On June 22, 2021 Nordic Nanovector ASA (OSE: NANOV) reported that encouraging initial results from the LYMRIT 37-05 Phase 1 trial investigating Betalutin (177Lu lilotomab satetraxetan) in patients with relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) not eligible for stem cell transplantation (Press release, Nordic Nanovector, JUN 22, 2021, View Source [SID1234584254]).

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The initial results from the completed Phase I study (n=16 treated with Betalutin) show that Betalutin was well tolerated, with a good safety profile consistent with all previous studies with Betalutin. As reported previously, a single, reversible dose-limiting toxicity (DLT) was seen in the last cohort investigating the highest dosing regimen (20 MBq/kg Betalutin and 100 mg/m2 lilotomab), which on review by the Independent Review Committee (IRC) resulted in three additional patients being enrolled. No further DLTs were seen. Clinical activity of Betalutin was seen in 6 evaluable patients receiving the highest dosing regimen including one complete response and one partial response.The IRC commented that the safety and anti-tumour activity of the highest dosing regimen could be considered for investigation in combination with other therapies used in R/R DLBCL which the Company is now evaluating, with an emphasis on combination partners that would not compromise the current safety profile of Betalutin.

Peter Braun, Nordic Nanovector CEO, commented: "We continue to be very encouraged by the overall safety profile that Betalutin exhibits in even the most fragile and highly pre-treated NHL patients. We have also seen clinical activity in DLBCL patients from a single administration of Betalutin and we will now consider the next steps for its development in this large patient population potentially in combination with other therapies, as part of our overall strategy to develop Betalutin for difficult to treat haematological tumours. Our near-term focus remains very much on completing PARADIGME in 3rd-line follicular lymphoma and delivering top line 3-month data by the end of 2021."

About LYMRIT 37-05

The LYMRIT 37-05 study is a Phase 1 open-label, single-arm, dose-escalation study designed to assess the safety and preliminary anti-tumour activity of a single administration of Betalutin. Patients were enrolled at clinical trial sites in the US and Europe. More information on this study can be found at www.clinicaltrials.gov (NCT02658968).

The starting doses of Betalutin and lilotomab were 10MBq/kg and 60mg (Cohort 1, n=3), respectively, and then Betalutin 10MBq/kg and lilotomab 100mg (Cohort 2, n=3 treated with Betalutin). Cohort 3 received 15MBq/kg and lilotomab 100mg (n=3) and Cohort 4 received 20MBq/kg and lilotomab 100mg (n=7 treated, 6 evaluable).

About DLBCL

DLBCL is an aggressive form of non-Hodgkin’s Lymphoma (NHL) that accounts for up to 43% of all NHL cases, making it the most common form of the disease. Approximately 40% of DLBCL patients relapse after first-line combination treatment with rituximab and chemotherapy and only 30-40% of relapsed patients respond with subsequent high-dose chemotherapy followed by Stem Cell Transplantation (ref. 1). There are currently very few therapeutic options for patients not eligible for SCT, which makes relapsed DLBCL a serious unmet medical need. The number of diagnosed incident cases of DLBCL in the 7 major markets (U.S., and key 5 European markets and Japan) was 64,172 in 2018 and is expected to be 74,927 in 2028 (ref. 2). The value of the 3L DLBCL market segment in the key 7 pharma markets is expected to increase from USD 0.6B in 2019 to USD 1.3B in 2028, the value of the 2L DLBCL market segment is expected to increase from USD 0.4B in 2019 to USD 2.0B in 2028. (ref. 2).

1. L.S. Raut and P. P. Chakrabarti: Management of relapsed-refractory diffuse large B cell lymphoma (2014) South Asian J. Cancer 3(1): 66–70

2. NHL and CLL Report, CRG, 2000, Disease Landscape and Forecast

On June 22, 2021 Alchemab Therapeutics, a biotechnology company focused on the discovery and development of naturally-occurring protective antibodies in neurodegeneration and oncology, reported the appointment of Douglas A. Treco, PhD as Chief Executive Officer (Press release, Alchemab Therapeutics, JUN 22, 2021, View Source [SID1234584217]). Dr. Treco will join the Company’s Board of Directors and will be based in Boston, Massachusetts.

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"We’re excited that Doug will bring his extensive experience to our leadership team as we seek to grow our highly differentiated platform," said Houman Ashrafian, BM BCh, DPhil, Managing Partner at SV Health Investors and Chairman of Alchemab’s Board of Directors. "Our goal is to develop a world class management team with footprints on both sides of the Atlantic and broaden the Company’s exposure to US investigators, investors, and potential employees."

Most recently, Dr. Treco was Co-Founder, President, and Chief Executive Officer of Ra Pharmaceuticals, Inc., a biotechnology company focused on peptide and small molecule inhibitors of the complement pathway. The company was acquired in April 2020 by UCB S.A. for $2.5 billion.

"Alchemab’s approach of identifying protective antibodies truly changes the game in terms of creating the next major antibody therapeutics company," said Dr. Treco. "We believe this platform gives us a unique ability to develop a robust pipeline of truly innovative neurodegenerative and oncology medicines."

"Doug has a proven track record of building successful life science companies and we’re pleased to team up with him again," said Andrew Levin, MD, PhD, Managing Director at RA Capital. "In addition, Alchemab has tremendous scientific leadership, with Jane Osbourn, PhD FMedSci, OBE, spearheading this novel approach. Together, we’re confident that they will continue to grow Alchemab into a major source of novel drugs to benefit patients."

"We are tremendously grateful to Alchemab’s founding CEO, Alex Leech, who led the Company since its inception and oversaw the Series A financing," noted SV’s Professor Ashrafian. Mr. Leech will return to company creation as a Venture Partner at SV Health Investors.

Previously, Dr. Treco co-founded Transkaryotic Therapies, Inc. (TKT), which was acquired in 2005 by Shire plc. In his position as Senior Vice President of Research and Development, he established and directed TKT’s gene activation and protein production efforts, which led to the approval of Dynepo, Replagal, Elaprase, and Vpriv. Dr. Treco is Chairman of the Board of Directors of Inozyme Pharma and is a member of the Board of Directors of CRISPR Therapeutics AG. He is also a scientific advisor to Lightstone Ventures. From January 2008 to May 2014, Dr. Treco served as an entrepreneur-in-residence with Morgenthaler Ventures and was a visiting scientist in the Department of Molecular Biology at Massachusetts General Hospital and a lecturer in genetics at Harvard Medical School from 2004 to 2007. He received a BA in Biology from the University of Delaware, a PhD in Biochemistry and Molecular Biology from the State University of New York at Stony Brook and performed post-doctoral research at the Salk Institute for Biological Studies and Massachusetts General Hospital.

In April 2021, Alchemab raised $82 million in a Series A financing led by RA Capital and joined by Lightstone Ventures, DHVC, and Data Collective VC Bio. In 2019, Alchemab raised a seed round led by SV Health Investors and the Dementia Discovery Fund. The Company collaborates closely with Illumina, having been part of the first cohort of companies joining the Illumina Accelerator in Cambridge, UK, and recently announced a partnership with AstraZeneca where Alchemab will use its platform to aid in the development of new treatments for prostate cancer.

On June 22, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that Ellen Kim, MD, Medical Director, Dermatology Clinic, Perelman Center for Advanced Medicine, Professor of Dermatology at the Hospital of the University of Pennsylvania, and the Lead Principal Investigator for the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study in cutaneous T-cell lymphoma (CTCL), will present key details of HyBryte (hypericin ointment 0.25%) efficacy and safety profile demonstrated in the FLASH study at the United States Cutaneous Lymphoma Consortium (USCLC) Annual Meeting, to be held on June 26, 2021 (Press release, Soligenix, JUN 22, 2021, View Source [SID1234584233]). Dr. Kim plans to detail the success of HyBryte in the broad CTCL patient population, as well as the safety and tolerability of HyBryte, and to compare the data from the study to the currently available treatment options. The FLASH trial is the largest multicenter, randomized, double-blind, placebo-controlled, skin-directed therapy study in CTCL to date, enrolling a total of 169 patients.

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Oral Presentation:

Topical hypericin ointment photodynamic therapy is effective and safe in CTCL (FLASH study) Dr. Kim’s presentation will be given during the 11:15 am – 12:15 pm Eastern Time session on June 26, 2021. Only registered attendees can view and participate in this virtual conference. Attendees can register for United States Cutaneous Lymphoma Consortium.

"The audience at the USCLC consists of key disease state experts and potential future prescribers of this novel photodynamic therapy assuming it is approved for distribution, many of whom were investigators with me in this pivotal Phase 3 clinical trial. I am very excited to meet with them to share and analyze our data together," noted Dr. Kim. "To be selected to present our data at this forum is a great opportunity, as I believe HyBryte can better meet the therapeutic needs of patients with CTCL where additional treatment options are desperately needed. I would like to thank all site investigators, and especially our patients, for their contributions to developing this promising new therapy."

About the United States Cutaneous Lymphoma Consortium

The USCLC meeting is an annual meeting, dedicated specifically to cutaneous lymphomas. This meeting is comprised of the leading experts in this specific discipline. The USCLC represents the only organization in America where professionals from various disciplines (including dermatology, medical oncology, radiation oncology, and pathology) can exchange ideas and practice tips, discuss and plan scientific collaborations, develop new educational tools, and help shape the research agenda in cutaneous lymphoma. USCLC’s mission is to foster a multidisciplinary approach to patient care, education, and clinical and basic research in the area of cutaneous lymphomas.

About HyBryte

HyBryte (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).

The Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte’s mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

On June 22, 2021 SAB Biotherapeutics (SAB), a clinical-stage biopharmaceutical company with a novel immunotherapy platform that produces targeted, high potency, fully-human polyclonal antibodies at scale, and Big Cypress Acquisition Corp. (NASDAQ: BCYP), a blank check company focused on innovative biopharmaceutical firms, reported that they have entered into a definitive business combination agreement (Press release, SAB Biotherapeutics, JUN 22, 2021, View Source [SID1234584257]). Upon closing of the proposed transaction, the combined company will operate as SAB Biotherapeutics and will continue to operate under the SAB management team, with Big Cypress Acquisition Corp.’s Samuel J. Reich and Jeffrey G. Spragens joining the SAB Board of Directors. SAB co-founder and current executive chairman, Dr. Edward Hamilton, plans to transition to a board observer role while remaining active in the company. Mr. Reich is expected to assume the role of executive chairman.

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SAB plans to be listed on NASDAQ following the closing of the transaction. The proposed transaction implies an enterprise value for SAB, on a post-merger basis, of approximately $325 million (assuming a share price of $10.10 per share) and is expected to provide the combined company with approximately $118 million of pro forma cash (assuming no redemptions from Big Cypress’ trust account), to fuel development and commercialization of SAB’s unique DiversitAb platform that leverages its proprietary transchromosomic (Tc) bovine herds to produce highly-potent targeted fully-human polyclonal antibody therapeutics for a wide range of immune system disorders, cancer and infectious diseases without the need for human donors.

Proceeds from the business combination are expected to support advancement of SAB’s robust pipeline and platform through multiple catalysts across several programs. These potentially include multiple clinical data read-outs, which represent potential value inflection events.

"The execution of this merger agreement caps 18 months of tremendous progress at SAB," said Eddie Sullivan, PhD, co-founder and chief executive officer of SAB Biotherapeutics. "Years of innovation and investment in our unique human polyclonal antibody platform have enabled us to rapidly respond to the COVID-19 pandemic, moving a therapeutic candidate from concept to the clinic in less than five months. The awarded funding and our collaboration with the federal government, as part of COVID-19 Response (formerly Operation Warp Speed), has enabled us to test, refine and advance our novel therapeutic development and internal production and regulatory processes, as well as provide a model for future rapid response. This period of intense activity leaves us well-positioned to advance our portfolio of novel therapies for immune system disorders, cancer and other infectious diseases. The unique attributes of our DiversitAb platform power our diversified strategy that includes rapid response, development of our own novel therapeutics, and a variety of pharma collaborations. We are excited at the expanded opportunities afforded by the merger to put our platform to work generating important new therapies for unmet medical needs."

"At Big Cypress we screened more than 60 biotech companies in our search for the perfect merger partner," noted Sam Reich, chief executive officer of Big Cypress Acquisition Corp. "We were thrilled when we found SAB Biotherapeutics, a distinctive and exciting approach to marrying the power of nature with advanced genomic technology. SAB met or exceeded our criteria, demonstrating de-risked early development processes, clinical proof-of-concept, therapeutic targets with large unmet needs and what we expect to be manageable Phase 3 trials, high-value indications, and the ability to efficiently put additional capital to work advancing a high potential pipeline. We like that SAB’s innovative technology and management team are unconventional by biotech standards, with their novel DiversitAb platform leveraging Tc bovine herds that rapidly produce fully-human polyclonal antibodies with almost unlimited therapeutic potential, as well as their location in our nation’s heartland. We look forward to working with the exceptional SAB team to advance this important technology."

SAB Biotherapeutics Overview

SAB Biotherapeutics is a clinical-stage biopharmaceutical company advancing a new class of immunotherapies based on its human polyclonal antibodies. Applying advanced genetic engineering and antibody science, SAB develops fully-human antibodies produced from transchromosomic (Tc) bovine herds targeted at addressing specific diseases, including infectious diseases such as COVID-19 and influenza, immune system disorders including type 1 diabetes and organ transplantation, and cancer. SAB’s versatile and scalable DiversitAb platform is applicable to a wide range of serious human diseases. It rapidly produces natural, specifically targeted, high-potency, human polyclonal immunotherapies at commercial scale. The platform has been developed and validated through funding awarded from US government emerging disease and rapid response programs. SAB is currently advancing multiple clinical programs in a number of indications, in addition to its collaborations with global pharmaceutical and other partners.

Summary of the Transaction

Upon the closing of the business combination, and assuming a share price of $10.10 per share and no redemptions of shares of Big Cypress by its public stockholders, SAB would be expected to have an enterprise value of approximately $325 million and cash resources of approximately $118 million, including the contribution of up to $116 million from cash held in Big Cypress’ trust account (less any redemptions). Pro forma for the business combination, legacy shareholders of SAB will own approximately 68% of the post-merger public company, excluding any contingent consideration and before giving effect to any potential exercise of Big Cypress’ common stock purchase warrants into shares of common stock following the closing. There is no minimum cash closing condition for the transaction.

The transaction has been unanimously approved by both Big Cypress’ and SAB’s respective Boards of Directors. The proposed transaction is subject to the approval of Big Cypress and SAB stockholders and the satisfaction or waiver of other customary conditions, including a registration statement being declared effective by the U.S. Securities and Exchange Commission (the "SEC"), and is expected to close in the fourth quarter of 2021.

Additional information about the proposed transaction, including a copy of the merger agreement and an investor presentation, will be provided in a Current Report on Form 8-K to be filed by Big Cypress with the SEC, which will be available at www.sec.gov.

Advisors

Lazard is serving as exclusive financial advisor to SAB and Stradling Yocca Carlson & Rauth is serving as legal counsel. Chardan is serving as exclusive M&A advisor and financial advisor to Big Cypress and Dentons US LLP is serving as legal counsel. Ladenburg Thalmann & Co. Inc. acted as sole book-running manager and Brookline Capital Markets, a division of Arcadia Securities, LLC, acted as co-manager of Big Cypress $115 million IPO in January 2021 and Ladenburg Thalmann & Co. Inc. is acting as a capital markets advisor to Big Cypress.

Conference Call and Webcast Details

SAB and Big Cypress Acquisition Corp. will host a conference call and live audio webcast to discuss the proposed transaction today at 8:00 a.m. ET. To access the live conference call, please dial (833) 882-5274 (domestic) or (409) 937-8892 (international) at least five minutes prior to the start time and refer to conference ID 1865196.

A live audio webcast of the call can be accessed here and an archive will be available approximately two hours after the event.

On June 22, 2021 AstraZeneca and MSD reported that its Koselugo (selumetinib) has been granted conditional approval in the European Union (EU) 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, JUN 22, 2021, View Source [SID1234584201]).

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NF1 is a debilitating genetic condition affecting one in 3,000 individuals worldwide.1,2 In 30-50% of people with NF1, tumours develop on the nerve sheaths (plexiform neurofibromas) and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.3-7

The approval by the European Commission was based on positive results from the SPRINT Stratum 1 Phase II trial sponsored by the National Institute of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). This trial showed Koselugo reduced the size of inoperable tumours in children, reducing pain and improving quality of life.7,8 This is the first approval of a medicine for NF1 PN in the EU and follows the positive recommendation by the Committee for Medicinal Products for Human Use of the European Medicines Agency in April 2021. Safety and efficacy data from the SPRINT Phase II trial with longer follow up will be provided as one of the conditions of approval.

Brigitte C. Widemann, MD, Principal Investigator of the SPRINT trial and Chief, NCI Pediatric Oncology Branch, said: "For children with neurofibromatosis type 1, plexiform neurofibromas can grow and develop so significantly that, in some cases, it becomes debilitating. In the SPRINT trial, selumetinib shrank NF1-associated PNs in 66% of patients and showed clinically meaningful improvements in PN-related symptoms."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "As the first medicine approved in the EU for patients with neurofibromatosis type 1, Koselugo has the potential to transform the way plexiform neurofibromas are managed and treated. The SPRINT data showed that Koselugo not only shrank tumours in some children, but also reduced pain and improved their quality of life. This significant milestone was made possible thanks to our research partners, the National Cancer Institute, the Neurofibromatosis Therapeutic Acceleration Program, the Children’s Tumor Foundation, the patient community and every child, parent and doctor involved in the clinical trial."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Before this approval, surgery was the only treatment option for children in the EU with neurofibromatosis type 1 plexiform neurofibromas. This approval marks a significant step forward in addressing the debilitating impact of these tumours."

The SPRINT Stratum 1 Phase II trial showed Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with Koselugo as twice-daily oral monotherapy.8 ORR is defined as the percentage of patients with confirmed complete (disappearance of PN) or partial response (at least 20% reduction in tumour volume).8 Results were published in The New England Journal of Medicine.7

Koselugo is approved in the US and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PN. Further regulatory submissions are underway. Clinical trials of Koselugo in adult patients with NF1 PN, including an alternative age-appropriate formulation for paediatric patients, are scheduled to begin this year.

NF1
NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots). In 30-50% of people, tumours develop on the nerve sheaths.1,3,9,10 These PN can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bladder/bowel dysfunction and disfigurement, as well as having the potential to transform into malignant peripheral nerve sheath tumours.4-7,10 PN begin developing during early childhood, with varying degrees of severity, and can reduce life expectancy by eight to 15 years.3,6,11,12

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 selumetinib monotherapy.7 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 an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).8 MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.13

Koselugo received US FDA Breakthrough Therapy Designation in April 2019, Rare Pediatric Disease Designation in December 2019 and US Orphan Drug Designation in February 2018. Further orphan designations have been granted in the EU, Japan, Russia, Switzerland, South Korea, Taiwan and Australia.

AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.