On May 5, 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 first quarter ended March 31, 2022 and provided a business update (Press release, Insmed, MAY 5, 2022, View Source [SID1234613617]).

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"I am proud of Insmed’s solid progress in the first quarter of 2022, with steady ARIKAYCE performance in three territories around the globe, advancement of seven clinical trials, including our ASPEN and ARISE/ENCORE trials, and continued development of our early-stage research programs," commented Will Lewis, Chair and Chief Executive Officer of Insmed. "We ended the quarter in a position of financial strength, with a strong balance sheet and steady revenue stream. The remainder of 2022 will be a critical execution period across the four pillars of our business, and I am confident we have the ambition, determination, and talent to achieve our objectives and deliver high-impact medicines for patients in need."

Recent Pillar Highlights

ARIKAYCE

In the first quarter of 2022, ARIKAYCE revenue grew 32% over the first quarter of 2021, reflecting steady U.S. performance and ongoing launch activities in Japan and Europe.
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 enrollment in ARISE in 2022 and having topline data in the first half of 2023; the Company also anticipates completing enrollment in ENCORE by the end of 2023.
Insmed announced that it will present data at the American Thoracic Society (ATS) 2022 International Conference, taking place May 13-18, 2022, highlighting the reduction in hospitalizations after initiation of ARIKAYCE treatment in a retrospective cohort study of patients in real-world settings.
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 early 2023.
A Phase 2 pharmacokinetic/pharmacodynamic study of brensocatib in patients with cystic fibrosis is underway and Insmed continues to anticipate sharing data by early 2023.
As previously shared, Insmed plans to develop brensocatib in two new potential indications – chronic rhinosinusitis without nasal polyps and hidradenitis suppurativa.
Insmed will present data at the ATS 2022 International Conference evaluating the benefit-risk profile in a post-hoc analysis of the Phase 2 WILLOW study of brensocatib in patients with bronchiectasis.
TPIP

Insmed remains on track to share preliminary data from a small number of patients in a Phase 2a trial of treprostinil palmitil inhalation powder (TPIP) in patients with pulmonary arterial hypertension (PAH) this year. The Phase 2a study will measure the impact of TPIP on pulmonary vascular resistance (PVR) over a 24-hour period.
Insmed is also advancing a Phase 2b study to evaluate the effect of TPIP on PVR and 6-minute walk distance over a 16-week treatment period in patients with PAH, and a Phase 2 study to assess the safety and tolerability of TPIP in patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD) over a 16-week treatment 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 at least one Investigational New Drug Application per year from this portfolio.
First Quarter 2022 Financial Results

Total revenue for the first quarter ended March 31, 2022, was $53.1 million, compared to total revenue of $40.2 million for the first quarter of 2021. Total revenue for the first quarter of 2022 comprised ARIKAYCE net sales of $40.8 million in the U.S., $10.7 million in Japan, and $1.6 million in Europe and rest of world.
Cost of product revenues (excluding amortization of intangible assets) was $12.2 million for the first quarter of 2022, compared to $9.8 million for the first quarter of 2021.
Research and development (R&D) expenses were $84.4 million for the first quarter of 2022, compared to $61.4 million for the first quarter of 2021.
Selling, general and administrative (SG&A) expenses for the first quarter of 2022 were $56.7 million, compared to $51.6 million for the first quarter of 2021.
For the first quarter of 2022, Insmed reported a net loss of $94.6 million, or $0.80 per share, compared to a net loss of $91.6 million, or $0.89 per share, for the first quarter of 2021.
Balance Sheet, Financial Guidance, and Planned Investments

As of March 31, 2022, Insmed had cash and cash equivalents and marketable securities of $664.7 million. The Company’s total operating expenses for the first quarter of 2022 were $142.9 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 in 2022:

(i)

commercialization and expansion of ARIKAYCE globally;

(ii)

launch activities for ARIKAYCE in Japan; and

(iii)

clinical trial activities, including (a) advancement of the confirmatory, frontline clinical trial program for ARIKAYCE (ARISE and ENCORE), (b) advancement of brensocatib, including the Phase 3 ASPEN study in patients with bronchiectasis, (c) advancement of the Phase 2 clinical development programs for TPIP, and (d) advancement of 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 (844) 200-6205 (U.S. toll free), (646) 904-5544 (U.S. local), or +1-929-526-1599 (international) and referencing access code 388457. 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 1 hour after its completion through June 4, 2022, by dialing (866) 813-9403 (U.S. toll free), (929) 458-6194 (U.S. local), or +44-204-525-0658 (international) and referencing access code 252664. 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 May 5, 2022 Elevation Oncology, Inc. (Nasdaq: ELEV), a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, reported financial results for the quarter ended March 31, 2022, and highlighted recent progress (Press release, Elevation Oncology, MAY 5, 2022, View Source [SID1234613615]).

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"CRESTONE is an important study for both Elevation Oncology and for patients with NRG1 fusions, a genomically defined patient population with no approved therapies addressing the unmet medical need. We are honored for the opportunity to share the initial and first ever clinical data of seribantumab in this patient population through an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting in June 2022." said Shawn M. Leland, PharmD, RPh, Founder and Chief Executive Officer of Elevation Oncology. "In addition to executing on CRESTONE, we are building an industry-leading team, led by people like Dr. David Dornan, our newly appointed Chief Scientific Officer, who brings a wealth of cancer drug development experience to advance our mission of maximizing the potential of seribantumab and building out an industry-leading precision oncology pipeline. Together with our diagnostic and academic partners, we look forward to advancing the field of precision oncology by expanding upon our operational platform and continuing to make genomic testing results therapeutically actionable."

Recent Progress and Highlights

Seribantumab

●Initial CRESTONE data selected for oral presentation at ASCO (Free ASCO Whitepaper) 2022. ASCO (Free ASCO Whitepaper) abstracts to be published on May 26, 2022 at 5:00PM ET. The presentation, scheduled for June 7, 2022, from 9:45AM-12:45PM CT, will highlight initial clinical data from approximately 10 patients from Cohort 1 in the ongoing Phase 2 CRESTONE study evaluating seribantumab at 3 grams weekly in patients with solid tumors harboring NRG1 gene fusions.

Corporate

●David Dornan, PhD appointed Chief Scientific Officer. In March, Elevation Oncology announced the appointment of Dr. Dornan, PhD, as the Company’s first Chief Scientific Officer, bringing deep oncology R&D and drug development expertise and further strengthening the leadership team.
Expected Upcoming Milestones and Operational Objectives

●Initial Phase 2 CRESTONE data to be presented in an oral presentation at ASCO (Free ASCO Whitepaper) 2022.
●Complete enrollment of the first 20 patients in Cohort 1 of the CRESTONE study in mid-2022.
●Ongoing target evaluation and continued execution of our strategy for future pipeline expansion.
First Quarter 2022 Financial Results

As of March 31, 2022, the Company had cash, cash equivalents and marketable securities totaling $132.1 million, compared to $146.3 million as of December 31, 2021.

Research and development expenses for the first quarter 2022 were $13.6 million, compared to $4.1 million for the first quarter 2021. The increase in R&D expense was primarily related to an increase in manufacturing, personnel costs and other expenses associated with the CRESTONE study.

General and administrative expenses for the first quarter 2022 were $3.8 million, compared to $1.0 million for the first quarter 2021. The increase in G&A expense was primarily related to personnel costs, professional services and other administrative costs.

Net loss for the first quarter 2022 was $17.3 million, compared to $5.1 million for the first quarter 2021.

Financial Outlook

Elevation Oncology expects its existing cash, cash equivalents and marketable securities as of March 31, 2022 will be sufficient to fund its current operations into the fourth quarter of 2023.

About Seribantumab and NRG1 Gene Fusions

Seribantumab is a fully human IgG2 monoclonal antibody that binds to human epidermal growth factor receptor 3 (HER3). HER3 is traditionally activated through binding of its primary ligand, neuregulin-1 (NRG1). The NRG1 gene fusion is a rare genomic alteration that combines NRG1 with another partner protein to create chimeric NRG1 "fusion proteins." The NRG1 fusion protein is often also able to activate the HER3 pathway, leading to unregulated cell growth and proliferation. Importantly, NRG1 gene fusions are predominantly mutually exclusive of other known genomic driver mutations and are considered a unique oncogenic driver event associated with tumor cell survival.

NRG1 fusions have been identified in a variety of solid tumors, including lung, pancreatic, gallbladder, breast, ovarian, colorectal, neuroendocrine, cholangiocarcinomas, and sarcomas. In preclinical experiments, seribantumab prevented the activation of HER3 signaling in cells that harbor an NRG1 gene fusion and destabilized the entire ERBB family signaling pathway, including the activation of HER2, EGFR, and HER4. In addition to extensive nonclinical characterization and testing, seribantumab has been administered to more than 800 patients across twelve Phase 1 and 2 studies, both as a monotherapy and in combination with various anti-cancer therapies. Seribantumab is currently being evaluated in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 fusion.

About the Phase 2 CRESTONE Study

Clinical Study of Response to Seribantumab in Tumors with Neuregulin-1 (NRG1) Fusions (CRESTONE) is a Phase 2 tumor-agnostic "basket trial" evaluating the safety and efficacy of seribantumab in patients with solid tumors that harbor an NRG1 fusion and have progressed after at least one prior line of standard therapy. The primary objective of the study is to describe the anti-tumor activity and safety of seribantumab as a monotherapy specifically in patients whose solid tumor is uniquely driven by an NRG1 gene fusion. CRESTONE offers a clinical trial opportunity for patients with advanced solid tumors who have not responded or are no longer responding to treatment. Patients are encouraged to talk to their doctor about genomic testing of their tumor. CRESTONE is open and enrolling patients in the United States, Australia, and Canada. For more information visit www.NRG1fusion.com.

On May 5, 2022 Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) reported financial results for the first quarter of 2022 (Press release, Concert Pharmaceuticals, MAY 5, 2022, View Source [SID1234613614]).

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"We are at an exciting point in our progress with CTP-543, our drug candidate for alopecia areata, a serious autoimmune disorder with significant unmet medical need. Our pivotal trials, THRIVE-AA1 and THRIVE-AA2, have collectively enrolled over 1,200 patients in the U.S., Canada and Europe. We’re grateful to the many individuals who chose to participate in our studies to advance research for this important autoimmune disorder and, in the event of positive data, provide us with the information we need to support filing our New Drug Application with the FDA, which is planned for the first half of next year," stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "We continue to expect to report topline results from the THRIVE‑AA1 trial this quarter. The THRIVE-AA2 topline results are expected to follow soon thereafter, in the third quarter of 2022."

Recent Business Highlights and Upcoming Milestones

CTP-543: An Investigational Treatment in Phase 3 Trials for Moderate to Severe Alopecia Areata

Topline Data for THRIVE-AA1 Expected in the Second Quarter of 2022. The Company expects to report topline results from the first CTP-543 Phase 3 trial, THRIVE-AA1, in the second quarter of 2022. THRIVE-AA1 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial of CTP‑543 to evaluate hair regrowth using the Severity of Alopecia Tool (SALT) after 24 weeks of dosing. The trial is evaluating 8 mg and 12 mg twice-daily oral doses of CTP-543 compared to placebo. The trial enrolled 708 adult patients with moderate to severe alopecia areata at sites in the U.S., Canada and Europe.
Topline Data for THRIVE-AA2 Expected in the Third Quarter of 2022. The Company expects to report topline results from the THRIVE-AA2 trial in the third quarter of 2022. THRIVE-AA2 is a randomized, double-blind, placebo-controlled Phase 3 clinical trial of CTP‑543 to evaluate hair regrowth using SALT after 24 weeks of dosing. The trial is evaluating 8 mg and 12 mg twice-daily oral doses of CTP-543 compared to placebo. The trial enrolled 517 adult patients with moderate to severe alopecia areata at sites in the U.S., Canada and Europe.
New Drug Application (NDA) Filing for Alopecia Areata Expected in the First Half of 2023. If the CTP-543 clinical program is successful, the Company intends to file an NDA with the U.S. Food and Drug Administration (FDA) in the first half of 2023. If approved, the Company expects that CTP-543 would be one of the first FDA-approved treatments for alopecia areata. The FDA has granted CTP-543 Breakthrough Therapy designation for the treatment of adult patients with moderate to severe alopecia areata and Fast Track designation for the treatment of alopecia areata. Alopecia areata is an autoimmune disease that may affect up to approximately 1.5 million Americans at any given time.1
First Quarter 2022 Financial Results

Cash and Investment Position. Cash, cash equivalents and investments as of March 31, 2022 totaled $109.0 million as compared to $141.6 million as of December 31, 2021. Under its current operating plan, the Company expects its current cash, cash equivalents and investments to fund the Company into the fourth quarter of 2022. In addition, Concert has the potential to receive an additional $103.1 million upon the full exercise of the warrants issued in connection with its November 2021 financing.
R&D Expenses. Research and development expenses were $30.5 million for the quarter ended March 31, 2022, compared to $18.5 million for the same period in 2021. The increase in research and development expenses relates primarily to the clinical development for CTP-543.
G&A Expenses. General and administrative expenses were $5.5 million for each of the quarters ended March 31, 2022 and 2021. General and administrative expenses primarily consist of employee benefits and compensation and external professional services.
Net Loss. Net loss applicable to common stockholders was $37.7 million, or $1.03 per share, for the quarter ended March 31, 2022, as compared to net loss applicable to common stockholders of $22.7 million, or $0.67 per share, for the quarter ended March 31, 2021.
Conference Call and Webcast

The Company will host a conference call and webcast today at 8:30 a.m. ET to provide an update on the Company and discuss its first quarter 2022 financial results. To access the conference call, please dial (855) 354-1855 (U.S. and Canada) or (484) 365-2865 (International) five minutes prior to the start time.

A live webcast may be accessed in the Investors section of the Company’s website at www.concertpharma.com. Please log on to the Concert website approximately 15 minutes prior to the scheduled webcast to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Concert’s website for three months.

On May 5, 2022 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported its financial results for the first quarter ended March 31, 2022 and provided an update on the Company’s operations (Press release, BridgeBio, MAY 5, 2022, View Source [SID1234613613]).

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"Building on our positive Phase 2 data in LGMD2i, we are excited for a summer of ongoing focused execution where we have the opportunity to deliver meaningful data in two additional large conditions with great unmet need: ADH1 and achondroplasia," said Neil Kumar, Ph.D., founder and CEO of BridgeBio.

BridgeBio’s key programs:

Acoramidis (AG10) – Transthyretin (TTR) stabilizer for transthyretin amyloid cardiomyopathy (ATTR-CM): The Company released updated data from its ongoing Phase 2 open-label extension (OLE) study of acoramidis in patients with ATTR-CM, which demonstrated that acoramidis continued to be well-tolerated and potently stabilize TTR. NT-proBNP, a biomarker of cardiac failure and independent predictor of mortality in ATTR-CM patients, was stable or improving throughout the study and serum TTR levels were sustainably increased from baseline at Month 30. Topline data from the Month 30 primary endpoint of the ongoing Phase 3 trial of acoramidis in ATTR-CM (ATTRibute-CM), a hierarchical composite including all-cause mortality and cardiovascular hospitalizations, are expected in mid-2023.

Encaleret – Calcium-sensing receptor (CaSR) inhibitor for autosomal dominant hypocalcemia type 1 (ADH1): Data from BridgeBio’s Phase 2b study of encaleret in ADH1 to be presented at the Endocrine Society’s 2022 ENDO conference in Atlanta from June 11-14, 2022. The planned presentation will include the profile of encaleret on clinically relevant mineral homeostasis parameters over 24 weeks of outpatient evaluation. Achieving simultaneous blood and urine calcium normalization is a challenge for patients with ADH1 due to the limitations of the current standard of care. If approved, encaleret could be the first therapy indicated for the treatment of ADH1, a condition caused by gain of function variants of the CASR gene estimated to be carried by 12,000 individuals in the United States alone. The Company intends to initiate a Phase 3 registrational trial of encaleret in patients with ADH1 in 2022 and anticipates Phase 3 topline data in 2023. Issued and filed patents are expected to provide market protection for encaleret in ADH1 to 2041.

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia and hypochondroplasia: Initial data from the ongoing Phase 2 dose-escalation and expansion study is anticipated in mid-2022. The Company also plans to share multiple presentations at the Endocrine Society’s 2022 ENDO conference in Atlanta from June 11-14, 2022, including preclinical data for low-dose infigratinib in a mouse model of hypochondroplasia (an FGFR3-driven skeletal dysplasia), new analyses of natural history data in achondroplasia, and the results of a qualitative research study conducted to identify concepts and quality-of-life questionnaires relevant for the achondroplasia community. Achondroplasia is the most common form of genetic short stature and one of the most common genetic diseases, with a prevalence of over 55,000 cases in the United States and European Union. Low-dose infigratinib is the only known product candidate in clinical development for achondroplasia that is designed to target the disease at its genetic source and the only orally administered product candidate in clinical development for achondroplasia.

BBP-631 – AAV5 gene therapy candidate for congenital adrenal hyperplasia (CAH): Dosed first patient in Phase 1/2 gene therapy trial in January 2022. Initial Phase 1/2 data readout anticipated in second half of 2022. CAH is one of the most prevalent genetic diseases potentially addressable with adeno-associated virus (AAV) gene therapy, with more than 75,000 cases estimated in the United States and European Union. The disease is caused by deleterious mutations in the gene encoding an enzyme called 21-hydroxylase, leading to a lack of endogenous cortisol production. BBP-631 is designed to provide a functional copy of the 21-hydroxylase-encoding gene (CYP21A2) and potentially address many aspects of the disease course.

BBP-418 – Glycosylation substrate for limb-girdle muscular dystrophy type 2i (LGMD2i): Announced positive Phase 2 data at the Muscular Dystrophy Association 2022 Annual Meeting on March 14, 2022. BridgeBio plans to engage with regulatory health bodies in 2022 to discuss potential paths to approval and subsequently intends to initiate a Phase 3 clinical trial. If successful, BBP-418 could be the first approved therapy for patients with LGMD2i. BridgeBio believes its initial Phase 2 results indicate the potential for BBP-418 to increase glycosylation of αDG and drive functional improvements for patients, as well as reduce CK, a key marker of muscle breakdown. Furthermore, the 90- and 180-day data show improvements on walk tests from baseline, which the Company believes suggests a potential impact on clinical function and on the rate of disease progression.

RAS cancer portfolio: BridgeBio expects to select a RAS development candidate in 2022. It previously announced the discovery of its next-generation KRAS G12C dual inhibitors, the first-known compounds that directly bind and inhibit KRAS in both its active (GTP bound) and inactive (GDP bound) conformations, and PI3ka:RAS breakers, small molecules that block RAS driven PI3Ka activation – a novel approach with the potential to inhibit oncogenic PI3Ka signaling without adverse effects on glucose metabolism. RAS is one of the most well-known oncogenic drivers with approximately 30% of all cancers being driven by RAS mutations, including large proportions of lung, colorectal and pancreatic tumors.

Recent corporate updates

Sale to Sentynl Therapeutics, Inc., of NULIBRY (Fosdenopterin) for Injection: Sentynl has acquired global rights to NULIBRY and will be responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing and commercializing fosdenopterin globally. BridgeBio will share development responsibilities for fosdenopterin through approval of the marketing authorization application already under accelerated assessment with the European Medicines Agency and through approval of its regulatory submission with the Israeli Ministry of Health. Sentynl will provide cash payments upon the achievement of certain regulatory milestones. BridgeBio will be eligible to receive commercial milestone payments as well as tiered royalties on adjusted net sales of NULIBRY. NULIBRY is approved by the U.S. Food and Drug Administration (FDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency Type A, an ultra-rare, life-threatening pediatric genetic disorder.

Updated strategic collaboration with Helsinn Group to develop, manufacture and commercialize infigratinib in oncology indications in the U.S.: Under the terms of the amended and restated agreement, Helsinn will gain an exclusive license to commercialize infigratinib in the United States and will be responsible for developing, manufacturing and commercializing infigratinib in oncology indications worldwide except for achondroplasia or any other skeletal dysplasias, and except in mainland China, Hong Kong and Macau. BridgeBio will be eligible to receive regulatory and commercial milestone payments as well as tiered royalties on adjusted net sales from Helsinn. BridgeBio will retain all rights to develop, manufacture and commercialize infigratinib in skeletal dysplasia, including achondroplasia. In 2021, Helsinn and BridgeBio obtained accelerated approval for TRUSELTIQ (infigratinib) from the FDA for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 fusion or other rearrangement as detected by an FDA-approved test.

Business development – Due to the need to conserve capital and prioritize focused execution, BridgeBio is engaged in partnering and out-licensing discussions for the following programs:

BBP-589 – Recombinant collagen 7 for dystrophic epidermolysis bullosa
Completed Phase 2 clinical trial evaluating safety and tolerability of BBP-589, as well as clinical proof-of-concept, and initiated a Phase 2 extension study
BBP-681 – Topical PI3KA inhibitor for venous and lymphatic malformations
Phase 1/2 clinical trial underway
BBP-561 – KLK 5/7 inhibitor for Netherton syndrome
Investigational New Drug application (IND) enabling studies underway
BBP-815 – AAV gene therapy for nonsyndromic hearing loss
Preclinical development
BBP-818 – AAV gene therapy for classic galactosemia
IND-enabling studies underway
BBP-472 – PI3KB inhibitor for PTEN autism
Preclinical development

First Quarter 2022 Financial Results:

Cash, Cash Equivalents and Marketable Securities

Cash, cash equivalents and marketable securities, excluding restricted cash, totaled $633.5 million as of March 31, 2022, compared to $787.5 million as of December 31, 2021. The net decrease of $154.0 million pertains primarily to payments for operating costs and expenses of $160.6 million, which includes $18.8 million in payments for debt-related interests, timing of payments of accrued compensation and benefits of $16.9 million and $3.9 million in payments related to the restructuring initiative that the Company implemented during the current quarter as described below. During the current quarter, the Company received an upfront payment of $10.0 million upon closing of the asset purchase agreement between its affiliate, Origin Biosciences, and Sentynl Therapeutics.

"We made significant progress in reducing our expenditures in the first quarter, highlighted by a combined reduction of over $100 million in 2022-2023 operating expenses through the Helsinn and Sentynl deals, but we are not yet done. Our priorities are focusing resources towards select internal programs while finding value-creating partnerships for others that we believe allow science to advance seamlessly towards patients. Our expectation is that operating expenses and cash burn will meaningfully decline in the second quarter as restructuring charges from the beginning of the year decline and anticipated additional business development activity allows us to decrease further from that baseline, establishing a reduced run rate for the rest of the year. We currently expect to have runway up to and beyond the readout of our ATTR catalyst in mid-2023 without requiring equity dilution. We reiterate our guidance of runway into 2024," said Brian Stephenson, Ph.D., CFA, BridgeBio’s Chief Financial Officer.

BridgeBio is engaged in partnering and out-licensing discussions for a select number of programs, which are listed on the Company’s website.

Operating Costs and Expenses

Operating costs and expenses increased by $7.4 million to $175.4 million for the first quarter of 2022 as compared to $168.0 million for the same period in the prior year. During the current quarter, the Company incurred $22.7 million in restructuring, impairment and related charges, as part of its restructuring initiative. In January 2022, the Company committed to a restructuring initiative designed to drive operational changes in its business processes, efficiencies and cost savings to advance corporate strategy and development programs. The restructuring initiative included, among other components, consolidation and rationalization of facilities, reprioritization of development programs and the reduction of our workforce. The Company has substantially completed the activities and has incurred most of the costs related to the restructuring initiative during the current quarter. The Company estimates to incur total charges in the range of approximately $23.0 million to $25.0 million for the fiscal year 2022, consisting primarily of impairments and write-offs of long-lived assets, severance and employee-related costs, and exit and other related costs. The estimate of the range of costs is subject to certain assumptions, such as the Company’s ability to sublease certain office spaces. Actual results may differ from those estimates or assumptions. The nonrecurring charges related to the restructuring initiative were partially offset by a $15.3 million decrease in the Company’s normal operating expenses compared to the same period in the prior year, primarily due to cost savings and impacts from such restructuring initiative.

The Company’s research and development expenses have not been significantly impacted by the global COVID-19 pandemic for the periods presented. While BridgeBio experienced some delays in certain of its clinical enrollment and trial commencement activities, it continues to adapt in this unprecedented time to enable alternative site, telehealth and home visits, at-home drug delivery, as well as mitigation strategies with its contract manufacturing organizations. The longer-term impact, if any, of COVID-19 on BridgeBio’s operating costs and expenses is currently unknown.

On May 5, 2022 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported it will release its unaudited financial results for the quarter ended March 31, 2022 on Wednesday, May 11, 2022, before the US markets open (Press release, BioLineRx, MAY 5, 2022, View Source [SID1234613612]).

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The Company will host a conference call on Wednesday, May 11, 2022 at 10:00 a.m. EDT featuring remarks by Philip Serlin, Chief Executive Officer. The conference call will be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

To dial into the conference call, please dial +1-866-744-5399 from the U.S. or +972-3-918-0644 internationally. A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until May 13, 2022; please dial +1-888-295-2634 from the U.S. or +972-3-925-5904 internationally.