for ITI-1284.

Phosphodiesterase type I inhibitor (PDE1) program: In our PDE 1 inhibitor program, for lenrispodun, we have recently completed or have ongoing Phase 1 trials including drug-drug interaction, bioavailability from scale up batches and food effect studies.

ITI-333 program: ITI-333 neuroimaging studies are ongoing. These studies are investigating brain occupancy for receptors related to substance use disorder and pain. Following these studies, we plan to initiate a multiple ascending dose study.

Presentations:

We presented at several medical conferences featuring CAPLYTA including the US Psych Congress and the European College of Neuropsychopharmacology (ECNP) Congress. In these conferences, we presented important data substantiating the favorable long-term safety profile of lumateperone in patients with bipolar depression, consistent with the long-term safety profile seen in our studies of lumateperone in patients with schizophrenia. We also presented analyses from our bipolar program including findings consistent with broad antidepressant effects, marked improvements in patients’ daily functioning, and further evidence of a favorable metabolic profile.

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 (https://register.vevent.com/register/BIc63e50ba892948f5932b126e73007d48). 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:

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.

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:

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

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.

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.

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.

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.

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.

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.

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

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

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.

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.

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.

Intellia Therapeutics Announces Third Quarter 2022 Financial Results and Highlights Recent Company Progress

On November 3, 2022 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 third quarter ended September 30, 2022 (Press release, Intellia, NOV 3, 2022, View Source [SID1234623002]).

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"During the most recent quarter, Intellia achieved significant clinical milestones across two landmark first-in-human studies," said Intellia President and Chief Executive Officer John Leonard, M.D. "We believe the latest data from our lead in vivo program, NTLA-2001, indicate it has the potential to set a new standard for how people living with ATTR amyloidosis are treated for this life-threatening disease. We are rapidly completing the dose-expansion portion of the study and are engaging with regulatory agencies, including in the U.S., as we move closer to a global, pivotal trial. Interim data from our second in vivo program, NTLA-2002, demonstrated robust reductions in both plasma kallikrein levels and the rate of swelling attacks in patients with hereditary angioedema. These initial data support the potential for a single dose of NTLA-2002 to permanently prevent the debilitating and potentially fatal HAE attacks that characterize this lifelong genetic disease. We look forward to presenting additional data from both NTLA-2001 and NTLA-2002 later this month."

Dr. Leonard continued, "Together, these interim datasets demonstrate the modularity of our CRISPR-based platform, providing powerful evidence of our ability to apply this technology broadly to address a wide range of diseases. Beyond gene knockout programs, we are advancing our first in vivo insertion candidate, NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency, with plans to submit a regulatory filing next year."

Third Quarter 2022 and Recent Operational Highlights

In Vivo Program Updates

Transthyretin (ATTR) Amyloidosis

NTLA-2001: NTLA-2001 is the first investigational CRISPR-based therapy to be systemically delivered to edit genes inside the human body and has the potential to be the first single-dose treatment for ATTR amyloidosis. Delivered with the Company’s in vivo lipid nanoparticle (LNP) technology, NTLA-2001 offers the possibility of halting and reversing the disease by driving a deep, potentially lifelong reduction in transthyretin (TTR) protein after a single dose. NTLA-2001 is being evaluated in a Phase 1, two-part, open-label study in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). NTLA-2001 is subject to a co-development/co-promotion agreement between Intellia, the lead party for this program, and Regeneron Pharmaceuticals, Inc.
ATTR-CM arm: Intellia announced in September positive interim results from the cardiomyopathy arm of the ongoing Phase 1 clinical trial of NTLA-2001. The interim data were from 12 adult patients with ATTR-CM with New York Heart Association (NYHA) Class I – III heart failure. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 were administered via intravenous infusion, and the change from baseline in serum TTR protein concentration was measured for each patient. These data showed deep and sustained mean serum TTR reductions of 93% and 92% at the 0.7 mg/kg and 1.0 mg/kg doses, respectively, at day 28. At both dose levels, NTLA-2001 was generally well-tolerated. Two of 12 patients reported transient infusion reactions, which was the only observed treatment-related adverse event. One patient in the 0.7 mg/kg dose NYHA Class III cohort experienced a Grade 3 infusion-related reaction, which resolved without clinical consequence. No clinically significant laboratory abnormalities were observed at either dose level. These data support NTLA-2001’s potential as a one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein in people with ATTR-CM.
Intellia initiated dosing at a 55 mg dose, the fixed dose corresponding to 0.7 mg/kg, in Part 2, the dose-expansion portion of the study. The Company remains on track to complete, by the end of this year, planned enrollment of both arms of the Phase 1 study that will inform the dose selection for subsequent pivotal studies.
Intellia will be presenting interim clinical data from the cardiomyopathy arm in a Late-Breaking Science session on November 5, 2022, at this year’s American Heart Association (AHA) Scientific Sessions, taking place in Chicago, Illinois.
Hereditary Angioedema (HAE)

NTLA-2002: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology 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, and 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 in September positive interim results from an ongoing Phase 1/2 clinical study of NTLA-2002, its second in vivo genome editing candidate, at the 2022 Bradykinin Symposium held in Berlin, Germany. Administration of single doses of NTLA-2002 led to dose-dependent reductions in plasma kallikrein, with mean reductions of 65% and 92% in the 25 mg (n=3) and 75 mg (n=3) dose cohorts, respectively, by week eight. In addition to plasma kallikrein levels, HAE attack rates are also being measured in the study, with the first analysis occurring at the end of the pre-specified 16-week primary observation period. A single dose of 25 mg of NTLA-2002 resulted in a 91% mean reduction in HAE attacks through the 16-week observation period. Additionally, two of the three patients have not had a single HAE attack since treatment, and all three patients have been attack-free since week 10 (based on follow-up through weeks 24 – 32). Patients in the 75 mg cohort had not completed the primary 16-week observation period. At both dose levels, NTLA-2002 was generally well-tolerated, and the majority of adverse events were mild in severity.
Intellia will be presenting additional data at the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place November 10 – 14 in Louisville, Kentucky. The new presentation is expected to include interim safety and kallikrein reduction data from the 50 mg dose cohort, and additional safety, kallikrein reduction and attack rate data from the 25 mg and 75 mg dose cohorts from the dose-escalation portion of the study.
Intellia expects to select up to two doses to further evaluate in the Phase 2, placebo-controlled, dose-expansion portion of the study, slated to begin in the first half of 2023. Intellia anticipates expanding country and site participation, including U.S. clinical sites, as part of the Phase 2 study.
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 IV infusions of A1AT augmentation therapy or lung transplant in severe cases.
Intellia is conducting Investigational New Drug (IND)-enabling activities for NTLA-3001, with plans to file an IND or IND-equivalent in 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.
Ex Vivo Program Updates

CD30+ Lymphomas

NTLA-6001: NTLA-6001 is an allogeneic CAR-T development candidate targeting CD30 for the treatment of CD30-expressing hematologic cancers, including relapsed or refractory classical Hodgkin lymphoma (cHL). NTLA-6001 is the first wholly owned candidate developed using Intellia’s proprietary allogeneic cell engineering platform.
Intellia is conducting IND-enabling activities for NTLA-6001.
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.
Intellia plans to advance at least one additional new in vivo development candidate by the end of 2022.
Allogeneic Platform: In October, at the European Society of Gene & Cell Therapy 29th Congress, Intellia highlighted its proprietary allogeneic solution to create engineered T cells with high anti-tumor activity, which have the potential to be uniquely capable of persisting in the patient to maintain durable responses. Notably, a novel combination of gene edits, including knockout of HLA Class II and HLA-A while retaining HLA-B and HLA-C proteins, yielded T cells capable of avoiding rejection by host T and natural killer (NK) cells in preclinical models. By only matching for HLA-B and HLA-C with homozygous donors for a 2/2 match, Intellia’s approach allows for a simplified HLA matching strategy between healthy donor T cells and recipient patients. Development of such an "off-the-shelf" therapy aims to address the majority of the patient population with only a small set of donors. Intellia’s allogeneic platform is being deployed for investigational TCR-T and CAR-T cell therapies.
Upcoming Events

The Company will participate in the following events during the fourth quarter of 2022:

American Heart Association Scientific Sessions 2022, November 5, Chicago
American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting, November 12, Louisville
Piper Sandler 34th Annual Healthcare Conference, November 29, New York
Upcoming Milestones

The Company has set forth the following for pipeline progression:

In Vivo

NTLA-2001 for ATTR amyloidosis:
Present additional interim data from ATTR-CM arm of Phase 1 study at AHA
Complete planned enrollment of Phase 1 study for both ATTRv-PN and ATTR-CM subjects by the end of this year
NTLA-2002 for HAE:
Present additional interim data from Phase 1/2 study at ACAAI
Initiate Phase 2 portion of the study in 1H 2023
NTLA-3001 for AATD: File an IND or IND-equivalent in 2023
Advance at least one additional new in vivo development candidate by the end of 2022
Third Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $848.7 million as of September 30, 2022, compared to $1.1 billion as of December 31, 2021. The decrease was driven by cash used to fund operations of approximately $276.2 million as well as the acquisition of Rewrite for $45.0 million. The decrease was offset in part by $62.1 million in net equity proceeds raised from the Company’s "At the Market" (ATM) agreement and $15.1 million in proceeds from employee-based stock plans. Subsequent to the end of the third quarter, through October 27, 2022, the Company increased its cash position by approximately $115 million. This included an additional $107.2 million in net equity proceeds raised from its ATM agreement and an additional $8.3 million received for ATM transactions with trade dates in September that were settled in October.
Collaboration Revenue: Collaboration revenue increased by approximately $6.1 million to $13.3 million during the third quarter of 2022, compared to $7.2 million during the third quarter of 2021. The increase was primarily driven by our collaborations with AvenCell and Kyverna.
R&D Expenses: Research and development expenses increased by $36.2 million to $96.7 million during the third quarter of 2022, compared to $60.5 million during the third quarter of 2021. This increase was primarily driven by the advancement of our lead programs and personnel growth to support these programs.
G&A Expenses: General and administrative expenses increased by $3.4 million to $22.1 million during the third quarter of 2022, compared to $18.7 million during the third quarter of 2021. This increase was primarily related to employee-related expenses, including stock-based compensation of $2.4 million.
Net Loss: The Company’s net loss was $113.2 million for the third quarter of 2022, compared to $71.6 million during the third quarter of 2021.
Conference Call to Discuss Third Quarter 2022 Results

The Company will discuss these results on a conference call today, Thursday, November 3, 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 November 3, at 12 p.m. ET.

X4 Pharmaceuticals Reports Third Quarter 2022 Financial Results and Provides Corporate Update

On November 3, 2022 X4 Pharmaceuticals Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with diseases of the immune system, reported financial results for the third quarter ended September 30, 2022 and highlighted recent and upcoming expected milestones (Press release, X4 Pharmaceuticals, NOV 3, 2022, View Source [SID1234623001]).

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"This is a truly exciting time for X4 as we near the announcement of top-line data from our first pivotal clinical trial and continue to advance our lead candidate, mavorixafor, towards commercialization," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Following our September release of positive data from the Phase 1b clinical trial of mavorixafor across a number of chronic neutropenic disorders, we now eagerly await the results from the 4WHIM registration trial. With no oral therapeutic approved for these patients, we believe mavorixafor, if approved, could represent a new opportunity to transform the treatment landscape and create a new standard of care for almost 50,000 underserved patients in the U.S."

Recent Highlights & Anticipated Upcoming Milestones
•In July, X4 completed a private investment in public equity (PIPE) financing, receiving aggregate gross proceeds of approximately $55 million; the financing included participation from new and existing investors.
•In July, the company announced a strategic re-prioritization of resources towards advancing mavorixafor in chronic neutropenic disorder indications, including WHIM syndrome, while progressing oncology programs only upon completion of strategic partnership(s); the announced strategic update was also inclusive of cost-cutting initiatives estimated to extend X4’s cash runway into the third quarter of 2023.
•In late September, X4 held an event highlighting new positive data from its Phase 1b clinical trial demonstrating the ability of mavorixafor to increase and normalize absolute neutrophil counts (ANC) in people with idiopathic, cyclic, or congenital chronic neutropenia (CN) as monotherapy or concurrently with injectable granulocyte colony-stimulating factor (G-CSF). The company believes that these results suggest an expanded market opportunity for mavorixafor that could include almost 50,000 additional diagnosed patients in the U.S.
•The Phase 1b trial in chronic neutropenic disorders is currently being amended and expanded into a Phase 1b/Phase 2 clinical trial to assess the long-term durability, safety, and tolerability of mavorixafor in a larger patient population; X4 anticipates the amended trial to begin generating additional clinical data in the first half of 2023.

Exhibit 99.1
•The company recently announced the appointment of industry veteran Mark Baldry to the position of Chief Commercial Officer. In this key role, Mr. Baldry will lead all pre-commercial and product launch efforts for mavorixafor as clinical development advances.
•Following recent completion of the last patient/last visit in the company’s Phase 3 clinical trial of mavorixafor in WHIM syndrome (the 4WHIM trial), X4 continues to anticipate the announcement of top-line results from the trial in the fourth quarter of 2022 and the submission for U.S. regulatory approval of mavorixafor in WHIM early in the second half of 2023, if the data are positive.

X4 today also announced that its Chief Medical Officer, Dr. Diego Cadavid, intends to depart the company by the end of 2022, but will continue to support X4 as a consultant. Dr. Cadavid, still a practicing neurologist, is leaving to pursue an opportunity that closely aligns with his commitment to patients with intractable CNS disorders.

X4 is appointing Dr. Murray Stewart as interim Chief Medical Officer while it conducts a search for a permanent replacement for Dr. Cadavid. Dr. Stewart, who has served as a member of the X4 Board of Directors since March 2019, has had a distinguished career in the life sciences industry, including an 18-year tenure at GlaxoSmithKline (GSK), where he held multiple research and development leadership roles, including Chief Medical Officer, Clinical Head of the Biopharma Unit, and Therapy Area Head for metabolic and cardiovascular diseases. Dr. Stewart also previously served as Chief Medical Officer of Rhythm Pharmaceuticals, leading the successful NDA submission and approval of ImcivreeTM (setmelanotide), an innovative treatment for rare causes of obesity.

Dr. Stewart commented: "I am very pleased to be able to support X4 through this exciting time, as we prepare to unblind the mavorixafor Phase 3 WHIM data and advance to an NDA submission if the data are positive. I look forward to partnering with the clinical and executive management teams to continue X4’s journey towards providing people with WHIM and other CN disorders with a potentially transformative new therapy."

Third Quarter 2022 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $81.1 million in cash, cash equivalents, and restricted cash as of September 30, 2022. X4 believes that it has sufficient funds to support company operations into the third quarter of 2023.
•Research and Development (R&D) Expenses were $14.1 million for the third quarter of 2022 as compared to $13.2 million for the comparable period in 2021. R&D expenses include $0.6 million and $0.6 million of certain non-cash expenses for the third quarter of 2022 and 2021, respectively.
•Selling, General, and Administrative Expenses (SG&A) were $6.0 million for the third quarter of 2022 as compared to $5.9 million for the comparable period in 2021. SG&A expenses include $0.5 million and $0.9 million of certain non-cash expenses for the third quarter of 2022 and 2021, respectively.
•Net Loss: X4 reported a net loss of $21.6 million for the third quarter of 2022, as compared to $20.2 million for the comparable period in 2021. Net losses include $1.1 million and $1.5 million of certain non-cash expenses for the third quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am ET to discuss important upcoming milestones, including the pending release of results from the company’s Phase 3 trial of its lead candidate, mavorixafor, in the treatment of WHIM syndrome. The conference call can be accessed by dialing 1-855-327-6837 within the United States or 1-631-891-4304 internationally, followed by the

Exhibit 99.1
conference ID: 10020371. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.