On May 8, 2023 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a commercial stage biotechnology company, and Sarah Cannon Research Institute (SCRI), one of the world’s leading oncology research organizations conducting community-based clinical trials, reported a strategic partnership aimed at increasing diversity in clinical study recruitment practices (Press release, Mirati, MAY 8, 2023, View Source [SID1234631189]).

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According to the American Association for Cancer Research (AACR) (Free AACR Whitepaper), nearly 85 percent of patients living with cancer are treated in community centers1. Ensuring equitable access to and participation in clinical studies requires improved awareness and targeted efforts to remove systemic barriers that may limit access to clinical studies for underrepresented patient groups.

As part of this partnership, Mirati and SCRI will implement practices and programs focused on removing obstacles to clinical study participation, including expanding physician and patient education and increasing access to community-based clinical trials. Initiatives will be focused on reducing complexities associated with activating clinical trials thereby enabling a greater number of community practices to participate in research studies.

"The opportunity to partner with SCRI enables Mirati to advance diversity in our clinical studies, which is a key pillar of our Diversity, Equity, and Inclusion efforts. We are excited about the opportunity to leverage the expertise and scale of SCRI as we collaborate on a range of initiatives aimed at improving health outcomes for a broad range of patients." said Alan Sandler, M.D., chief medical officer, Mirati Therapeutics, Inc. "By focusing on community-based sites, we can better understand the health needs of patients living with cancer and provide more tailored, patient-centered care. This partnership will allow us to gain a deeper understanding of the challenges patients face to enable us to better design our clinical studies to be representative of all patient communities."

SCRI’s research network brings together more than 1,300 physicians who are actively accruing patients to clinical trials at more than 250 locations in 24 states across the U.S. Mirati will work directly with Development Innovations, SCRI’s full-service, oncology-focused contract research organization (CRO), to execute the collaboration initiatives across the SCRI network. Through SCRI’s Personalized Medicine Program, the partnership will utilize robust clinical trial matching data to better understand barriers to trial participation and ultimately design protocols to address challenges.

"SCRI is uniquely positioned to advance diversification strategies given our national reach, physician expertise and personalized medicine capabilities," said Howard A. "Skip" Burris, III, MD, president, SCRI. "Through our collaboration with Mirati, we can advance our goals to design and deliver clinical trials that can be accessed by more patient populations and accelerate drug development."

On May 8, 2023 Citius Pharmaceuticals Inc. (Nasdaq: CTXR) ("Citius" or the "Company"), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, reported the closing of its previously announced registered direct offering with certain healthcare-focused and institutional investors for the purchase of an aggregate of 12,500,001 shares of its common stock and accompanying warrants to purchase up to an aggregate of 12,500,001 shares of its common stock, at a purchase price of $1.20 per share and accompanying warrant.

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The warrants have an exercise price of $1.50 per share, are exercisable six months from the date of issuance, and will expire five years from the date of issuance.

The aggregate gross proceeds to the Company from the offering were approximately $15 million, before deducting the placement agent fees and other offering expenses payable by the Company. Citius currently intends to use the net proceeds from the offering for general corporate purposes, including pre-clinical and clinical development of its product candidates and working capital and capital expenditures.

The securities described above were offered pursuant to a "shelf" registration statement (File No. 333-255005) filed with the Securities and Exchange Commission (SEC) and declared effective on April 16, 2021. The offering was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and the accompanying prospectus relating to the securities offered was filed with the SEC and is available at the SEC’s website at www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying prospectus relating to the securities offered may also be obtained by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by calling (646) 975-6996 or emailing [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

On May 8, 2023 Astrazeneca reported that Koselugo (selumetinib) has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above (Press release, AstraZeneca, MAY 8, 2023, View Source [SID1234631187]).

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The approval by the National Medical Products Administration (NMPA) in China was based on positive results from the SPRINT Stratum 1 trial sponsored by the National Institutes of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). The trial showed Koselugo, an oral treatment option, reduced the size of inoperable tumours in children.1,2

NF1 is a rare, progressive genetic condition affecting one in 3,000 individuals worldwide, most commonly diagnosed in children under the age of 10.3,4 In 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas) and cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.2,5-8

Li Qingfeng, Vice President of Shanghai Ninth People’s Hospital, JiaoTong University School of Medicine, Chairman of Department of Plastic and Reconstructive Surgery, said: "Children living with NF1 PN may face physical challenges and significant disruption to their daily lives, and early intervention is essential as tumour growth is often progressive and rapid, especially in the first decade of life. The approval of Koselugo in China has the potential to change the treatment trajectory of this debilitating genetic condition, providing a new option for addressing inoperable tumours by targeting the underlying cause of tumour growth through MEK inhibition."

Marc Dunoyer, Chief Executive Officer, Alexion, said: "Koselugo offers hope for children whose quality of life and overall well-being is impacted by the growth of painful, debilitating tumours throughout the body. We are proud to bring forward a new, innovative treatment option to the NF1 community in China, delivering on Alexion’s commitment to reach more people living with rare diseases in China, and transform the lives of patients and caregivers across the globe."

Results from the SPRINT Stratum 1 Phase II trial were published in The New England Journal of Medicine and showed that Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial responses) in paediatric patients with PNs in NF1 when treated with Koselugo as twice-daily oral monotherapy.1,2 ORR is defined as the percentage of patients with confirmed complete (disappearance of PNs) or partial response (at least 20% reduction in tumour volume).1 The most common adverse reactions in the SPRINT trial were vomiting, blood creatine phosphokinase increase, diarrhoea and nausea.1

In addition to China, Koselugo is also approved in the US, EU, Japan and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PNs.

Notes

NF1
NF1 is a debilitating genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.9 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots) and, in 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas).5,9 These plexiform neurofibromas (PNs) can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.2,5-8 PNs begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.5,8-10

SPRINT
The SPRINT Stratum 1 Phase II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with Koselugo (selumetinib) monotherapy.2 This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).

Koselugo
Koselugo (selumetinib) is the first and only approved therapy by China’s NMPA for the treatment of symptomatic, inoperable PNs in paediatric patients with NF1 aged three years and above.1 Koselugo blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow.1 In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way. By blocking these enzymes, Koselugo slows down the growth of tumour cells.1

Koselugo is approved for use in the US, EU, Japan and China and has received Orphan Drug Designation in the US, EU, Russia, Switzerland, South Korea, Taiwan, Japan and Australia, and health authorities worldwide are reviewing regulatory submissions.

On May 8, 2023 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the first quarter of 2023 (Press release, Y-mAbs Therapeutics, MAY 8, 2023, View Source [SID1234631185]).

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"The first quarter of 2023 marked a period of significant progress for DANYELZA and we believe has set up 2023 to be a very productive year. We are thrilled to report record DANYELZA net revenues of $20.3 million in the first quarter of 2023, a 24% sequential increase compared to the previous quarter and near doubling year-over-year. The increase allows us to update our FY 2023 DANYELZA revenue guidance from $60-65 million to $80-85 million," said Thomas Gad, President, and Interim Chief Executive Officer. "In Q1 2023, we implemented a restructuring plan to prioritize resources on the DANYELZA franchise and development of our SADA technology in the fight against cancer. With a 35% reduction in force and an anticipated 28% reduction in annual operating expenses for 2023, we emerge leaner and supported by a robust balance sheet with $92.6 million in cash and cash equivalents as of March 31, 2023, which we estimate should support our business operations as currently planned into the first quarter of 2026. In addition, we achieved a major milestone of treating the first patient in our GD2-SADA trial in April 2023."

First Quarter 2023 and Recent Corporate Developments

● On April 18, 2023, Y-mAbs announced that positive preclinical data on naxitamab in triple-negative breast cancer was presented at the AACR (Free AACR Whitepaper) Annual Meeting
● On April 5, 2023, Y-mAbs announced that the first patient had been dosed in the Phase 1 trial of GD2-SADA
● On February 2, 2023, Y-mAbs announced that the European Medicines Agency agreed to the Company’s Pediatric Investigational Plan for naxitamab.
● On January 4, 2023, Y-mAbs announced a restructuring plan including a 35% reduction in workforce and an anticipated 28% reduction in annual operating expenses for 2023.

Financial Results

Revenues

Y-mAbs reported DANYELZA net product revenues of $20.3 million for the quarter ended March 31, 2023, which reflects a 93% increase over $10.5 million in the comparable quarter of 2022. The DANYELZA net product revenues of $20.3 million for the quarter ended March 31, 2023 represents an increase of 24% compared to the fourth quarter 2022 DANYELZA net product revenues of $16.4 million. The $3.9 million sequential increase was primarily driven by an increase in new US patients and an incremental benefit from international revenues. Our net product revenues from other countries for the three months ended March 31, 2023 included $2.5 million of product revenue from our distribution partner in connection with the early access program for DANYELZA in Europe. These sales reflected an initial inventory stocking and we do not anticipate recurring sales under this program to be as high in future quarters, which is reflected in our updated revenue guidance for 2023.

As of March 31, 2023, Y-mAbs has delivered DANYELZA to 53 centers across the United States, corresponding to a sequential increase of 10% in the number of centers since the fourth quarter of 2022. During the first quarter of 2023, approximately 62% of the vials sold in the United States were sold outside Memorial Sloan Kettering ("MSK"), an increase from the prior quarter as a result of growth in new patients at institutions outside MSK outpacing MSK’s growth of new patients.

Operating Expenses

Research and Development

Research and development expenses were $13.4 million for the three months ended March 31, 2023, compared to $22.9 million for the three months ended March 31, 2022. The $9.5 million decrease was primarily due to the decreased spending on deprioritized programs, which resulted in decreased costs for outsourced manufacturing services due to decreased clinical trial activities in 2023, outsourced research and supplies and spending for clinical trials, partially offset by increased personnel-related costs, inclusive of stock-based compensation, which includes an incremental impact for accrued severance related benefits of $1.1 million and accelerated stock-based compensation expense of $0.9 million associated with our first quarter of 2023 restructuring charge in connection with the implementation of our restructuring plan.

Selling, General, and Administration

Selling, general, and administrative expenses decreased by $1.2 million to $12.2 million for the three months ended March 31, 2023, compared to $13.4 million for the three months ended March 31, 2022. The decrease in selling, general and administrative expenses was primarily the result of decreased personnel-related costs and insurance costs. The decrease in personnel-related costs is after $0.8 million accelerated stock-based compensation expense related to our reduction in force.

Net Loss

We reported a net loss for the first quarter ended March 31, 2023, of ($6.4) million, or ($0.15) per basic and diluted share, compared to a net loss of ($28.1) million, or ($0.64) per basic and diluted share, for the quarter ended March 31, 2022. The favorable improvement in net loss compared to 2022 was primarily driven by increased DANYELZA revenues and decreased research and development expenses, partially offset by a $2.8 million incremental impact of the restructuring charge in the first quarter of 2023.

Cash and Cash Equivalents

We had approximately $92.6 million in cash and cash equivalents as of March 31, 2023, and we expect a full-year 2023 cash burn of $40-50 million. Our existing cash and cash equivalents, when combined with anticipated DANYELZA revenues, which are assumed to increase by 10% for 2024 and 2025 for the purpose of our analysis of runway, is expected to be sufficient to fund our operations as currently planned into 2026. In terms of development activities, we have assumed that our prioritized programs will be advanced at our own expense and no new programs are assumed at this point. We assume no new partnerships or other new business development, and no further development of the omburtamab program. This estimate reflects our current business plan that is supported by assumptions that may prove to be inaccurate, such that we could use our available capital resources sooner than we currently expect.

Financial Guidance

We are updating our full-year 2023 financial guidance, which now reflects:

● Anticipated DANYELZA net product revenues now expected to be $80-85 million (previously $60-65 million);
● Anticipated operating expenses continues to be expected to be $115-120 million;
● Anticipated total annual cash burn now expected to be $40-50 million (previously $50-55 million); and
● Cash and cash equivalents anticipated to support operations as currently planned into the first quarter of 2026.

Webcast and Conference Call

Y-mAbs will host a conference call on Tuesday, May 9, 2023, at 9 a.m. Eastern Time. To participate in the call, please use the following dial-in information.

Investors (domestic): 888-886-7786
Investors (international): 416-764-8658
Conference ID: 09065062

To access a live webcast of the update, please use this link.

On May 8, 2023 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies and cytokines for the treatment of patients with cancer and autoimmune diseases, reported financial results for the first quarter ended March 31, 2023 and provided a review of recent business and clinical highlights (Press release, Xencor, MAY 8, 2023, View Source [SID1234631184]).

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"In the first quarter, we and our partners continued to enroll patients across multiple Phase 1 and Phase 2 clinical studies of XmAb drug candidates in oncology and autoimmune diseases. Two new programs are planned to advance into clinical development this year, XmAb662, our engineered IL12-Fc cytokine, on track to start a Phase 1 study mid-year, and XmAb541, our CLDN6 x CD3 bispecific antibody for ovarian cancer, for which we anticipate submitting the IND by year end," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "And recently, we added to our team the expertise and talents of Nancy Valente, M.D., who will lead our clinical programs as our new executive vice president and chief development officer."

Program and Corporate Updates

XmAb564 (IL2-Fc): The Company will present updated results from the Phase 1a single-ascending dose study in healthy volunteers at European Alliance of Associations for Rheumatology (EULAR) Congress, being held from May 31 to June 1, in Milan, Italy. The update will include additional biomarker data. Data previously presented demonstrate a single dose, administered subcutaneously in healthy volunteers, was well tolerated and generated durable, dose-dependent and selective expansion of regulatory T cells.
XmAb104 (PD-1 x ICOS): XmAb104 is a bispecific antibody that targets PD-1, an immune checkpoint receptor, and ICOS, an immune co-stimulatory receptor, to selectively activate the tumor microenvironment. Initial dose-escalation data from a Phase 1 study, presented at ASCO (Free ASCO Whitepaper) 2022, indicates that XmAb104 was well tolerated and exhibited a distinct safety profile compared to other clinical-stage ICOS programs. Anti-tumor activity was observed in patients, and biomarker activity was consistent with engagement with T cells. The Company has opened an expansion portion in the study (Part C) to evaluate XmAb104 in combination with ipilimumab in patients with microsatellite stable or proficient mismatch repair colorectal cancer.
Preclinical Data Presentation: Preclinical data generated from engineered CD28 bispecific antibodies targeting the solid tumor antigens CEACAM5, ENPP3, mesothelin, STEAP1 and Trop-2 were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023.
Corporate Update: Nancy Valente, M.D., was appointed as Xencor’s Executive Vice President, Chief Development Officer, effective May 1, having previously served as an independent member of the Company’s Board of Directors from September 2022 through April 2023. Dr. Valente brings with her more than 20 years of experience in late-stage biopharmaceutical product development. She most recently served as a senior vice president at Genentech, a member of the Roche Group, and as its global head and co-lead of global product development of its oncology and hematology therapeutic area.
Progress Across Partnered Programs

Alexion Pharmaceuticals, Inc.: In April 2023, Ultomiris (ravulizumab-cwvz), which incorporates Xencor’s Xtend Fc domain, was recommended for marketing authorization in the EU for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 antibody positive. In addition, AstraZeneca recently announced that a Phase 3 study of Ultomiris has been initiated in cardiac surgery-associated acute kidney injury. In the first quarter of 2023, Xencor earned $10.5 million of royalty revenue from Alexion on net sales of Ultomiris.
Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

Financial Results for the First Quarter Ended March 31, 2023

Cash, cash equivalents, receivables and marketable debt securities totaled $568.2 million as of March 31, 2023, compared to $613.5 million on December 31, 2022.

Revenues for the first quarter ended March 31, 2023 were $19.0 million, compared to $85.5 million for the same period in 2022. Total revenues earned in the first quarter of 2023 included milestone revenue earned from Xencor’s Janssen collaboration and royalties from the Alexion agreement, compared to revenue earned from the Janssen collaboration, milestone revenue from Astellas, and royalties from the Alexion, MorphoSys and Vir agreements in the first quarter of 2022.

Research and development expenses for the first quarter ended March 31, 2023 were $64.4 million, compared to $47.8 million for the same period in 2022. Increased research and development spending for first quarter of 2023 compared to 2022 reflects increased spending on the Company’s development programs including XmAb541 and XmAb564 and other research and early-stage programs.

General and administrative expenses for the first quarter ended March 31, 2023 were $14.0 million, compared to $11.3 million in the same period in 2022. Increased general and administrative spending for the first quarter of 2023 compared to 2022 reflects increased facility expenses and additional spending on professional fees.

Non-cash, stock-based compensation expense for the first quarter ended March 31, 2023 was $12.6 million, compared to $10.8 million for the same period in 2022.

Net loss for the first quarter ended March 31, 2023 was $60.8 million, or $(1.02) on a fully diluted per share basis, compared to a net income of $23.6 million, or $0.39 on a fully diluted per share basis, for the same period in 2022. Net loss reported for the first quarter of 2023 compared to net income for the same period in 2022 is primarily due to decreased royalties from the Vir agreement.

The total shares outstanding were 60,381,600 as of March 31, 2023, compared to 59,529,192 as of March 31, 2022.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations through the end of 2025. The Company expects to end 2023 with between $425 million and $475 million in cash, cash equivalents, receivables and marketable debt securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss the first quarter 2023 financial results and provide a corporate update.

The live webcast will be available under "Events & Presentations" in the Investors section of the Company’s website located at investors.xencor.com and will be archived for at least 30 days. Active participants in the conference call may receive credentials for telephone access by registering at the following link: https://register.vevent.com/register/BI0da076297f5f4f3b920845d8b0e7f2d3.