On December 28, 2020 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 that it has entered into a definitive agreement to sell its Priority Review Voucher ("PRV") to United Therapeutics Corporation (Nasdaq: UTHR), based on an agreed valuation of $105 million (Press release, Y-mAbs Therapeutics, DEC 28, 2020, View Source [SID1234573288]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The PRV was granted in conjunction with the approval by the U.S. Food and Drug Administration ("FDA") of DANYELZA, for the treatment of refractory/relapsed high-risk neuroblastoma.

Under the terms of the Company’s license agreement with Memorial Sloan Kettering Cancer Center ("MSK"), Y-mAbs is entitled to retain 60% of the net proceeds from monetization of the PRV, and the remaining 40% will be paid to MSK. The transaction remains subject to customary closing conditions, including anti-trust review.

"We are pleased to announce the sale of the PRV, which will provide an important source of non-dilutive capital to fund additional investment in our pipeline. These efforts will be critical to our growth over the coming year, and we are committed to our mission of becoming a world leader in developing better and safer antibody-based oncology products addressing unmet pediatric and adult medical needs," said Thomas Gad, founder, Chairman and President.

Jefferies LLC acted as exclusive financial advisor to Y-mAbs on this transaction.

Researchers at MSK developed DANYELZA, which is exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests related to the compound and Y-mAbs.

About DANYELZA (naxitamab-gqgk)

DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

On December 28, 2020 Hutchison China MediTech Limited ("Chi-Med") (Nasdaq/AIM: HCM) reported that it has initiated the filing of a New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") – the first portion of a rolling submission for surufatinib for the treatment of pancreatic and non-pancreatic neuroendocrine tumors ("NET") (Press release, Hutchison China MediTech, DEC 28, 2020, https://www.chi-med.com/chi-med-initiates-rolling-submission-of-nda-to-us-fda-for-surufatinib-for-the-treatment-of-advanced-net/ [SID1234573283]). Chi-Med plans to complete the NDA submission in the first half of 2021, which would be the company’s first NDA in the U.S.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Fast Track Designation granted earlier this year by the FDA permits the company to submit sections of the NDA on a rolling basis. The NDA is supported by data from the two positive Phase III studies of surufatinib in NET in China, along with data from surufatinib studies in U.S. non-pancreatic and pancreatic NET patients. The company previously announced that it had reached an agreement with the FDA during a pre-NDA meeting, whereby these studies may serve as the basis to support an NDA submission. Filing acceptance of the NDA is subject to FDA review of the complete application. The data package will also be used to file a Marketing Authorization Application ("MAA") to the European Medicines Agency ("EMA") in 2021, based on scientific advice from the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Marek Kania, M.D., Managing Director and Chief Medical Officer of Hutchison MediPharma International, said, "With the initiation of our first regulatory filing in the U.S., we are executing our strategy of building a global pharmaceutical company that brings innovative cancer therapies to patients worldwide. There is a great need for additional therapies to treat neuroendocrine tumors, and surufatinib has demonstrated significant clinical benefit in patients with advanced tumors. The NDA filing to the U.S. FDA represents a significant step toward our goal of commercializing surufatinib and other novel therapies globally."

The company plans to initiate an Expanded Access Protocol to ensure patients with limited therapeutic options have access to this important treatment. Enrollment into this early access program is anticipated to begin in the first quarter of 2021, once regulatory clearance of the protocol has been granted by the FDA.

The FDA granted two Fast Track Designations to surufatinib for development in pancreatic and non-pancreatic (extra-pancreatic) NET in April 2020 and Orphan Drug Designation for pancreatic NET in November 2019.

About NET
NET form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. NET are typically classified as pancreatic NET or non-pancreatic NET. Approved targeted therapies include Sutent and Afinitor for pancreatic NET, or well-differentiated, non-functional gastrointestinal or lung NET.

According to Frost and Sullivan, there were 19,000 newly diagnosed cases of NET in the U.S. in 2018. Importantly, NET are associated with a relatively long duration of survival compared to other tumors. As a result, there were approximately 141,000 estimated patients living with NET in the U.S. in 2018.[1]

About Surufatinib
Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.

Chi-Med currently retains all rights to surufatinib worldwide.

About Surufatinib Development
NET in the U.S., Europe and Japan: In the U.S., surufatinib was granted Fast Track Designations for development in pancreatic and non-pancreatic (extra-pancreatic) NET in April 2020 and Orphan Drug Designation for pancreatic NET in November 2019. A U.S. FDA NDA submission was initiated in December 2020, to be followed by a MAA submission to the EMA in Europe. The basis to support these filings includes the completed SANET-ep and SANET-p[2] studies, along with existing data from surufatinib in U.S. non-pancreatic and pancreatic NET patients (clinicaltrials.gov identifier: NCT02549937).

Non-pancreatic NET in China: In November 2019, a NDA for surufatinib for the treatment of patients with advanced non-pancreatic NET was accepted for review by the National Medical Products Administration of China ("NMPA") and granted Priority Review status in December 2019. The NDA is supported by data from the successful SANET-ep[3] study, a Phase III trial (clinicaltrials.gov identifier: NCT02588170) in patients with advanced non-pancreatic NETs conducted in China. The study had met the pre-defined primary endpoint of progression-free survival ("PFS") at a preplanned interim analysis. The positive results of this trial were highlighted in an oral presentation at the 2019 ESMO (Free ESMO Whitepaper) Congress and published in The Lancet Oncology in September 2020.[4] SANET-ep demonstrated that surufatinib reduces risk of progression or death by 67%, with median PFS significantly longer for patients treated with surufatinib at 9.2 months, compared to 3.8 months for patients in the placebo group (HR 0.334; 95% CI: 0.223-0.499; p<0.0001). Surufatinib had an acceptable risk/benefit ratio.

Pancreatic NET in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pancreatic NET in China. It was terminated early as the pre-defined primary endpoint of PFS was met (clinicaltrials.gov identifier: NCT02589821) at a preplanned interim analysis, leading to a second NDA accepted by the China NMPA in September 2020. The positive results of this study were presented at the 2020 ESMO (Free ESMO Whitepaper) Virtual Congress and published simultaneously in The Lancet Oncology[5], demonstrating that surufatinib reduces the risk of disease progression or death by 51% in patients, with median PFS of 10.9 months compared to 3.7 months on placebo (HR 0.491; 95% CI: 0.391-0.755; p=0.0011). The safety profile of surufatinib was manageable and consistent with observations in prior studies.

Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier: NCT03873532).

Immunotherapy combinations: We have entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies, including with tislelizumab (BGB-A317), Tuoyi (toripalimab) and Tyvyt (sintilimab), which are approved as monotherapies in China.

On December 28, 2020 Myovant Sciences (NYSE: MYOV) and Pfizer Inc. (NYSE: PFE) reported a collaboration to develop and commercialize relugolix – a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist – in oncology and women’s health in the U.S. and Canada (Press release, Myovant Sciences, DEC 28, 2020, View Source [SID1234573282]). Pfizer will also receive an exclusive option to commercialize relugolix in oncology outside the U.S. and Canada, excluding certain Asian countries.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are thrilled to partner with Pfizer to unlock the full potential of ORGOVYX in advanced prostate cancer and relugolix combination tablet in uterine fibroids and endometriosis, advancing our mission to redefine care for women and for men," said Lynn Seely, M.D., Chief Executive Officer, Myovant Sciences, Inc. "Pfizer is the ideal partner for Myovant given its impressive capabilities and track record across both oncology and women’s health. This transformative collaboration will significantly strengthen the upcoming launch of ORGOVYX and the potential launches of relugolix combination tablet in women’s health, while substantially enhancing our financial position and enabling us to expand our pipeline of potential new medicines."

Under the terms of the agreement, Myovant and Pfizer will jointly develop and commercialize ORGOVYX (relugolix) in advanced prostate cancer and, if approved, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) in women’s health in the U.S. and Canada. Myovant and Pfizer will begin co-promoting ORGOVYX for advanced prostate cancer in early 2021. Myovant and Pfizer will equally share profits and certain expenses for ORGOVYX and relugolix combination tablet with Myovant recording revenues. Myovant will remain responsible for regulatory interactions and drug supply and continue to lead clinical development for relugolix combination tablet. Myovant will receive up to $4.2 billion, including an upfront payment of $650 million, $200 million in potential regulatory milestones for U.S. Food and Drug Administration (FDA) approvals for relugolix combination tablet in women’s health, and tiered sales milestones upon reaching certain thresholds up to $2.5 billion in net sales for prostate cancer and also for the combined women’s health indications. If Pfizer exercises the option to commercialize relugolix in oncology outside of the U.S. and Canada, excluding certain Asian countries, Myovant will receive $50 million and be entitled to receive double-digit royalties on sales.

"We are excited to join forces with Myovant and combine our capabilities to bring ORGOVYX to patients with advanced prostate cancer," said Andy Schmeltz, Global President, Pfizer Oncology. "This strategic collaboration builds on our leadership in serving prostate cancer patients in the U.S. and aligns with our goal to deliver more breakthroughs across the prostate cancer treatment paradigm."

"There continues to be a high unmet need among the millions of women who experience the common and debilitating symptoms associated with uterine fibroids and endometriosis," said Nick Lagunowich, Global President, Pfizer Internal Medicine. "We believe our deep heritage and leadership in women’s health combined with our experienced women’s health field force will enable us to maximize these opportunities with Myovant, potentially bringing valuable new treatment options to these women."

The FDA approved ORGOVYX on December 18, 2020 for the treatment of adult patients with advanced prostate cancer. ORGOVYX is the first and only oral GnRH antagonist for men with advanced prostate cancer. Relugolix combination tablet is currently under regulatory review by the FDA for women with uterine fibroids, with a target action date of June 1, 2021. Relugolix combination tablet is also under development for women with endometriosis, with a New Drug Application submission anticipated in the first half of 2021.

Myovant Sciences Conference Call
Myovant will hold a webcast and conference call at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time today, December 28, 2020. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant’s website at investors.myovant.com. Institutional investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S.

The webcast will be archived on Myovant’s Investor Relations website following the call.

About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol, a hormone known to stimulate the growth of uterine fibroids and endometriosis. Relugolix (120 mg) is FDA-approved as ORGOVYX for adult patients with advanced prostate cancer. Relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is under regulatory review in Europe and the U.S. for women with uterine fibroids and is under development for women with endometriosis.

About ORGOVYX (relugolix)
ORGOVYX (relugolix) is the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist approved by the FDA for the treatment of adult patients with advanced prostate cancer. As a GnRH antagonist, ORGOVYX blocks the GnRH receptor and reduces production of testicular testosterone, a hormone known to stimulate the growth of prostate cancer.

For full prescribing information, including patient information, please click here.

Indication

ORGOVYX is approved for the treatment of adult patients with advanced prostate cancer.

Select Important Safety Information

Androgen deprivation therapy, such as ORGOVYX, may prolong the QT/QTc interval. Providers should consider whether the benefits of androgen deprivation therapy outweigh the potential risks in patients with congenital long QT syndrome, congestive heart failure, or frequent electrolyte abnormalities and in patients taking drugs known to prolong the QT interval. Electrolyte abnormalities should be corrected. Consider periodic monitoring of electrocardiograms and electrolytes.

The safety and efficacy of ORGOVYX have not been established in females. Based on findings in animals and mechanism of action, ORGOVYX can cause fetal harm and loss of pregnancy when administered to a pregnant female. Advise males with female partners of reproductive potential to use effective contraception during treatment and for 2 weeks after the last dose of ORGOVYX.

Most common adverse reactions (≥ 10%) in patients receiving ORGOVYX were hot flush (54%), musculoskeletal pain (30%), fatigue (26%), constipation (12%), and diarrhea (12%).

Most common laboratory abnormalities (≥ 15%) in patients receiving ORGOVYX were glucose increased (44%), triglycerides increased (35%), hemoglobin decreased (28%), alanine aminotransferase increased (27%), and aspartate aminotransferase increased (18%).

Co-administration of ORGOVYX with a P-gp inhibitor increases the area under the curve (AUC) and maximum concentration (Cmax) of ORGOVYX, which may increase the risk of adverse reactions associated with ORGOVYX. Avoid co-administration of ORGOVYX with oral P-gp inhibitors. If co-administration is unavoidable, take ORGOVYX first, separate dosing by at least 6 hours, and monitor patients more frequently for adverse reactions.

Co-administration of ORGOVYX with a combined P-gp and strong CYP3A inducer decreases the AUC and Cmax of ORGOVYX, which may reduce the effects of ORGOVYX. Avoid co-administration of ORGOVYX with combined P-gp and strong CYP3A inducers. If co-administration is unavoidable, increase the ORGOVYX dose to 240 mg once daily.

On December 28, 2020 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation using its PROTAC Discovery Engine, reported that the underwriters of its previously announced underwritten public offering of common stock, which closed on December 18, 2020, have exercised in full their option to purchase additional shares of common stock at the public offering price, less underwriting discounts and commissions (Press release, Arvinas, DEC 28, 2020, View Source [SID1234573280]). After giving effect to the sale of 857,142 additional shares of common stock in the option closing, the total number of shares of common stock sold by Arvinas in the offering increased to 6,571,428 shares, which resulted in aggregate net proceeds of approximately $431.9 million. All of the shares were offered by Arvinas.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Goldman Sachs & Co. LLC and Piper Sandler & Co. acted as joint book-running managers for the offering. Cantor Fitzgerald & Co. and BMO Capital Markets acted as co-managers for the offering.

An automatically effective shelf registration statement relating to the shares of common stock offered in the public offering was previously filed with the Securities and Exchange Commission. Copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained by contacting: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-906-9316 or by emailing [email protected]; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, by telephone at 800-747-3924 or by email at [email protected].

This press release does not constitute an offer to sell, or a solicitation of an offer to buy these securities, 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 registration or qualification under the securities laws of any such state or jurisdiction.

On December 28, 2020 Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, reported results of the primary data cut from its Phase 3 clinical trial evaluating the safety and efficacy of eprenetapopt with azacitidine (AZA) versus AZA alone in TP53 mutant myelodysplastic syndromes (MDS) (Press release, Aprea, DEC 28, 2020, View Source [SID1234573279]). The trial did not meet the predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a higher CR rate in the experimental arm receiving eprenetapopt with AZA versus the control arm receiving AZA alone, but did not reach statistical significance. In the intention-to-treat population of 154 patients, the CR rate in the eprenetapopt with AZA arm was 33.3% (95% CI: 23.1% – 44.9%) compared to 22.4% (95% CI: 13.6% – 33.4%) in the AZA alone arm (P = 0.13).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

While analysis of certain secondary endpoints (ORR and duration of responses) appears to favor the experimental arm at this data cut, they are not significantly different. The median duration of overall survival at the primary data cut was similar between the arms. Additional patients in the study who have not achieved a CR remain on study treatment and the data will be analyzed at future pre-specified timepoints as set forth in the statistical analysis plan. The combination of eprenetapopt with AZA appeared well-tolerated, with an adverse event profile that was similar to the Company’s prior Phase 2 clinical trials. Subsequent analyses of the trial data, including secondary endpoints, will be conducted as the duration of patient follow-up increases. The Company expects to present the data at a future scientific conference.

"Though we are disappointed the topline results did not reach statistical significance, we continue to believe that eprenetapopt can offer clinical benefit to patients with TP53 mutant malignancies," said Dr. Eyal Attar, Chief Medical Officer of Aprea. "We will continue to analyze data as it matures and follow patients who are still receiving study treatment. Our other clinical trials continue to progress and we remain committed to pursuing our clinical development programs."

About the Phase 3 Trial in TP53 Mutant MDS

The Phase 3 trial enrolled 154 TP53 mutant MDS patients, randomized 1:1 to either the eprenetapopt with AZA arm or the AZA alone arm. Response criteria are those defined by International Working Group 2006 (IWG 2006) and include measures of peripheral blood counts and bone marrow blasts.