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.

On May 5, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) reported financial results for the first quarter ended March 31, 2022, and provided a corporate update (Press release, BioCryst Pharmaceuticals, MAY 5, 2022, View Source [SID1234613611]).

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"We are now over a year into the ORLADEYO launch and are excited to see strong and continuing patient demand and steady expansion in our prescriber base among both new and existing prescribers. These trends continued in the first quarter of 2022 and reinforce our confidence that we will achieve no less than $250 million in net ORLADEYO revenue in 2022 and peak ORLADEYO sales of $1 billion," said Jon Stonehouse, president and chief executive officer of BioCryst.

"We also have made substantial progress in our investigation with BCX9930. Based on our initial findings, we believe that both dose and dosing regimen could be contributing factors to the safety signal we have observed. By the end of the third quarter, we plan to discuss our proposed approach to resume the REDEEM trials, under a revised dosing protocol, with regulators," Stonehouse added.

Program Updates and Key Milestones

ORLADEYO (berotralstat): Oral, Once-daily Treatment for Prevention of Hereditary Angioedema (HAE) Attacks

U.S. Launch

ORLADEYO net revenue in the first quarter of 2022 was $49.7 million.
Approximately 50 percent of patients currently on ORLADEYO have switched from another prophylactic therapy, and half of those patients have come from lanadelumab. These trends continued with new patients starting ORLADEYO in Q1 2022.
In the first quarter, new patient prescriptions were evenly split between repeat prescribers and new prescribers, with an approximately equal number of new prescriptions coming from the top 500 HAE treaters and the broader set of other HAE treaters.
The company has completed its latest quarterly survey of another 60 allergists who treat an average of eight HAE patients. These physicians were already using ORLADEYO on 13 percent of their patients and predicted growth to 23 percent over the next 12 months. These findings were in line with previously reported market research from August 2021.
Approximately 80 percent of HAE patients in the U.S. are insured by payors and pharmacy benefit managers that cover ORLADEYO. Despite prior authorization headwinds early in 2022, approximately 80 percent of patients on ORLADEYO were receiving paid product by the end of the first quarter, a substantial increase from approximately 66 percent of patients in the second half of 2021.
Most patients are well-controlled on ORLADEYO and remain on therapy. Once payor prior authorization is complete, 78 percent of patients switching from lanadelumab and 73 percent of patients switching from subcutaneous C1 inhibitor remain on ORLADEYO for at least six months. About 70 percent of all patients who receive reimbursed product stay on ORLADEYO for at least 12 months, compared to 60 percent of patients who remain on long-term free product.
The company expects steady quarterly ORLADEYO net revenue growth throughout the remainder of 2022, with total 2022 ORLADEYO net revenue of no less than $250 million.
"We are very pleased that strong patient demand continued to drive growth in the first quarter and overcome the traditional Q1 reimbursement headwinds. With outstanding reimbursement in place, patients continuing to enjoy an excellent experience on ORLADEYO and the expansion of both our new and existing prescriber bases, we expect steady growth throughout the remainder of 2022 as we achieve no less than $250 million in ORLADEYO net revenues for the year," said Charlie Gayer, chief commercial officer of BioCryst.

ORLADEYO: Global Updates

ORLADEYO has been launched in Denmark, France, Germany, Japan, Norway, Sweden, the United Arab Emirates and the United Kingdom. The company expects launches in additional countries throughout the year.
Complement Oral Factor D Inhibitor Program – BCX9930

On April 8, 2022, BioCryst announced that the company was voluntarily pausing enrollment in BCX9930 clinical trials while it investigated observed elevations in serum creatinine seen in some patients.

Patients in the REDEEM-1, REDEEM-2 and RENEW clinical trials randomized to BCX9930 began those trials by starting immediately at a dose of 500 mg twice-daily. Patients receiving BCX9930 in the long-term extension trial started at lower doses as part of the proof-of-concept dose escalation regimen and were ultimately moved up to 500 mg twice-daily.

Preliminary evidence from the investigation points to both the 500 mg twice-daily dosing level and the immediate start of that dose, without a period at a lower dose first, as plausible contributory factors for the observed increases in serum creatinine.

Based on the initial results of the investigation, and the safety and efficacy data observed in the BCX9930 clinical program at 400 mg twice-daily, the company plans to discuss with regulators whether clinical trials with amended protocols could resume using stepped dosing to 400 mg twice-daily. The company expects to have discussions with regulators by the end of the third quarter.

During the ongoing investigation the company has observed the following:

Three patients with PNH receiving BCX9930 in the REDEEM trials had early onset, and moderate or severe, elevations in their serum creatinine (2-4 xULN) after several weeks of dosing with 500 mg twice-daily. Two of these patients have been discontinued from therapy and one patient (who had the smallest increase in serum creatinine) continues on BCX9930 at this time.
The company estimates that one-third of subjects randomized to BCX9930 in the REDEEM studies have had early increases in serum creatinine.
The company also found a different pattern of slowly evolving, late onset, mild to moderate increases in serum creatinine in approximately 40 percent of patients in the long-term extension of the proof-of-concept trial, after those patients switched to the 500 mg twice-daily dose. This pattern was not observed during treatment with doses lower than 500 mg.
Subsequent to BioCryst voluntarily pausing trial enrollments, the U.S. Food and Drug Administration (FDA) informed the company that it has placed the clinical program for BCX9930 on a partial clinical hold. Consistent with BioCryst’s voluntary action, the company may not enroll new patients in its BCX9930 clinical trials, however patients already enrolled who are receiving clinical benefit from BCX9930 treatment, and have no other available treatment options, can continue to be dosed and remain in the trials.

"As we complete our investigation, we will continue to be comprehensive and deliberate, with a primary focus on patient safety. After consultation with regulators, we will determine the next step for the BCX9930 program," said Dr. William Sheridan, Chief Medical Officer of BioCryst.

The company does not plan to provide additional updates on the BCX9930 program until it completes additional regulatory discussions and has more clarity on the next steps for the program.

Additional Updates

On February 8, 2022, the company announced the appointment of Machelle Sanders to its board of directors.
First Quarter 2022 Financial Results

For the three months ended March 31, 2022, total revenues were $49.9 million, compared to $19.1 million in the first quarter of 2021 (+161.3 percent year-over-year (y-o-y)). The increase was primarily due to $49.7 million in ORLADEYO net revenue in the first quarter of 2022, compared to $10.9 million in ORLADEYO net revenue in the first quarter of 2021 (+356.0 percent y-o-y).

Research and development (R&D) expenses for the first quarter of 2022 increased to $65.4 million from $42.4 million in the first quarter of 2021 (+54.2 percent y-o-y), primarily due to increased investment in the development of our Factor D program, including BCX9930, as well as other research, preclinical and development costs.

Selling, general and administrative (SG&A) expenses for the first quarter of 2022 increased to $34.3 million, compared to $22.1 million in the first quarter of 2021 (+55.2 percent y-o-y). The increase was primarily due to increased investment to support the commercial launch of ORLADEYO and expanded international operations.

Interest expense was $23.8 million in the first quarter of 2022, compared to $12.9 million in the first quarter of 2021 (+84.5 percent y-o-y). The increase was due to service on the royalty financings, which were completed in November 2021.

Net loss for the first quarter of 2022 was $74.2 million, or $0.40 per share, compared to a net loss of $64.3 million, or $0.36 per share, for the first quarter of 2021.

Cash, cash equivalents, restricted cash and investments totaled $446.8 million at March 31, 2022, compared to $244.4 million at March 31, 2021. Operating cash use for the first quarter of 2022 was $71.0 million.

Financial Outlook for 2022

Based on the strength of the ORLADEYO launch, and continued steady growth from new patient demand anticipated throughout the year, the company expects full year 2022 net ORLADEYO revenue to be no less than $250 million.

The company had previously expected operating expenses for full year 2022, not including non-cash stock compensation, to be between $440 million to $480 million. Once the company completes its investigation into BCX9930 and has clarity on the next step for the program it expects to provide an updated outlook on full year 2022 operating expenses. If BCX9930 program enrollment resumes, then operating expenses are likely to be at the lower end of the previously provided range. If we discontinue the BCX9930 program, then operating expenses for the year would be lower than that.

Conference Call and Webcast

BioCryst management will host a conference call and webcast at 8:30 a.m. ET today to discuss the financial results and provide a corporate update. The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 9498023. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 9498023.

On May 5, 2022 Bicycle Therapeutics plc (NASDAQ: BCYC), a biotechnology company pioneering a new and differentiated class of therapeutics based on its proprietary bicyclic peptide (Bicycle) technology, reported financial results for the first quarter ended March 31, 2022 and provided recent corporate updates (Press release, Bicycle Therapeutics, MAY 5, 2022, View Source [SID1234613610]).

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"We have made significant progress recently across our oncology pipeline," said Kevin Lee, Ph.D., Chief Executive Officer of Bicycle Therapeutics. "We reported interim data for the Phase I portion of the Phase I/II trial in BT8009, showing an overall response rate, or ORR, of 50% in the 5mg/m2 weekly cohort in patients with urothelial cancer and an encouraging tolerability profile. We believe BT8009 has the potential to offer clinically meaningful differentiation compared to currently available therapies and we look forward to advancing the program once the dose escalation phase is complete. We also look forward to initiating the BT5528 Phase II expansion cohorts soon."

Dr. Lee continued: "Beyond our Bicycle Toxin Conjugates, or BTCs, we continue to be encouraged as we progress our Bicycle TICA programs forward, including BT7480, which is advancing in the dose escalation portion of the Phase I clinical trial. Underscoring our efforts is a strong balance sheet and we anticipate our cash and cash equivalents will provide financial runway through 2024."

First Quarter 2022 and Recent Highlights

Announced Interim BT8009 Phase I Clinical Trial Results at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. In April 2022, Bicycle announced interim Phase I results from the Phase I/II trial of BT8009, a second-generation BTC targeting Nectin-4. As of March 7, 2022, thirty-seven patients have been dosed in the Phase I/II trial of BT8009. A total of twelve response evaluable urothelial cancer (UC) patients have been dosed in monotherapy cohorts of 2.5mg/m2 and 5.0mg/m2 weekly in the ongoing trial. Four response evaluable UC patients were dosed at 2.5mg/m2 weekly, with one patient observed to have tumor reductions constituting a confirmed partial response (PR) and two patients observed to have stable disease (SD). Eight response evaluable UC patients were dosed at 5.0mg/m2 weekly, with four patients observed to have a confirmed complete response (CR) or PR, including one patient with a CR and three patients with a PR, and two patients with SD, reflecting a 50% ORR and 75% disease control rate. The median duration of response has not yet been reached in either the 2.5 mg/m2 or 5.0mg/m2 cohort, with four of the five responders in these cohorts still on therapy after at least 24 weeks, and a tolerability profile that remains consistent with earlier results from this trial.

Exploration of additional doses and frequencies continues, and Bicycle intends to provide further updates this year.
Announced Updated Preclinical Data on SARS-CoV-2 Antivirals at the 2022 Microbiology Society Annual Meeting. Bicycle created a wide range of <10kDa molecules with high binding affinity to the SARS-CoV-2 spike protein and then tested them in partnership with researchers at the Medical Research Council Laboratory of Molecular Biology. They were shown to be highly active in vitro at preventing SARS-CoV-2 infection of human cells. These compounds were further tested in partnership with experts at the University of Liverpool and shown to be highly potent when administered in vivo to rodent models of SARS-CoV-2 infection. These molecules have also been shown to be active in vitro across many of the SARS-CoV-2 variant strains, including Alpha, Beta, Delta and Omicron.
Financial Results

Cash and cash equivalents were $407.4 million as of March 31, 2022, compared to $438.7 million as of December 31, 2021. The decrease in cash is primarily due to cash used in operating activities.
Research and development expenses were $14.3 million for the three months ended March 31, 2022, compared to $9.7 million for the three months ended March 31, 2021. The increase in expense of $4.6 million was primarily due to increased discovery and platform-related expenses, as well as increased personnel-related expenses, including $1.2 million of incremental non-cash share-based compensation expense.
General and administrative expenses were $17.0 million for the three months ended March 31, 2022, compared to $8.1 million for the three months ended March 31, 2021. The increase of $8.9 million for the three months ended March 31, 2022 as compared to the same period in the prior year was primarily due to $5.2 million incremental non-cash share-based compensation expense, personnel-related costs and increased professional and consulting costs.
Net loss was $27.6 million, or $(0.93) basic and diluted net loss per share, for the three months ended March 31, 2022, compared to net loss of $16.2 million, or $(0.73) basic and diluted net loss per share, for three months ended March 31, 2021.