On August 4, 2022 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported financial results for the second quarter ended June 30, 2022 and provided a business update (Press release, Insmed, AUG 4, 2022, View Source [SID1234617580]).

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"Insmed finished the second quarter of 2022 in a stronger position than ever before, with significant sales growth for ARIKAYCE and steady progress across our clinical programs, which continue to track in line with expectations," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "We begin the second half of the year with meaningful commercial momentum and a strong financial position that we believe will support continued execution across our commercial business, clinical pipeline, and early-stage research. I am incredibly proud of our talented, patient-focused team and excited about the future of our organization as we prepare to serve significantly more patients with serious and rare diseases."

Recent Pillar Highlights
ARIKAYCE

In the second quarter of 2022, ARIKAYCE revenue grew 44% over the second quarter of 2021, reflecting strong growth in U.S. sales and ongoing launch activities in Japan.
Enrollment remains on track in the post-marketing confirmatory, frontline clinical trial program of ARIKAYCE in patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex (MAC), consisting of the ARISE and ENCORE trials. Insmed anticipates completing patient screening in ARISE by the end of August 2022, completing enrollment by the end of 2022, and sharing data from the trial over the course of 2023; the Company also anticipates completing enrollment in ENCORE by the end of 2023.
Brensocatib

Enrollment remains on track in the Phase 3 ASPEN study, a global, randomized, double-blind, placebo-controlled trial to assess the efficacy, safety, and tolerability of brensocatib in patients with bronchiectasis. Insmed continues to anticipate completing enrollment in this trial in the first quarter of 2023.
A Phase 2 pharmacokinetic/pharmacodynamic study of brensocatib in patients with cystic fibrosis (CF), which includes both patients who are on background CF transmembrane conductance regulator (CFTR) modulator drugs and patients who are not on CFTR modulator drugs, is underway. Enrollment is now complete in the CFTR modulator arm of the study, and Insmed anticipates having top-line data by end of 2022.
As previously shared, Insmed plans to develop brensocatib in two new potential indications – chronic rhinosinusitis without nasal polyps (CRSsNP) and hidradenitis suppurativa (HS). Insmed anticipates moving brensocatib into clinical development for CRSsNP by the middle of 2023, followed by HS.
TPIP

Insmed is advancing a Phase 2 study to assess the safety and tolerability of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) over a 16-week treatment period, as well as a Phase 2b study to evaluate the effect of TPIP on pulmonary vascular resistance (PVR) and 6-minute walk distance over a 16-week treatment period in patients with pulmonary arterial hypertension (PAH).
One patient with PAH has now completed the Phase 2a trial measuring the impact of TPIP on PVR over a 24-hour period. The patient also completed a 16-week extension period and was successfully titrated to a dose of 320 micrograms. No safety concerns were observed and Insmed identified a trend in improvement in various cardiac measures during the 24-hour period.
Translational Medicine

Insmed is advancing a translational medicine portfolio encompassing a wide range of technologies and modalities, including gene therapy, gene editing, protein deimmunization, and manufacturing capabilities. The Company anticipates filing one to two Investigational New Drug Applications per year from this portfolio.
Second Quarter 2022 Financial Results
Total revenue for the second quarter ended June 30, 2022, was $65.2 million, compared to total revenue of $45.4 million for the second quarter of 2021. Total revenue for the second quarter of 2022 comprised ARIKAYCE net sales of $47.2 million in the U.S., $15.8 million in Japan, and $2.2 million in Europe and rest of world.
Cost of product revenues (excluding amortization of intangible assets) was $16.4 million for the second quarter of 2022, compared to $10.8 million for the second quarter of 2021.
Research and development (R&D) expenses were $88.5 million for the second quarter of 2022, compared to $64.7 million for the second quarter of 2021.
Selling, general and administrative (SG&A) expenses for the second quarter of 2022 were $60.0 million, compared to $57.2 million for the second quarter of 2021.
For the second quarter of 2022, Insmed reported a net loss of $95.6 million, or $0.80 per share, compared to a net loss of $117.3 million, or $1.07 per share, for the second quarter of 2021.
Balance Sheet, Financial Guidance, and Planned Investments
As of June 30, 2022, Insmed had cash and cash equivalents and marketable securities of $564.6 million. The Company’s total operating expenses for the second quarter of 2022 were $153.5 million.

Insmed continues to expect full-year 2022 global revenues for ARIKAYCE to increase at least 30% year over year from 2021. The Company also continues to anticipate that its cash on hand will support its ongoing business into 2024.

The Company plans to continue to invest in the following key activities during the remainder of 2022:

(i)commercialization and expansion of ARIKAYCE globally;

(ii)advancement of brensocatib, including the Phase 3 ASPEN study in patients with bronchiectasis and commercial launch readiness activities;

(iii)advancement of the confirmatory, frontline clinical trial program for ARIKAYCE (ARISE and ENCORE); and

(iv)advancement of our earlier-stage pipeline, including the Phase 2 clinical development programs for TPIP and our translational medicine efforts.

Conference Call
Insmed will host a conference call beginning today at 8:30 AM Eastern Time. Shareholders and other interested parties may participate in the conference call by dialing (888) 210-2654 (U.S.) or (646) 960-0278 (international) and referencing access code 7862189. The call will also be webcast live on the company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately 2 hours after its completion through September 3, 2022, by dialing (800) 770-2030 (U.S.) or (647) 362-9199 (international) and referencing access code 7862189. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company’s website at www.insmed.com.

About ARIKAYCE
ARIKAYCE is approved in the United States as ARIKAYCE (amikacin liposome inhalation suspension), in Europe as ARIKAYCE Liposomal 590 mg Nebuliser Dispersion, and in Japan as ARIKAYCE inhalation 590 mg (amikacin sulfate inhalation drug product). Current international treatment guidelines recommend the use of ARIKAYCE for appropriate patients. ARIKAYCE is a novel, inhaled, once-daily formulation of amikacin, an established antibiotic that was historically administered intravenously and associated with severe toxicity to hearing, balance, and kidney function. Insmed’s proprietary PULMOVANCE liposomal technology enables the delivery of amikacin directly to the lungs, where liposomal amikacin is taken up by lung macrophages where the infection resides, while limiting systemic exposure. ARIKAYCE is administered once daily using the Lamira Nebulizer System manufactured by PARI Pharma GmbH (PARI).

About PARI Pharma and the Lamira Nebulizer System
ARIKAYCE is delivered by a novel inhalation device, the Lamira Nebulizer System, developed by PARI. Lamira is a quiet, portable nebulizer that enables efficient aerosolization of ARIKAYCE via a vibrating, perforated membrane. Based on PARI’s 100-year history working with aerosols, PARI is dedicated to advancing inhalation therapies by developing innovative delivery platforms to improve patient care.

About Brensocatib
Brensocatib is a small molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of patients with bronchiectasis and other neutrophil-mediated diseases. DPP1 is an enzyme responsible for activating neutrophil serine proteases (NSPs), such as neutrophil elastase, in neutrophils when they are formed in the bone marrow. Neutrophils are the most common type of white blood cell and play an essential role in pathogen destruction and inflammatory mediation. In chronic inflammatory lung diseases, neutrophils accumulate in the airways and result in excessive active NSPs that cause lung destruction and inflammation. Brensocatib may decrease the damaging effects of inflammatory diseases such as bronchiectasis by inhibiting DPP1 and its activation of NSPs. Brensocatib is an investigational drug product that has not been approved for any indication in any jurisdiction.

About TPIP
Treprostinil palmitil inhalation powder (TPIP) is a dry powder formulation of treprostinil palmitil, a treprostinil prodrug consisting of treprostinil linked by an ester bond to a 16-carbon chain. Developed entirely in Insmed’s laboratories, TPIP is a potentially highly differentiated prostanoid being evaluated for the treatment of patients with PAH, PH-ILD, and other rare and serious pulmonary disorders. TPIP is administered in a capsule-based inhalation device. TPIP is an investigational drug product that has not been approved for any indication in any jurisdiction.

IMPORTANT SAFETY INFORMATION FOR ARIKAYCE IN THE U.S.
WARNING: RISK OF INCREASED RESPIRATORY ADVERSE REACTIONS

ARIKAYCE has been associated with an increased risk of respiratory adverse reactions, including hypersensitivity pneumonitis, hemoptysis, bronchospasm, and exacerbation of underlying pulmonary disease that have led to hospitalizations in some cases.

Hypersensitivity Pneumonitis has been reported with the use of ARIKAYCE in the clinical trials. Hypersensitivity pneumonitis (reported as allergic alveolitis, pneumonitis, interstitial lung disease, allergic reaction to ARIKAYCE) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (3.1%) compared to patients treated with a background regimen alone (0%). Most patients with hypersensitivity pneumonitis discontinued treatment with ARIKAYCE and received treatment with corticosteroids. If hypersensitivity pneumonitis occurs, discontinue ARIKAYCE and manage patients as medically appropriate.

Hemoptysis has been reported with the use of ARIKAYCE in the clinical trials. Hemoptysis was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (17.9%) compared to patients treated with a background regimen alone (12.5%). If hemoptysis occurs, manage patients as medically appropriate.

Bronchospasm has been reported with the use of ARIKAYCE in the clinical trials. Bronchospasm (reported as asthma, bronchial hyperreactivity, bronchospasm, dyspnea, dyspnea exertional, prolonged expiration, throat tightness, wheezing) was reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (28.7%) compared to patients treated with a background regimen alone (10.7%). If bronchospasm occurs during the use of ARIKAYCE, treat patients as medically appropriate.

Exacerbations of underlying pulmonary disease has been reported with the use of ARIKAYCE in the clinical trials. Exacerbations of underlying pulmonary disease (reported as chronic obstructive pulmonary disease (COPD), infective exacerbation of COPD, infective exacerbation of bronchiectasis) have been reported at a higher frequency in patients treated with ARIKAYCE plus background regimen (14.8%) compared to patients treated with background regimen alone (9.8%). If exacerbations of underlying pulmonary disease occur during the use of ARIKAYCE, treat patients as medically appropriate.

Anaphylaxis and Hypersensitivity Reactions: Serious and potentially life-threatening hypersensitivity reactions, including anaphylaxis, have been reported in patients taking ARIKAYCE. Signs and symptoms include acute onset of skin and mucosal tissue hypersensitivity reactions (hives, itching, flushing, swollen lips/tongue/uvula), respiratory difficulty (shortness of breath, wheezing, stridor, cough), gastrointestinal symptoms (nausea, vomiting, diarrhea, crampy abdominal pain), and cardiovascular signs and symptoms of anaphylaxis (tachycardia, low blood pressure, syncope, incontinence, dizziness). Before therapy with ARIKAYCE is instituted, evaluate for previous hypersensitivity reactions to aminoglycosides. If anaphylaxis or a hypersensitivity reaction occurs, discontinue ARIKAYCE and institute appropriate supportive measures.

Ototoxicity has been reported with the use of ARIKAYCE in the clinical trials. Ototoxicity (including deafness, dizziness, presyncope, tinnitus, and vertigo) were reported with a higher frequency in patients treated with ARIKAYCE plus background regimen (17%) compared to patients treated with background regimen alone (9.8%). This was primarily driven by tinnitus (7.6% in ARIKAYCE plus background regimen vs 0.9% in the background regimen alone arm) and dizziness (6.3% in ARIKAYCE plus background regimen vs 2.7% in the background regimen alone arm). Closely monitor patients with known or suspected auditory or vestibular dysfunction during treatment with ARIKAYCE. If ototoxicity occurs, manage patients as medically appropriate, including potentially discontinuing ARIKAYCE.

Nephrotoxicity was observed during the clinical trials of ARIKAYCE in patients with MAC lung disease but not at a higher frequency than background regimen alone. Nephrotoxicity has been associated with the aminoglycosides. Close monitoring of patients with known or suspected renal dysfunction may be needed when prescribing ARIKAYCE.

Neuromuscular Blockade: Patients with neuromuscular disorders were not enrolled in ARIKAYCE clinical trials. Patients with known or suspected neuromuscular disorders, such as myasthenia gravis, should be closely monitored since aminoglycosides may aggravate muscle weakness by blocking the release of acetylcholine at neuromuscular junctions.

Embryo-Fetal Toxicity: Aminoglycosides can cause fetal harm when administered to a pregnant woman. Aminoglycosides, including ARIKAYCE, may be associated with total, irreversible, bilateral congenital deafness in pediatric patients exposed in utero. Patients who use ARIKAYCE during pregnancy, or become pregnant while taking ARIKAYCE should be apprised of the potential hazard to the fetus.

Contraindications: ARIKAYCE is contraindicated in patients with known hypersensitivity to any aminoglycoside.

Most Common Adverse Reactions: The most common adverse reactions in Trial 1 at an incidence ≥5% for patients using ARIKAYCE plus background regimen compared to patients treated with background regimen alone were dysphonia (47% vs 1%), cough (39% vs 17%), bronchospasm (29% vs 11%), hemoptysis (18% vs 13%), ototoxicity (17% vs 10%), upper airway irritation (17% vs 2%), musculoskeletal pain (17% vs 8%), fatigue and asthenia (16% vs 10%), exacerbation of underlying pulmonary disease (15% vs 10%), diarrhea (13% vs 5%), nausea (12% vs 4%), pneumonia (10% vs 8%), headache (10% vs 5%), pyrexia (7% vs 5%), vomiting (7% vs 4%), rash (6% vs 2%), decreased weight (6% vs 1%), change in sputum (5% vs 1%), and chest discomfort (5% vs 3%).

Drug Interactions: Avoid concomitant use of ARIKAYCE with medications associated with neurotoxicity, nephrotoxicity, and ototoxicity. Some diuretics can enhance aminoglycoside toxicity by altering aminoglycoside concentrations in serum and tissue. Avoid concomitant use of ARIKAYCE with ethacrynic acid, furosemide, urea, or intravenous mannitol.

Overdosage: Adverse reactions specifically associated with overdose of ARIKAYCE have not been identified. Acute toxicity should be treated with immediate withdrawal of ARIKAYCE, and baseline tests of renal function should be undertaken. Hemodialysis may be helpful in removing amikacin from the body. In all cases of suspected overdosage, physicians should contact the Regional Poison Control Center for information about effective treatment.

U.S. INDICATION
LIMITED POPULATION: ARIKAYCE is indicated in adults, who have limited or no alternative treatment options, for the treatment of Mycobacterium avium complex (MAC) lung disease as part of a combination antibacterial drug regimen in patients who do not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. As only limited clinical safety and effectiveness data for ARIKAYCE are currently available, reserve ARIKAYCE for use in adults who have limited or no alternative treatment options. This drug is indicated for use in a limited and specific population of patients.

This indication is approved under accelerated approval based on achieving sputum culture conversion (defined as 3 consecutive negative monthly sputum cultures) by Month 6. Clinical benefit has not yet been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Limitation of Use: ARIKAYCE has only been studied in patients with refractory MAC lung disease defined as patients who did not achieve negative sputum cultures after a minimum of 6 consecutive months of a multidrug background regimen therapy. The use of ARIKAYCE is not recommended for patients with non-refractory MAC lung disease.

Patients are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1‑800‑FDA‑1088. You can also call the Company at 1-844-4-INSMED.

On August 4, 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 second quarter ended June 30, 2022 (Press release, Intellia, AUG 4, 2022, View Source [SID1234617579]).

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"We continue to make excellent progress in both the cardiomyopathy and polyneuropathy arms of the landmark Phase 1 study of NTLA-2001," said Intellia President and Chief Executive Officer John Leonard, M.D. "In June, we presented durability data demonstrating deep reductions in a disease-causing protein were sustained over time following a single-dose treatment. Additionally, we’ve completed the dose-escalation portion of the cardiomyopathy arm and look forward to starting the dose-expansion portion soon."

Dr. Leonard continued, "As the leading full-spectrum genome editing company, our ex vivo capabilities and platform are also advancing. We believe our proprietary, differentiated cell engineering platform can solve many of the known challenges faced by both autologous and current allogeneic approaches. As a result, we plan to focus exclusively on developing allogeneic cell therapies, including an allogeneic version of NTLA-5001. Finally, we expect several important milestones later this year, which will include interim clinical data updates from the NTLA-2001 and NTLA-2002 programs."

Second 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.

To date, over 30 patients have been dosed across the polyneuropathy and cardiomyopathy arms. The growing body of data, particularly at therapeutically relevant doses, demonstrated treatment with NTLA-2001 resulted in rapid, deep and consistent reductions of serum TTR.
ATTR-CM arm: Intellia announced today the completion of the dose-escalation portion of the cardiomyopathy arm. The Company is finalizing selection of a fixed dose, at or near the fixed-dose equivalent of the 0.7 mg/kg dose, for evaluation in the dose-expansion portion of the study, subject to regulatory approval. The selection is based on clinical data from patients with ATTR-CM dosed at the 0.7 mg/kg and 1.0 mg/kg doses, which yielded similar TTR reductions. Additionally, both doses were generally well-tolerated. The Company remains on track to present interim data from the cardiomyopathy arm later this year.
ATTRv-PN arm: The Company announced today, subject to regulatory approval, plans to add a second cohort to the dose-expansion portion of the polyneuropathy arm, which will evaluate the same fixed dose selected for the dose-expansion portion of the cardiomyopathy arm. The decision to study a second dose is based on the following: (1) the emerging data from the dose-escalation portion of the cardiomyopathy arm showed similar serum TTR reduction at both the 0.7 mg/kg and 1.0 mg/kg doses, (2) the comparability of performance at the 0.7 mg/kg and 1.0 mg/kg doses in the dose-escalation portion of the polyneuropathy arm, which led to an 86% and 93% mean and 97% and 98% maximum TTR reduction at day 28, respectively, and (3) a significant elevation in liver enzymes, which normalized without medical intervention, observed at day 28 in a patient treated in the dose-expansion portion of the polyneuropathy arm at the 80 mg dose (the fixed dose corresponding to 1.0 mg/kg). While the adverse event is considered possibly related to study drug, this patient was asymptomatic, had no increase in bilirubin and the event was deemed nonserious by the investigator.
Intellia plans to submit a protocol amendment to evaluate a fixed dose corresponding to 0.7 mg/kg in the dose-expansion portion, with enrollment across both arms expected to be completed by the end of 2022, subject to regulatory feedback.
In June, Intellia presented updated interim data from its ongoing Phase 1 study of NTLA-2001 in patients with ATTRv-PN at the European Association for the Study of the Liver International Liver Congress 2022. Extended follow-up data from 15 ATTRv-PN patients, treated across all four single-ascending dose cohorts, showed deep, dose-dependent reductions in serum TTR observed with prior readouts were sustained through the last measured timepoint of follow-up, reaching 12 months in the 0.1 mg/kg and 0.3 mg/kg cohorts and six months in the 0.7 mg/kg and 1.0 mg/kg cohorts. Both 0.7 mg/kg and 1.0 mg/kg doses led to greater than 85% mean TTR reduction at day 28. The durability and persistence of effect continue to support NTLA-2001 as a potential one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein.
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 is progressing the single-ascending dose portion of its first-in-human study. The Company anticipates presenting interim data in the second half of 2022, including safety, kallikrein reduction and HAE attack rate data. These initial results are expected to characterize the emerging safety and activity profile of NTLA-2002 and potentially demonstrate the modularity of Intellia’s proprietary CRISPR-based, LNP platform.
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

Acute Myeloid Leukemia (AML)

NTLA-5001: NTLA-5001 is an investigational autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor 1 (WT1) antigen for the treatment of all genetic subtypes of AML. In March, Intellia announced that the first patient was dosed in the Phase 1/2a first-in-human trial of NTLA-5001.

Intellia has decided to concentrate its ex vivo development efforts exclusively on allogeneic cell therapies manufactured from healthy donors. The Company’s proprietary technologies, including its LNP-based cell engineering platform and novel allogeneic solution, offer significant advantages over both autologous and current investigational allogeneic approaches. Preclinical data presented on its differentiated allogeneic engineering platform showed allogeneic T cells were shielded from immune rejection, both host T and natural killer (NK) cell attack.
Intellia announced today plans to discontinue its first-in-human study of NTLA-5001, an investigational autologous TCR-T cell therapy, and is pivoting to an allogeneic version of this program currently in preclinical development. This decision is not due to any safety or efficacy data emerging from the trial. It is instead based on the potential of Intellia’s allogeneic platform to consistently deliver a high-quality, readily available and persistent cell product for treatment of aggressive cancers.
Preclinical data supporting the development of a WT1-directed allogeneic TCR-T cell candidate will be presented at a future scientific conference in 2022.
CD30+ Lymphomas

NTLA-6001: NTLA-6001 is a wholly owned, 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 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.
The Company plans to highlight additional advances to its proprietary technology capabilities, including both genome editing and delivery tools, at upcoming scientific conferences in 2022.
Upcoming Milestones

The Company has set forth the following for pipeline progression:

In Vivo

NTLA-2001 for ATTR amyloidosis:

Present interim data from ATTR-CM arm of Phase 1 study in 2H 2022
Complete enrollment of Phase 1 study for both ATTRv-PN and ATTR-CM subjects by the end of 2022
NTLA-2002 for HAE: Present interim data from Phase 1/2 study in 2H 2022
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
Ex Vivo

Present on its proprietary allogeneic cell engineering platform at an upcoming scientific conference in 2022
Modular Platform

Advance additional novel platform capabilities in 2022
Second Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $906.9 million as of June 30, 2022, compared to $1.1 billion as of December 31, 2021. The decrease was driven by cash used to fund operations of approximately $191.2 million as well as the acquisition of Rewrite for $45.0 million. The decrease was offset in part by $38.9 million in net equity proceeds raised from the Company’s "At the Market" (ATM) agreement and $14.3 million in proceeds from employee-based stock plans.
Collaboration Revenue: Collaboration revenue increased by approximately $7.5 million to $14.0 million during the second quarter of 2022, compared to $6.6 million during the second quarter of 2021. The increase was primarily driven by our collaborations with AvenCell and Kyverna.
R&D Expenses: Research and development expenses increased by $31.3 million to $90.2 million during the second quarter of 2022, compared to $58.9 million during the second quarter of 2021. This increase was primarily driven by the advancement of our lead programs, research personnel growth to support these programs and expansion of the development organization.
G&A Expenses: General and administrative expenses increased by $5.4 million to $22.1 million during the second quarter of 2022, compared to $16.7 million during the second quarter of 2021. This increase was primarily related to employee-related expenses, including stock-based compensation of $4.5 million.
Net Loss: The Company’s net loss was $100.7 million for the second quarter of 2022, compared to $68.8 million during the second quarter of 2021.
Conference Call to Discuss Second Quarter 2022 Results

The Company will discuss these results on a conference call today, Thursday, August 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 August 4, at 12 p.m. ET.

On August 4, 2022 BioVaxys Technology Corp. (CSE: BIOV) (FRA: 5LB) (OTCQB: BVAXF) ("BioVaxys" or the "Company") reported a non-brokered private placement (the "Private Placement") consisting of up to 10,000,000 units ("Units") at a price of $0.10 per Unit for total gross proceeds of up to approximately $1,000,000 (Press release, BioVaxys Technology, AUG 4, 2022, View Source [SID1234617578]). Each Unit consists of one common share (a "Common Share") and one whole Common Share purchase warrant (a "Warrant") . Each Warrant is exercisable for one additional Common Share at an exercise price of $0.20 for a period of 48 months.

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All securities issued pursuant to the Private Placement are subject to a statutory hold period of four months and one day from the date of issuance. The Company may pay cash finder’s fees on all or a portion of the Private Placement.

The Company intends to use the net proceeds of the Private Placement for working capital purposes. Closing of the Private Placement is subject customary conditions of closing, including the approval of the Canadian Securities Exchange, and is expected to complete on or about August 19th, 2022.

On August 4, 2022 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported financial results for its second quarter ended June 30, 2022 and provided an update on business performance (Press release, Myriad Genetics, AUG 4, 2022, View Source [SID1234617577]).

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"We are pleased with our progress this quarter as we advance our strategic operating plan to drive long term growth and profitability. Our team continues to execute on a disciplined approach to growing our business while managing gross margins and operating expenses despite significant wage and supply chain inflationary pressures. We are particularly excited about the progress of our Mental Health business, as Myriad’s GeneSight Psychotropic test quarterly volumes grew 39% year-over-year and 13% sequentially. We believe this momentum can be sustained as providers and payers digest the positive results of the PRIME Care study where both co-primary endpoints were achieved and remission rates for major depressive disorders showed significant improvement when clinicians had access to GeneSight test results," said Paul J. Diaz, president and CEO, Myriad Genetics.

Mr. Diaz went on to comment, "We also see continued stabilization and opportunities in the marketplace to grow our hereditary cancer and prenatal businesses and are excited about the growth we continue to see in our companion diagnostic business and our prostate cancer prognostic test – Prolaris. I want to thank the entire Myriad Genetics team and our provider partners for their continued efforts to serve our patients and execute on our transformation plan to enhance our product offerings, the experience of our customers and access for our patients."

Financial and Operational Highlights:

Diagnostic test volumes of 260,000 in the second quarter of 2022 increased 9% year-over-year and 7% sequentially from the first quarter of 2022, excluding divested businesses. During the second quarter of 2022, the company’s hereditary cancer volumes stabilized, down 4% year-over-year as compared to the first quarter of 2022 when volumes were down 12% year-over-year.
The following table summarizes sequential and year-over-year quarterly volume changes in the company’s core product categories:

Overall, average selling price (ASP)1 in the second quarter of 2022 decreased 2% year-over-year and increased 2% sequentially, excluding divested businesses.
Total revenue in the second quarter of 2022 was $179.3 million, an increase of 7% year-over-year and 9% sequentially, excluding the divested business revenue from Myriad RBM, Autoimmune and myPath Melanoma. Total revenue was negatively impacted by approximately $2.0 million in the quarter and $3.0 million year-to-date as a result of fluctuations in foreign exchange rates compared to the the prior year. Excluding divestitures, revenue for the six months ended June 30, 2022, increased 9% to $343.9 million compared to revenue for the six months ended June 30, 2021, which is in line with the company’s long term revenue guidance of 9 to 12%.
The following table summarizes year-over-year quarterly revenue changes in the company’s core businesses by product category:
GAAP gross margin in the second quarter of 2022 was 72.3%; adjusted gross margin in the quarter was 72.4%, which improved 30 basis points year-over-year.
GAAP total operating expenses in the second quarter of 2022 were $147.4 million, decreasing $9.1 million year-over-year; adjusted operating expenses in the quarter increased $2.2 million year-over-year to $125.3 million.
GAAP operating loss in the second quarter of 2022 was $17.8 million, improving $3.0 million year-over-year; adjusted operating income was $4.5 million, down $9.0 million year-over-year from $13.5 million.
Diluted GAAP EPS in the second quarter of 2022 were $(0.18), decreasing $0.12 year-over-year; adjusted EPS were $0.04, decreasing $0.08 year-over-year from $0.12.
Ended the second quarter of 2022 with $283.6 million in cash, cash equivalents and investments as compared to $339.2 million at the beginning of the quarter. The decrease of $55.6 million was driven primarily by a $48.0 million payment in connection with the settlement of the qui tam lawsuit filed in 2016. The company ended the quarter with no debt outstanding.
Business Performance and Highlights:

Oncology
The Myriad Genetics Oncology business provides hereditary cancer testing, including the MyRisk hereditary cancer test for patients who have cancer. It also provides tumor profiling products such as the myChoice CDx companion diagnostic test, the Prolaris prostate cancer test, and the EndoPredict breast cancer prognostic test. The Oncology business delivered revenue of $76.1 million in the second quarter of 2022, a decrease of 3% year-over-year and an increase of 9% sequentially from the first quarter of 2022.

Myriad Genetics recently launched Precise Oncology Solutions, which combines the company’s MyRisk germline cancer testing technology and its myChoice CDx companion diagnostic test with Precise Tumor, a tumor profiling test powered by Illumina, Inc.’s TruSight Oncology 500 (TSO500) assay and processed by Intermountain Precision Genomics.
MyChoice CDx reported its highest quarterly volume level in the United States ever in the second quarter of 2022 with quarterly volumes up 63% year-over-year and 10% sequentially from the first quarter of 2022.
Prolaris is a prostate cancer prognostic test designed to assess prostate cancer aggressiveness. The Oncology business achieved its highest quarterly Prolaris volumes in the second quarter of 2022, beating its previous quarterly volume record by 6%.
Women’s Health
The Myriad Genetics Women’s Health business serves women of all ancestries by assessing their risk of cancer and offers prenatal testing solutions for those who are pregnant or planning a family. The Women’s Health business delivered revenue of $70.1 million in the second quarter of 2022, an increase of 4% year-over-year and 7% sequentially from the first quarter of 2022.

Hereditary Cancer
Myriad Genetics continues to address the health inequities and accessibility challenges that exist within the hereditary cancer testing market. Myriad Genetics’ MyRisk hereditary cancer test with RiskScore for all ancestries offers the first and only personalized 5-year and lifetime breast cancer risk assessment for all women, including those of non-European ancestry. RiskScore is available at no additional cost to women who take the MyRisk test.
Prenatal
Myriad Genetics offers its Prequel noninvasive prenatal screen (NIPS), including proprietary AMPLIFY technology, which significantly enhances the test’s performance and works to reduce test failure rates so that patients may avoid unnecessary invasive procedures.
The company also offers its Foresight carrier screen to provide critical genetic insights for patients of all ethnicities.
Mental Health
The Myriad Genetics Mental Health business consists of the GeneSight psychotropic test that covers 64 medications commonly prescribed for depression, anxiety, attention deficit hyperactivity disorder, and other psychiatric conditions. GeneSight helps physicians understand how genetic alterations impact patient response to antidepressants and other drugs. In the pharmacogenomics category, GeneSight recorded revenue of $33.1 million in the second quarter of 2022, an increase of 46% year-over-year and 13% sequentially from the first quarter of 2022.

For the second quarter of 2022, the Mental Health business achieved its highest quarterly GeneSight volumes, beating its previous quarterly volume record by more than 10%.
In July of 2022, the Journal of the American Medical Association published results from the PRIME Care study — the largest pharmacogenomics randomized controlled trial ever conducted in mental health. The study, funded and conducted by the U.S. Department of Veterans Affairs, achieved both of its co-primary endpoints and found that major depressive disorder remission rates were significantly improved when clinicians had access to GeneSight Psychotropic test results. The PRIME Care study further reinforces the body of evidence supporting the clinical utility of pharmacogenomics testing and the GeneSight test as important tools for medication selection in the treatment of major depressive disorder and other mental illnesses.
Key Accomplishments in the Quarter
Myriad Genetics saw additional developments in the second quarter of 2022 including:

Myriad Genetics announced a partnership with Epic Systems Corporation (Epic), the industry leading healthcare software company, to integrate the company’s full line of genetic testing solutions with Epic’s expansive network of 600,000 physicians and more than 250 million patients. We believe the partnership bolsters Myriad Genetics’ growth plans of scaling customer-centric, tech-enabled commercial capabilities with over 600 electronic health record integrations this year.
The company presented multiple studies and showcased Precise Oncology Solutions at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting, demonstrating the value of genetic testing in guiding and clarifying cancer treatment and risk assessment.
Myriad Genetics was selected to participate in United Healthcare’s Preferred Laboratory Network (PLN), effective July 1, 2022. The PLN consists of currently contracted independent, freestanding laboratory care providers that have met higher standards for access, cost, data, quality and service, based on a rigorous application and review process.
The company announced an expansion of its strategic partnership with Intermountain Precision Genomics to add a new liquid biopsy therapy selection test to its growing oncology portfolio. The liquid biopsy test will use Illumina’s TSO500 ctDNA assay and be processed by Intermountain Precision Genomics. The new Precise liquid biopsy offering is expected to be available in 2023.
Myriad Genetics released its first environmental, social and governance report outlining actions taken to expand access to genetic testing, advance social justice, and increase environmental sustainability.
Myriad Genetics’ fiscal year 2022 non-GAAP guidance begins with the comparable GAAP financial measure and excludes the estimated impact of stock-based compensation expense of approximately $40.0 million, non-cash amortization associated with acquisitions of approximately $40.0 million and special items such as costs related to transformation initiatives of approximately $15.0 million. These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release.

The company reiterates its fiscal year 2022 revenue and gross margin guidance and updates its guidance for total operating expense and earnings per share. The company is increasing its fiscal year 2022 operating expense guidance by approximately $20.0 million. As a result of current market conditions, the company plans to make opportunistic incremental investments in research and development, technology and sales and marketing programs to access available market share and support its growth initiatives. Inclusive of these additional investments, Myriad’s updated fiscal year 2022 total operating expense guidance reflects a 6% — 8% year-over-year increase from fiscal year 2021 total operating expense, excluding divested businesses. This increase remains in line with current inflationary trends and demonstrates strong cost management even as the company invests in additional commercial and innovation initiatives.
Myriad Genetics will host its 2022 Investor Day at the NASDAQ MarketSite in New York City on August 11, 2022. Investor Day presentations and Q&A will take place from 10:00 a.m. to 12:00 p.m. EDT. For those unable to attend in person, a webcast will be available at the investor site on www.myriad.com.

Conference Call and Webcast
A conference call will be held today, Thursday, August 4, 2022, at 4:30 p.m. EDT to discuss Myriad Genetics’ financial results and business developments for the second quarter 2022. The dial-in number for domestic callers is 1-800-918-9482. International callers may dial 1-212-231-2929. All callers will be asked to reference reservation number 22019815. An archived replay of the call will be available for seven days by dialing 1-800-633-8284 and entering the reservation number above. The conference call and slide presentation will be available through a live webcast at www.myriad.com.

On August 4, 2022 Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming platform, reported financial results for the second quarter ended June 30, 2022, and highlighted recent Company progress (Press release, Omega Therapeutics, AUG 4, 2022, View Source [SID1234617576]).

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"This has been an exciting second quarter for Omega, in which we were thrilled to receive FDA clearance of our first IND application for OTX-2002, representing the first ever Omega Epigenomic ControllerTM, a new class of programmable mRNA therapeutics. This is a critical milestone for Omega as we enter our next phase of growth and reflects our pioneering work to realize the potential of epigenomic programming," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "Additionally, we were also pleased to share exciting, new supportive preclinical data, both from our lead program OTX-2002 in hepatocellular carcinoma, as well as from another program in our pipeline focused on non-small cell lung cancer, a potential future indication. We look forward to continuing this momentum as we enter the clinic in the second half of this year and further exploring the broad ranging capabilities of our novel platform in additional therapeutic areas."

Recent Business Highlights

Development Pipeline and Platform

Received FDA Clearance of Investigational New Drug (IND) application for OTX-2002, the First Omega Epigenomic ControllerTM (OEC), for MYC driven Hepatocellular Carcinoma (HCC): OTX-2002 is a novel, engineered, and programmable mRNA therapeutic designed to downregulate c-Myc (MYC) expression pre-transcriptionally through epigenetic modulation while potentially overcoming MYC autoregulation. This represents the first ever epigenomic controller, a new class of programmable mRNA therapeutics, to receive IND clearance.
On Track to Launch a Phase 1/2 Clinical Trial Under the MYCHELANGELOTM Clinical Trial Program in HCC patients in the 2H’22: The study consists of Part 1 (OTX-2002 as monotherapy) and Part 2 (OTX-2002 combined with standards of care in HCC). The Phase 1/2 trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a monotherapy and in combination with standard of care therapies in patients with relapsed or refractory HCC and other solid tumor types known for association with the MYC oncogene. The study is expected to enroll approximately 190 patients at clinical trial sites in the United States, Asia, and Europe and the Company expects to dose the first patient during the fourth quarter of 2022.
Part 1: This dose escalation portion of the trial will evaluate OTX-2002 monotherapy in patients with relapsed or refractory HCC and other solid tumors. Investigators will utilize a 3+3 design to identify the following primary endpoints of any dose limiting toxicities (DLTs), maximum tolerated dose (MTD), incidence of treatment emergent adverse events (TEAEs), and the recommended dose for expansion (RDE). There will then be a monotherapy expansion for patients with relapsed or refractory HCC who have received at least 1 prior line of systemic anticancer treatment and are without available subsequent standards of care. These patients will receive the RDE of OTX-2002. The primary endpoints of the monotherapy expansion will be overall response rate (ORR) and duration of response (DOR).
Part 2: This portion of the trial will evaluate OTX-2002 in combination with standard of care therapies and will consist of safety run-in and expansion cohorts. During the safety run-in, patients with relapsed or refractory HCC will receive OTX-2002 at the selected dose in combination with one of three current standard of care therapies. These combination partners will be one of two types of tyrosine kinase inhibitors (TKIs) or an immune checkpoint inhibitor (ICI). The primary endpoints of the safety run-in will be DLT, MTD, and incidence of TEAEs. Once the combination therapies have been determined to be tolerable in the safety run-in, patients with relapsed or refractory HCC will be enrolled into the expansion cohorts for each of the combination therapies, with the primary endpoints of ORR and DOR.
New OTX-2002 Preclinical Data Presented at Three Major Medical Meetings Show Robust In Vivo Efficacy and In Vitro Loss of Cancer Cell Viability:
Non-Human Primate (NHP) Data for OTX-2002 in HCC Presented at ESMO (Free ESMO Whitepaper)-GI 2022: Results showed that treatment with OTX-2002 resulted in robust in vivo efficacy in xenograft tumor models and successfully achieved pre-transcriptional downregulation of hepatocyte MYC gene expression in NHPs. These data support the clinical potential of OTX-2002 in combination with existing standard of care therapies for HCC, including ICIs. The ESMO (Free ESMO Whitepaper)-GI poster presentation is available here.
In Vivo Data for OTX-2002 in HCC Presented at AACR (Free AACR Whitepaper) 2022: Results demonstrated that OTX-2002 suppresses MYC gene expression resulting in a loss of cancer cell viability in vitro and a reduction in tumor growth in in vivo xenograft models. These data support the potential of Omega’s platform to engineer programmable mRNA therapeutics that successfully regulate gene expression. The AACR (Free AACR Whitepaper) poster presentation is available here.
In Vivo OEC Data in Models of Non-Small Cell Lung Cancer (NSCLC) Presented at ASGCT (Free ASGCT Whitepaper) 2022: Results demonstrated that regulation of MYC gene expression via epigenomic programming resulted in decreased viability of cancer cells in vitro and reduced tumor burden in in vivo xenograft models. These data support the potential of OECs to precisely and durably regulate gene expression and their potential as a future treatment for NSCLC. The ASGCT (Free ASGCT Whitepaper) poster presentation is available here.
Additional OEC Development: Beyond HCC and NSCLC, the Company is advancing multiple programs through preclinical studies spanning oncology, multigenic diseases including immunology, regenerative medicine, and select monogenic diseases.
OMEGA Epigenomic ProgrammingTM Platform: Omega is creating a new generation of programmable mRNA medicines that are designed to control the fundamental epigenetic processes to correct the root cause of disease by restoring aberrant gene expression to normal levels without altering native nucleic acid sequences. Omega has developed a highly rational and deterministic approach to drug design that enables the Company to rapidly develop and optimize novel OECs with high target specificity to durably tune the expression of single or multiple genes. Omega is advancing multiple preclinical development programs spanning oncology, multigenic diseases including immunology, regenerative medicine, and select monogenic diseases.
Corporate

Joshua Reed Appointed Chief Financial Officer. Mr. Reed brings over 25 years of successful and diverse corporate and financial operations experience including capital raising, business development, and investor relations.
Second Quarter 2022 Financial Results

As of June 30, 2022, the Company had cash, cash equivalents and marketable securities totaling $173.7 million.

Research and development (R&D) expenses for the second quarter of 2022 were $19.4 million, compared to $11.2 million for the second quarter of 2021. The $8.2 million increase in R&D expense was primarily driven by an increase in personnel-related expenses, external manufacturing costs, and study costs in support of the advancement of our programs

General and administrative (G&A) expenses for the second quarter of 2022 were $6.2 million, compared to $3.6 million for the second quarter of 2021. The $2.6 million increase in G&A expense was primarily driven by an increase in personnel-related expenses to support business growth.

Net loss for the second quarter of 2022 was $25.9 million, compared to $15.4 million for the second quarter of 2021, driven predominantly by increased R&D and G&A expenses to support the Company’s growth and operations as a public company.