On August 3, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrine, oncologic, metabolic and neurological disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended June 30, 2022 (Press release, Corcept Therapeutics, AUG 3, 2022, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-second-quarter-financial-0 [SID1234617368]).

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Financial Results

Revenue of $103.4 million, compared to $91.6 million in second quarter 2021
Reiterated 2022 revenue guidance of $400 – $430 million
Diluted net income per common share of $0.24, compared to $0.21 in second quarter 2021
Cash and investments of $382.0 million, compared to $368.1 million at March 31, 2022
Corcept’s second quarter 2022 revenue was $103.4 million, compared to $91.6 million in the second quarter of 2021. Second quarter operating expenses were $72.0 million, compared to $59.6 million in the second quarter of 2021, due to increased clinical trial activity, expenses to support the expansion of our clinical development and commercial teams and legal fees. Net income was $27.4 million in the second quarter of 2022, compared to $26.5 million in the second quarter of 2021.

Cash and investments increased $13.9 million in the second quarter, to $382.0 million at June 30, 2022.

"Our revenue growth reflects the continued increase in our base of Korlym prescribers," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Korlym is an excellent treatment for patients with Cushing’s syndrome and there are many eligible patients who have yet to receive it. We are reiterating our 2022 revenue guidance of $400 – $430 million."

Clinical Development

"Data generated by our clinical development programs have provided increasing evidence of cortisol modulation’s potential to treat many serious diseases," added Dr. Belanoff. "Positive results from our Phase 2 trial of selective cortisol modulator relacorilant in women with platinum-resistant ovarian cancer led to the recent initiation of our Phase 3 ROSELLA trial. Later this year, we expect important data from our programs in antipsychotic-induced weight gain and non-alcoholic steatohepatitis."

Solid Tumors

ROSELLA initiated – 360-patient pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with recurrent, platinum-resistant ovarian cancer
Enrollment continues in 20-patient, open-label, Phase 1b trial of relacorilant plus
pembrolizumab in patients with adrenal cancer with cortisol excess
Randomized, placebo-controlled Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer to begin in collaboration with the University of Chicago
"Opening our ROSELLA study is an important step forward," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "The 40,000 women in the United States and Europe with platinum-resistant ovarian cancer have few good treatment options. Our Phase 2 study demonstrated improvements in progression-free survival, duration of response and overall survival without increased side effect burden. Simply replicating these positive results in ROSELLA would be of unprecedented benefit to women with platinum-resistant ovarian cancer, for whom relacorilant plus nab-paclitaxel has the potential to become a new standard."

"We are also pleased to collaborate with investigators at the University of Chicago in initiating a placebo-controlled, Phase 2 trial of relacorilant, combined with enzalutamide, in patients with prostate cancer, pre-prostatectomy," said Dr. Guyer. "There is a large patient population at this stage of disease and we hope to be able to offer them substantial benefit."

Metabolic Diseases

Enrollment completed in GRATITUDE and GRATITUDE II – two double-blind, placebo-controlled Phase 2 trials of miricorilant to reverse recent and long-standing antipsychotic-induced weight gain (AIWG); data from both trials expected in fourth quarter 2022
Enrollment continues in Phase 1b dose-finding trial of miricorilant in patients
with presumed non-alcoholic steatohepatitis (NASH); data expected in fourth quarter 2022
"Weight gain and other adverse metabolic effects caused by antipsychotic medications reduce the life expectancy of millions of patients. These side effects also dissuade many patients from adhering to their treatment regimen," said Dr. Guyer. "We expect our double-blind, placebo-controlled GRATITUDE and GRATITUDE II trials to build on the positive data from our Phase 1 studies in healthy volunteers."

Cushing’s Syndrome

Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome; new drug application (NDA) submission expected
in second half 2023
Enrollment continues in Phase 3 GRADIENT trial of relacorilant as a treatment for patients
with Cushing’s syndrome caused by adrenal adenomas
"We advanced relacorilant to Phase 3 in Cushing’s syndrome based on its extremely promising Phase 2 efficacy and safety data. We expect our GRACE trial to serve as the basis for relacorilant’s NDA in Cushing’s syndrome, which we plan to submit in the second half of 2023," said Dr. Guyer. "The Phase 3 GRADIENT trial will produce valuable data about an etiology of Cushing’s syndrome that has not been subject to rigorous, controlled study, but affects many patients."

Amyotrophic Lateral Sclerosis (ALS)

DAZALS, a 198-patient, randomized, double-blind, placebo-controlled Phase 2 trial of dazucorilant in patients with ALS, planned to start this quarter
"ALS, commonly known as Lou Gehrig’s disease, is a devastating illness with a significant need for better treatment," said Dr. Guyer. "Dazucorilant, a selective cortisol modulator that crosses the blood-brain barrier, showed outstanding promise in animal models of ALS. We plan to initiate a large, controlled Phase 2 trial, which we have named DAZALS, this quarter, at sites in Europe and the United States."

Conference Call

We will hold a conference call on August 3, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number, and a unique access PIN. Each access PIN will accommodate one caller. Additionally, a listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of www.corcept.com.

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. Corcept holds patents directed to the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.

On August 3, 2022 Charles River Laboratories International, Inc. (NYSE: CRL) reported its results for the second quarter of 2022 (Press release, Charles River Laboratories, AUG 3, 2022, View Source [SID1234617366]). For the quarter, revenue was $973.1 million, an increase of 6.4% from $914.6 million in the second quarter of 2021.

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Acquisitions contributed 2.3% to consolidated second-quarter revenue growth. The divestitures of the Research Models and Services operations in Japan (RMS Japan) and CDMO site in Sweden (CDMO Sweden) in October 2021 reduced reported revenue growth by 2.0%. The impact of foreign currency translation reduced reported revenue growth by 3.4%. Excluding the effect of these items, organic revenue growth of 9.5% was driven primarily by the Discovery and Safety Assessment (DSA) and Research Models and Services (RMS) business segments.

On a GAAP basis, second-quarter net income attributable to common shareholders was $109.3 million, an increase of 23.6% from net income of $88.4 million for the same period in 2021. Second-quarter diluted earnings per share on a GAAP basis were $2.13, an increase of 23.8% from $1.72 for the second quarter of 2021. The increases in GAAP net income and earnings per share were primarily driven by higher revenue and acquisition-related adjustments. In addition, GAAP net income and earnings per share included a loss from the Company’s venture capital and other strategic investments of $0.14 per share in the second quarter of 2022, compared to a gain of $0.14 per share for the same period in 2021. The Company’s venture capital and other strategic investment performance has been excluded from non-GAAP results.

On a non-GAAP basis, net income was $141.9 million for the second quarter of 2022, an increase of 6.0% from $133.8 million for the same period in 2021. Second‑quarter diluted earnings per share on a non-GAAP basis were $2.77, an increase of 6.1% from $2.61 per share for the second quarter of 2021. The non-GAAP net income and earnings per share increases were driven primarily by higher revenue and operating margin improvement, partially offset by higher interest expense and tax rate.

James C. Foster, Chairman, President and Chief Executive Officer, said, "Our second-quarter financial results reflect the sustained trends that continue to support our business, particularly our DSA and RMS business segments for which demand continues to be strong and the performance remains consistent with our initial outlook for the year. Safety Assessment continues to benefit from a growing backlog that is well above the prior-year level and solid booking activity, which support the anticipated DSA growth acceleration in the second half of the year."

"However, these robust trends were offset by headwinds from our CDMO business, as well as unfavorable changes in foreign exchange and interest rates, which have significantly intensified over the past two months. We have revised our financial outlook for 2022 to account for these escalating headwinds, resulting in lower revenue growth and earnings per share guidance for the year," Mr. Foster concluded.

Second-Quarter Segment Results

Research Models and Services (RMS)

Revenue for the RMS segment was $186.4 million in the second quarter of 2022, an increase of 5.5% from $176.7 million in the second quarter of 2021. Organic revenue growth of 8.5% was primarily driven by research model services, particularly the Insourcing Solutions (IS) and Genetically Engineered Models and Services (GEMS) business. Revenue growth for research models in North America and China also contributed. Revenue in China increased in the second quarter, but was negatively impacted by COVID-related restrictions that affected client order activity.

In the second quarter of 2022, the RMS segment’s GAAP operating margin decreased to 21.2% from 24.1% in the second quarter of 2021, and on a non-GAAP basis, the operating margin decreased to 24.9% from 27.4%. The GAAP and non-GAAP operating margin decreases were driven primarily by the COVID-related revenue impact in China.

Discovery and Safety Assessment (DSA)

Revenue for the DSA segment was $591.9 million in the second quarter of 2022, an increase of 9.6% from $540.1 million in the second quarter of 2021. Organic revenue growth of 12.9% was driven by strong demand and price increases in the Safety Assessment and Discovery Services businesses.

In the second quarter of 2022, the DSA segment’s GAAP operating margin increased to 21.8% from 19.4% in the second quarter of 2021, and on a non-GAAP basis, the operating margin increased to 25.3% from 23.5%. The GAAP and non-GAAP operating margin increases were driven primarily by operating leverage from higher revenue in both the Discovery Services and Safety Assessment businesses.

Manufacturing Solutions (Manufacturing)

Revenue for the Manufacturing segment was $194.8 million in the second quarter of 2022, a decrease of 1.5% from $197.8 million in the second quarter of 2021. Organic revenue growth of 1.0% reflected higher revenue in the Biologics Testing and Microbial Solutions businesses, which was effectively offset by a revenue decline in the CDMO business.

In the second quarter of 2022, the Manufacturing segment’s GAAP operating margin increased to 32.1% from 28.7% in the second quarter of 2021. The GAAP operating margin in the second quarter benefitted from acquisition-related adjustments associated with last year’s Cognate and Vigene CDMO transactions. On a non-GAAP basis, the operating margin decreased to 28.6% from 33.2% in the second quarter of 2021, primarily as a result of the CDMO business.

Reduces 2022 Guidance

The Company is reducing its 2022 financial guidance, due primarily to headwinds associated with the CDMO business, foreign exchange due to the strengthening U.S. dollar, and interest expense due to the rising interest rate environment.

Reported revenue growth guidance is being reduced by 450 basis points to reflect unfavorable movements in foreign currency translation, as well as a lower revenue growth rate in the Manufacturing segment, driven principally by the CDMO business.

Organic revenue growth guidance for 2022 is being reduced by 250 basis points, also driven largely by the CDMO business. The Company continues to expect the organic revenue growth rates for the DSA and RMS segments will be in line with the initial outlooks for the year.

The Company is also reducing GAAP and non-GAAP earnings per share guidance primarily to reflect the headwinds from the CDMO business and foreign exchange, as well as higher interest expense. These factors will be partially offset by discretionary cost controls.

The Company’s updated guidance for revenue growth, earnings per share, and cash flow is as follows:

Footnotes to Guidance Table:

(1) Organic revenue growth is defined as reported revenue growth adjusted for completed acquisitions and divestitures, the 53rd week in 2022, and foreign currency translation.
(2) These adjustments are related to the evaluation and integration of acquisitions and divestitures, and primarily include transaction, advisory, certain third-party integration costs, and certain costs associated with acquisition-related efficiency initiatives, offset by adjustments related to contingent consideration and certain indirect tax liabilities.
(3) Venture capital and other strategic investment performance only includes recognized gains or losses. The Company does not forecast the future performance of these investments.
(4) These items primarily relate to charges associated with U.S. and international tax legislation that necessitated changes to the Company’s international financing structure; certain third-party legal costs related to (a) environmental litigation related to the Microbial Solutions business and (b) responses to a U.S. government industry-wide supply chain management inquiry applicable to our Safety Assessment business; and severance and other costs related to the Company’s efficiency initiatives.

Webcast

Charles River has scheduled a live webcast on Wednesday, August 3rd, at 9:30 a.m. ET to discuss matters relating to this press release. To participate, please go to ir.criver.com and select the webcast link. You can also find the associated slide presentation and reconciliations of GAAP financial measures to non-GAAP financial measures on the website.

Non-GAAP Reconciliations

The Company reports non-GAAP results in this press release, which exclude often-one-time charges and other items that are outside of normal operations. A reconciliation of GAAP to non-GAAP results is provided in the schedules at the end of this press release.

On August 3, 2022 Celdara Medical, LLC reported that the National Cancer Institute of the National Institutes of Health (NIH) has awarded a Small Business Innovation Research (SBIR) Phase I award to fund the company’s proof of concept studies on a new platform to improve chimeric antigen receptor (CAR) T cells efficacy to treat solid tumors (Press release, Celdara Medical, AUG 3, 2022, View Source [SID1234617365]).

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Dr. Joana Murad-Mabaera, who leads this effort, stated, "CAR T-based cellular products have been very effective against liquid tumors. However, the impact on solid tumors has been far below the expectations set in hematologic malignancies, largely due to the immunosuppressive nature of the tumor microenvironment. This approach, with Dr. Huang, supports and protects CAR T cells in this environment and ultimately improves their efficacy in solid cancers. This is a major unmet need."

Dr. Jake Reder, Co-founder and Chief Executive Officer of Celdara Medical, added, "Celdara has been innovating in the CAR T space longer than just about any other company, and we’ve collaborated with leading academicians and public companies to bring those innovations to patients. This work will advance a platform with the potential help many more solid tumor patients become responders, and ultimately, survivors. This is our mission and our passion."

Dr. Yina Huang, Associate Professor of Microbiology and Immunology at Dartmouth’s Geisel School of Medicine and Dartmouth Cancer Center, stated, "We are truly excited for this opportunity to partner with Celdara Medical to expand CAR T cell treatment to patients with solid tumors. Generally, academics like me disseminate our research through publications, but NIH small business grants allow us to continue to advance our discoveries towards improving patient health by working directly with research and business experts in biotech."

Research reported in this press release is supported by the National Cancer Institute of the National Institutes of Health under award number RCA271880A. The content is solely the responsibility of Celdara Medical and does not necessarily represent the official views of the National Institutes of Health.

On August 3, 2022 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2022 (Press release, BioMarin, AUG 3, 2022, View Source [SID1234617364]).

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BioMarin Announces Record Revenues in Second Quarter 2022; Increases Full-year 2022 Top and Bottom-line Guidance
"Continued strong growth of VOXZOGO and solid contributions from our other franchises drove record revenues exceeding $1 billion in the first half of the year, leading us to increase our full-year 2022 top and bottom-line guidance," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We also achieved many other significant milestones in the second quarter, including the CHMP’s positive opinion for conditional marketing authorization of Roctavian, the first gene therapy to be recommended for approval in Europe for hemophilia A. Assuming a positive decision from the European Commission, BioMarin’s commercial organization is ready to launch Roctavian in Europe in the third quarter. Based on the updated multi-year hemostatic efficacy observed to date following treatment with a single intravenous administration of Roctavian, we are on-track to resubmit the BLA in the U.S. to the FDA this September."

Mr. Bienaimé added, "In addition to continued robust demand for Voxzogo throughout the United States and Europe, underscored by today’s significant increase in full-year 2022 guidance for Voxzogo net product revenues to between $130 million and $160 million, we were thrilled to receive commercial approval in both Japan and Australia in the quarter. As we said previously, in 2022 we expect to return to double-digit revenue growth and profitability, and we are tracking to plan as demonstrated by record revenues in both the first and second quarters of this year."

Financial Highlights:

Total Revenues for the second quarter of 2022 were $533.8 million, an increase of 6% compared to the same period in 2021 despite continued erosion of the U.S. Kuvan market. The increase in Total Revenues was primarily attributed to the following:
Voxzogo commercial sales due to new patients initiating therapy in Europe and the U.S. following regulatory approvals by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) in the third and fourth quarters of 2021, respectively.
Higher Aldurazyme product revenues due to timing of product fulfillment to Sanofi. BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Sanofi; partially offset by
Lower Kuvan product revenues primarily due to generic competition as a result of the loss of exclusivity in the U.S., consistent with expectations.
GAAP Net Income and Non-GAAP Income increased by $14.8 million and $11.6 million, respectively, for the second quarter of 2022 compared to the same period in 2021. The increase was primarily related to higher revenues partially offset by higher sales and marketing expenses to support the commercial launch of Voxzogo and pre-commercialization activities for Roctavian.
New Product Launches and Anticipated Approvals (Voxzogo and Roctavian)

The global launch of Voxzogo is actively underway, with market access and reimbursement progressing as anticipated. As of June 30, 2022, there were an estimated 446 children being treated with commercial Voxzogo globally, as compared to an estimated 284 children as of March 31, 2022. Of the estimated 446 children being treated with Voxzogo as of the end of the second quarter, 282 were from countries outside the U.S. and 164 were from the U.S. As of June 30, 2022, there were 20 active markets contributing to Voxzogo sales.
During the quarter, marketing authorization for Voxzogo was approved in both Japan and Australia, with commercial sales anticipated in these markets to begin in the third quarter of 2022. Japan accounts for approximately half of the 1,500 patient opportunity in the Asia-Pacific region.
During the quarter, the Company provided a top-line update on the Phase 2 randomized, double-blind, placebo-controlled Voxzogo study in infants and young children up to five years of age with achondroplasia at the 2022 Endocrine Society Annual Meeting (ENDO). BioMarin intends to initiate discussions on the favorable results from the study with regulatory health authorities to discuss next steps regarding efforts to expand access to Voxzogo treatment for this younger age group.
In the quarter, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending conditional marketing authorization for Roctavian, for adults with severe hemophilia A. The Company expects a final approval decision, which is typically consistent with the CHMP recommendation, from the European Commission in the third quarter of 2022.
BioMarin is targeting a BLA resubmission for Roctavian by the end of September 2022. Typically, BLA resubmissions are followed by a 6-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.
In July, at the International Society on Thrombosis and Haemostasis 2022 Congress, the Company reported that durable hemostatic efficacy was maintained over six years in the ongoing Phase 1/2 study with Roctavian in the 6e13vg/kg dose cohort, representing the longest duration of clinical observation with any gene therapy treatment for adults with severe hemophilia A.
Mid-stage Product Life Cycle Expansion Opportunities (Voxzogo and Roctavian)

Also at the ENDO meeting, the investigator-initiated study with Voxzogo in children with selected genetic causes of short stature, preliminary 6-month results from 12 subjects demonstrated a positive response in all subgroups with interindividual variability. The 52-week study is ongoing, and is expected to complete in 2023.
In the quarter, the Company’s interventional Phase 2 study with Voxzogo for the treatment of infants under the age of two who are at risk for foramen magnum compression completed enrollment. The study is investigating the safety of Voxzogo in infants at risk of requiring surgery to alleviate compression at the foramen magnum, the opening in the base of the skull through which the spinal cord passes. The study will also measure a secondary endpoint to evaluate the effect of Voxzogo on growth of the foramen magnum volume through MRI scans.
Product expansion opportunities with Roctavian are supported by a number of clinical studies currently underway. The Phase 3b study to evaluate Roctavian with prophylactic corticosteroids has completed enrollment and is expected to read-out in early 2023. Two additional studies, one investigating Roctavian treatment in those with active or prior inhibitors, as well as one study investigating Roctavian in people with pre-existing antibodies against AAV5, are actively recruiting at sites around the world.
Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001))

BMN 255 for primary hyperoxaluria type 1, a subset of chronic renal disease: The Company was recently given permission by the FDA to move forward with the multiple ascending dose portion of the First-in-Human study with BMN 255. BioMarin believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in certain people with chronic renal disease.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): The Company announced that it has dosed patients in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE.
BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling studies continue with BMN 351, an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, upstream, splice enhancer site demonstrating improved binding affinity and tolerability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation. BioMarin expects to file an IND for BMN 351 in the winter.
BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that is titratable with rapid onset and high potency and efficacy. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. BioMarin’s goal is to file an IND for BMN 349 in the second half of 2023.
BMN 293 (formerly DiNA-001) for MYBPC3 hypertrophic cardiomyopathy (HCM): Preclinical studies are underway with BMN 293 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. Mutations in MYBPC3 are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize relaxation kinetics and potentially ameliorate the disease phenotype in individuals suffering from cardiomyopathy. BioMarin’s goal is to file an IND for BMN 293 in 2023.

All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the Company’s Non-GAAP financial information and reconciliations to the corresponding GAAP reported information.

BioMarin will host a conference call and webcast to discuss second quarter and year to date 2022 financial results today, Wednesday, August 3, 2022 at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

On August 3, 2022 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported Scott Hutton, Chief Executive Officer of Biodesix, will present at the Canaccord Genuity 42nd Annual Growth Conference being held August 8-11, 2022 (Press release, Biodesix, AUG 3, 2022, View Source [SID1234617363]).

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Canaccord Genuity 42nd Annual Growth Conference
Date: Wednesday, August 10, 2022
Time: 3:00 PM ET
Location: InterContinental Boston

The presentation will be webcast live and available for replay under "News & Events" in the Investors section of the Company’s website at www.biodesix.com.