On July 29, 2021 Blueprint Medicines Corporation (NASDAQ:BPMC) reported financial results and provided a business update for the second quarter ended June 30, 2021 (Press release, Blueprint Medicines, JUL 29, 2021, View Source [SID1234585415]).

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"In the second quarter, we achieved one of our foundational corporate goals, securing U.S. approval of AYVAKIT for advanced systemic mastocytosis, and the launch of this therapy is off to a great start with broad prescriber demand," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In addition, we continued to progress our expanding clinical portfolio with the initiation of the Phase 1/2 trial of BLU-945, the initiation of the Phase 2/3 HARBOR trial of BLU-263 and the announcement of a strategic research collaboration with MD Anderson to accelerate the development of BLU-222, all of which exemplify the potential impact of our next wave of therapeutic candidates. This commercial and clinical progress, combined with our financial strength, provides a robust foundation for future growth as we look to address the needs of many more patients with cancer and hematologic disorders."

Second Quarter 2021 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): systemic mastocytosis (SM) and gastrointestinal stromal tumor (GIST)

Recorded $8.5 million in net product revenue during the second quarter of 2021 for AYVAKIT/AYVAKYT, which was approved by the U.S. Food and Drug Administration (FDA) in June 2021 for the treatment of adult patients with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL). Read the press release announcing the expansion of the FDA label for AYVAKIT into advanced SM here. AYVAKIT received its initial approval from the FDA in 2020 for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. Please click here to see the full Prescribing Information for AYVAKIT and visit www.AYVAKIT.com for more information on AYVAKIT.
GAVRETO (pralsetinib): RET-altered cancers

Recorded $2.9 million in net product revenue during the second quarter of 2021 for GAVRETO, which was approved by the FDA in September 2020 for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test and in December 2020 for the treatment of patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer. Blueprint Medicines is commercializing GAVRETO in the U.S. together with Genentech, Inc., a member of the Roche Group. Please click here to see the full Prescribing Information for GAVRETO and visit www.GAVRETO.com for more information on GAVRETO.
Reported updated data from the Phase 1/2 ARROW clinical trial of GAVRETO in metastatic RET fusion-positive NSCLC and other advanced solid tumors at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data showed high response rates to GAVRETO in treatment-naïve patients with RET fusion-positive NSCLC, clinical activity across a number of RET fusion-positive tumor types and a safety profile consistent with previously reported results. Read the press release here.
BLU-263: SM

Initiated the HARBOR trial, a randomized, double-blind, placebo-controlled Phase 2/3 trial in non-advanced SM.
BLU-945: treatment-resistant EGFR-driven NSCLC

Initiated the global Phase 1/2 trial of BLU-945 in patients with treatment-resistant EGFR-driven NSCLC.
BLU-222: Cyclin E-aberrant cancers

Announced a strategic research collaboration with The University of Texas MD Anderson Cancer Center focused on accelerating development of BLU-222, an investigational precision therapy designed to target cyclin-dependent kinase 2 (CDK2). Read the press release here.
Corporate:

Announced the appointment of Percy Carter, MBA, Ph.D., as Chief Scientific Officer. In this role, Dr. Carter oversees all research and preclinical development. Read the press release here.
Key Upcoming Milestones

The company expects to achieve the following near-term milestones:

Present preclinical data supporting combination of BLU-945 and BLU-701 in treatment-naïve EGFR-driven NSCLC in the second half of 2021.
Initiate a Phase 1 trial of BLU-701 in patients with treatment-resistant EGFR-driven NSCLC in the second half of 2021.
Initiate a Phase 1 trial of BLU-222, a CDK2 inhibitor targeting cyclin-E aberrant cancers, in the first half of 2022.
Disclose topline data for the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced systemic mastocytosis in mid-2022.
Second Quarter 2021 Financial Results

Revenues: Revenues were $27.3 million for the second quarter of 2021, including $8.5 million of net product revenues from sales of AYVAKIT/AYVAKYT, $2.9 million of net product sales from GAVRETO and $15.9 million in collaboration revenues. Blueprint recorded revenues of $8.3 million in the second quarter of 2020, including $5.7 million of net product revenues from sales of AYVAKIT and $2.7 million in collaboration revenues. The increase in net product revenues was driven by increased sales quantity and the increase in collaboration revenues was primarily driven by the sales of drug substance and drug product to our collaboration partners.
Cost of Sales: Cost of sales was $6.5 million for the second quarter of 2021, as compared to $0.1 million for the second quarter of 2020. Cost of sales includes manufacturing costs associated with our products sales as well as costs associated with the sale of drug product to our collaboration partners. The increase in costs of product sales was primarily driven by the lower margin product sales to our collaboration partners during the second quarter of 2021.
R&D Expenses: Research and development expenses were $80.0 million for the second quarter of 2021, as compared to $91.1 million for the second quarter of 2020. This decrease was primarily due to decreased expenses associated with clinical supply manufacturing activities and reimbursement from the global development cost sharing arrangement under our collaboration with Roche for pralsetinib, partially offset by increased costs related to early discovery activities. Research and development expenses included $10.5 million in stock-based compensation expenses for the second quarter of 2021.
SG&A Expenses: Selling, general and administrative expenses were $49.3 million for the second quarter of 2021, as compared to $42.2 million for the second quarter of 2020. This increase was primarily due to increased costs associated with building our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT and GAVRETO, partially offset by reimbursement under our collaboration with Roche for pralsetinib. General and administrative expenses included $13.8 million in stock-based compensation expenses for the second quarter of 2021.
Net Loss: Net loss was $108.4 million for the second quarter of 2021, or a net loss per share of $1.86, as compared to a net loss of $123.5 million for the second quarter of 2020, or a net loss per share of $2.28.
Cash Position: As of June 30, 2021, cash, cash equivalents and investments were $1,380.1 million, as compared to $1,549.7 million as of December 31, 2020.
Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss second quarter 2021 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or +44-208-0682-558 (international) and referring to conference ID 619159. A webcast of the call will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

Upcoming Investor Conference

Blueprint Medicines will participate in the 2021 Wedbush Pacgrow Healthcare Virtual Conference on Tuesday, August 10, 2021. Christina Rossi, Chief Commercial Officer, will participate in a panel discussion, "Building Back a Better Commercial Infrastructure – Selling in COVID Times," beginning at 8:35 a.m. ET. A live webcast of the panel discussion will be available by visiting the Investors & Media section of Blueprint Medicines’ website at View Source A replay of the webcast will be archived on Blueprint Medicines’ website for 30 days following the presentation.

On July 29, 2021 KORTUC Inc., a Japanese clinical-stage biopharmaceutical company focusing on the clinical and product development of a novel radiosensitizer improving the effectiveness of cancer radiotherapy (RT), reported the closing of an equity investment from AXA Japan (Press release, KORTUC, JUL 29, 2021, View Source [SID1234585478]).

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KORTUC will use the financing to fund Research and Development activities, accelerate its Pivotal Phase II Study in the UK and pursue additional Pivotal Studies in the US. Currently ongoing Pivotal Phase II Study focuses on application of Kortuc radiosensitizer to locally advanced/recurrent breast cancer patients undergoing RT.

Comments from KORTUC
"We are pleased to share our vision with and receive such strong support from our new investor as we are accelerating our clinical research in the UK and the US to bring to cancer patients globally a product that can make a true difference in the near future – we firmly believe that Kortuc radiosensitizer can change the paradigm for use of radiotherapy across cancer types and stages" highlighted Kazu Matsuda, Chief Executive Officer of KORTUC.

Comments from AXA Japan
"As a worldwide leader in insurance and asset management, our purpose is to act for human progress by protecting what matters. As new cancer patient growth accelerates, this investment illustrates our purpose to support innovative technologies that can serve humanity." Jean-Baptiste Tricot, Director, Executive Officer and Chief Financial Officer of AXA Japan commented.
Boris Moutier, Corporate Officer of AXA Japan, Japan and Asia Chief Investment Officer, also highlights that "With this brand new radiosensitizer, KORTUC has great potential to become a ground-breaking player in cancer treatment field, and we are glad to engage in this greatly promising journey. Aligned with AXA Group’s vision, AXA Japan pursues investments into startups that may create innovations and produce tangible results."

On July 29, 2021 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and neuropsychiatric disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended June 30, 2021 (Press release, Corcept Therapeutics, JUL 29, 2021, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-second-quarter-financial-results [SID1234585367]).

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Financial Results

Revenue of $91.6 million, compared to $88.6 million in second quarter 2020
GAAP diluted net income of $0.21 per share, compared to $0.23 per share in second quarter 2020
Non-GAAP diluted net income of $0.30 per share, compared to $0.32 per share in second quarter 2020
Cash and investments of $471.6 million, compared to $454.8 million at March 31, 2021
Reiterated 2021 revenue guidance of $355 – $385 million
Second quarter 2021 revenue was $91.6 million, compared to $88.6 million in the second quarter of 2020. First quarter 2021 revenue was $79.4 million. Second quarter 2021 GAAP net income was $26.5 million, compared to $28.3 million in the second quarter of 2020. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the second quarter was $38.2 million, compared to $39.7 million in the second quarter of 2020. A reconciliation of GAAP to non-GAAP net income is included below.

Second quarter operating expenses were $59.6 million, compared to $53.3 million in the second quarter of 2020, due to increased employee compensation expenses, commercial spending, spending on clinical trials in Cushing’s syndrome and pre-clinical activities.

Cash and investments were $471.6 million at June 30, 2021, compared to $454.8 million at March 31, 2021. The balance at June 30, 2021 reflects the acquisition of $30.8 million of common stock in the second quarter – 1.4 million shares pursuant to the company’s stock repurchase program and 0.1 million shares in connection with the net exercise of stock options. Under the repurchase program’s current terms, $127.6 million remains available for the purchase of shares.

The company reiterated its 2021 revenue guidance of $355 – $385 million.

"Diminishing COVID-19 public health restrictions and the availability of safe and effective vaccines have allowed our commercial business to resume growing," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Our second quarter results reflect the fact that physicians are seeing their patients more frequently, which allows them to diagnose and optimally treat those who have Cushing’s syndrome. Provided COVID-19 restrictions continue to abate – as we believe they will – we expect growth to continue. Korlym is an excellent treatment for hypercortisolism and there are many eligible patients who have yet to receive it. Our planned successor to Korlym, relacorilant, which is currently in its pivotal trial, promises to be an even better medication."

Clinical Development

"We are enthusiastic about the potential of cortisol modulation to help treat many diseases, not just Cushing’s syndrome," added Dr. Belanoff. "Our programs in solid tumors, non-alcoholic steatohepatitis (NASH) and antipsychotic-induced weight gain (AIWG) continue to make progress. We are especially excited about our advancing platinum-resistant ovarian cancer program. Based on the statistically significant and clinically meaningful results of our 178-patient, controlled Phase 2 trial, we plan to initiate a pivotal trial in the first quarter of next year."

Solid Tumors

Phase 3 pivotal trial in patients with platinum-resistant ovarian cancer to start in the first quarter of 2022
Selection of the optimum dose of exicorilant plus enzalutamide in patients with castration-resistant prostate cancer (CRPC) expected later this year
Enrollment continues in 20-patient, open-label, Phase 1b trial of relacorilant plus PD-1 checkpoint inhibitor pembrolizumab in patients with adrenal cancer with cortisol excess
"We look forward to starting a Phase 3 pivotal trial in women with platinum-resistant ovarian cancer," said Andreas Grauer, MD, Corcept’s Chief Medical Officer. "Our Phase 2 results illustrated that women who received relacorilant with nab-paclitaxel experienced improved progression free survival (PFS) without experiencing additional side effects, a welcome finding for women with few good treatment options. We will present detailed results of our Phase 2 trial at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) meeting in September in a proffered paper oral presentation, with overall survival data expected later in the year. And we continue to evaluate relacorilant as a possible treatment for patients with other glucocorticoid receptor-expressing tumors."

Metabolic Diseases

Phase 1b dose-finding trial in patients with presumed NASH to start in the fourth quarter of 2021
Enrollment continues in GRATITUDE, a 100-patient double-blind, placebo-controlled, Phase 2 trial of miricorilant to reverse recent AIWG
Enrollment continues in GRATITUDE II, a 150-patient, double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse long-standing AIWG
"We plan to begin a Phase 1b, dose-finding trial of miricorilant in patients with presumed NASH in the fourth quarter of this year," said Dr. Grauer. "Patients who received miricorilant in our previous trial experienced exceptionally large and rapid reductions in liver fat accompanied by substantial, but transient, elevations of the liver enzymes ALT and AST. Our hypothesis is that the rapidity and magnitude of miricorilant’s fat reducing effect irritated the liver. The objective of our planned study is to identify a dosing regimen that can produce significant reductions in fat without causing liver irritation."

"Meanwhile, our Phase 2 trials evaluating miricorilant as a treatment for patients with AIWG – GRATITUDE and GRATITUDE II – continue to advance," added Dr. Grauer. "We expect to complete enrollment in GRATITUDE II by year-end and in GRATITUDE by mid-2022."

Cushing’s Syndrome

Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome; NDA submission expected in the second quarter of 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 based on its extremely promising Phase 2 efficacy and safety data, which we recently published in Frontiers in Endocrinology (Pivonello 2021)," said Dr. Grauer. "We expect our GRACE trial, which is accruing patients and generating data, to serve as the basis for relacorilant’s NDA in Cushing’s syndrome. We expect to submit this relacorilant NDA in the second quarter of 2023. The Phase 3 GRADIENT trial will produce valuable data about an etiology of Cushing’s syndrome that affects many patients, but has not been subject to rigorous, controlled study."

Conference Call

We will hold a conference call on July 29, 2021, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, Click this link (listen-only mode) or dial 1-833-693-0540 from the United States or 1-661-407-1581 internationally approximately 15 minutes before the start of the call. The passcode will be 1482035. A replay will be available on the Investors / Past Events tab of our website.

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 covering the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.

On July 29, 2021 AVEO Oncology (Nasdaq: AVEO), a commercial and clinical development stage biopharmaceutical company, reported that it will report second quarter 2021 financial results on Thursday, August 05, 2021 (Press release, AVEO, JUL 29, 2021, View Source [SID1234585383]). AVEO’s management team will host a conference call and audio webcast at 4:30 p.m. ET on Thursday, August 5, 2021, to discuss the financial results and provide a business update.

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The call can be accessed by dialing (844) 882-7841 (U.S. and Canada) or (574) 990-9828 (international). The passcode for the conference call is 3857963. To access the live webcast, or the subsequent archived recording, please visit the Calendar of Events sub-section within the Investors section of the AVEO website at www.aveooncology.com.

On July 29, 2021 CytRx Corporation (OTCQB: CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, reported that ImmunityBio, Inc. (NASDAQ: IBRX) ("ImmunityBio") has been granted a patent by the U.S. Patent and Trademark Office for its proprietary NANT Cancer Vaccine (U.S. Patent 11,071,774) (Press release, CytRx, JUL 29, 2021, View Source [SID1234585400]). ImmunityBio’s investigational treatment for cancer is designed to bolster a patient’s own immune response to cancerous cells, augment that response with additional natural killer and T-cell therapies to overcome the cancer’s resistance, and induce long-term T-cell memory to induce remission across multiple tumor types.

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As previously disclosed, CytRx out-licensed global development, manufacturing and commercialization rights for Aldoxorubicin to ImmunityBio in 2017. The Company has an agreement with ImmunityBio that can yield up to $343 million in potential milestone payments and single and double-digit royalties on sales of Aldoxorubicin.

To study the safety and early efficacy signals across multiple tumor types, ImmunityBio has launched a series of Quantitative Lifelong Trials (QUILT). To date, the vaccine has been studied in more than 100 patients across multiple tumor types, including pancreatic, breast, colorectal, and head and neck cancers. Among these studies is QUILT 88, a Phase 2 trial that includes Aldoxorubicin and is studying the vaccine in metastatic pancreatic cancer patients. Enrollment of Cohort C, which includes Aldoxorubicin and patients who have previously failed two lines of standard-of-care therapy, is expected to be completed in the third quarter of 2021 and an early readout of survival data is expected in the first quarter of 2022.

ImmunityBio’s Founder and Executive Chairman made the following statement this week:

"We are excited to be developing this orchestrated approach to activate as many elements of the immune system that we can and overcome cancer’s ability to evade the immune system. Our hypothesis is that by revealing tumor antigens to the immune system, we activate tumor-specific T cells and targeted natural killer cells to eradicate tumors by what is known as immunogenic cell death. The issuance of the NANT Cancer Vaccine patent is recognition of this innovative approach to therapy that not only potentially provides long-term immune system protection from cancer, it does so with a reduced risk of the toxicity risk that comes with using high-dose chemotherapy and radiation. This closely aligns with the FDA’s recent ‘Project Optimist’ guidelines for exploring lower doses of therapeutic agents."

Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:

"CytRx is very encouraged by ImmunityBio’s receipt of a U.S. patent for this novel immunotherapy vaccine that has been utilizing Aldoxorubicin in its pancreatic cancer trials. We continue to believe that Aldoxorubicin can be a foundational component of ImmunityBio’s innovative cancer treatments."