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I-Mab Biopharma and Junshi Biosciences Announce Collaboration to Evaluate TJD5 in Combination with Toripalimab for the Treatment of Patients with Cancers in China

On September 10, 2019 I-Mab Biopharma (I-Mab), a China and U.S.-based clinical stage biopharmaceutical company exclusively focused on the discovery and development of potential first-in-class and best-in-class biologics in immuno-oncology and autoimmune diseases reported the signing of a collaboration agreement with Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences), an innovation-driven biopharmaceutical company to evaluate the combination therapy of I-Mab’s TJD5, a proprietary innovative CD73 antibody with Junshi Biosciences’ Toripalimab (Trade name: Tuoyi), a recombinant humanized anti-PD-1 monoclonal antibody in patients with cancers in China (Press release, I-Mab Biopharma, SEP 10, 2019, View Source [SID1234539422]).

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Dr. Jingwu Zang, Founder and Chairman of I-Mab, commented, "TJD5 is an innovative CD73 antibody with best-in-class potential that has entered into Phase I trial in the US. We are very pleased to collaborate with Junshi to explore the clinical synergies with Toripalimab which is an innovative drug with distinctive treatment advantages. We are looking forward to bringing more clinical benefits to patients across various cancer types."

Dr. Ning Li, CEO of Junshi Biosciences, commented, "As an anti multi-tumor drug, toripalimab has shown good safety and efficacy in clinical trials with mono and combination therapy. We believe, through the cooperation with I-Mab, we could continue to explore the combination potential of toripalimab and innovative drugs to improve the outcomes of immune-oncology therapy, lightening hope for more patients.

About TJD5:

TJD5 is a novel and differentiated blocking antibody against CD73, a surface enzyme on stromal cells and cancer cells responsible for the production of adenosine, which is highly immunosuppressive. It is expected to stimulate the immunosuppressive tumor micro-environment and to work in concert with other cancer therapies such as PD-1 and PD-L1 antibodies. TJD5 is in a Phase 1 clinical trial in the US, it is a proprietary innovative CD73 monoclonal antibody from I-Mab’s discovery pipeline with best-in-class potential.

About Toripalimab (JS001, Trade name: Tuoyi)

Toripalimab. a recombinant humanized anti-PD-1 monoclonal antibody for injection is the first domestic-developed anti-PD-1 mAb granted marketing approval in China, supported by National Science and Technology Major Project. In March 2018, its New Drug Application ("NDA") was accepted by the NMPA and put into prioritized evaluation and approval process. On Dec 2018, Toripalimab was conditionally granted marketing approval for use in the treatment of unresectable or metastatic melanoma that has failed previous systemic therapy by the NMPA, which is the first commercialized product of Junshi Biosciences. In the pivotal clinical trial, patients were observed with the ORR of 17.3% and the DCR of 57.5%, and the one-year overall survival rate is 69.3%. The NDA approval of toripalimab witnessed the pivotal step from pre-revenue biotech start-up to a commercial-stage biopharmaceutical company.

Since Toripalimab started its clinical trials at the beginning of 2016, over 30 trials have been conducted in China, the US and other countries, covering 14 kinds of tumors, i.e. NPC, UC, lung cancer, EC, HCC, TNBC, etc. Cooperations with domestic and overseas innovation pharma companies on combination therapy are also under exploration.

Pfenex to Participate in the Oppenheimer Fall Summit

On September 10, 2019 Pfenex Inc. (NYSE American: PFNX), a clinical-stage development and licensing biotechnology company focused on leveraging its Pfēnex Expression Technology to improve protein therapies for unmet patient needs, reported that Eef Schimmelpennink, President and Chief Executive Officer, and Susan Knudson, Chief Financial Officer, will be participating in one-on-one investor meetings at the Oppenheimer Fall Summit focused on specialty pharma and rare disease companies, taking place at the Parker Hotel in New York City on September 23rd – 24th (Press release, Pfenex, SEP 10, 2019, View Source [SID1234539434]).

Oppenheimer & Co. Fall Summit

Date: September 24th

Format: One-on-One Meetings

Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO)

On September 10, 2019 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., (Kyowa Kirin, TYO: 4151) reported plans to submit a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for Crysvita (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized (Press release, Kyowa Hakko Kirin, SEP 10, 2019, View Source [SID1234539404]). The decision to submit follows the completion of a pre-sBLA meeting with the FDA and agreement on the filing package. The submission of the Crysvita sBLA is planned for the first half of 2020 and will be based on the current clinical data package.

"Based on productive discussions with the FDA, we will be moving forward expeditiously with an sBLA filing for Crysvita in tumor-induced osteomalacia," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to working with the agency during the review process, and we are committed to getting this therapy to patients with this serious disease with significant unmet medical need."

The BLA package will include data from two single-arm Phase 2 studies, a 144-week Phase 2 study in 14 adult patients conducted by Ultragenyx in the U.S. and an 88-week Phase 2 study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels led to improvements in osteomalacia, mobility, and vitality. Bone scans also demonstrated an increase in healed fractures and decrease in new fractures during Crysvita treatment. Adverse events generally reflected the patients’ underlying disease, and there were no serious treatment-related adverse events during the studies.

Crysvita is approved by the U.S. FDA, Health Canada, and Brazil’s National Health Surveillance Agency (ANVISA) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older, and has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.

See below for Important Safety Information for Crysvita in X-linked hypophosphatemia.

About Tumor-Induced Osteomalacia (TIO)

TIO is caused by typically benign tumors that produce excess levels of fibroblast growth factor 23 (FGF23), causing phosphate wasting in the urine that leads to severe hypophosphatemia, osteomalacia, muscle weakness, fatigue, bone pain, and fractures. The symptoms rapidly resolve if the causal tumors or lesion can be resected; however, there are cases in which resection is not feasible or recurrence of the tumor occurs after resection. In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or vitamin D replacement. Efficacy of this management is often limited, as it does not treat the underlying disease and its benefits must be balanced with monitoring for potential risks such as nephrocalcinosis, hypercalciuria, and hyperparathyroidism. There are an estimated 500-1,000 patients with TIO in the United States, and approximately half of all cases are inoperable.

About Crysvita

Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Hakko Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

Important Safety Information in X-Linked Hypophosphatemia

CONTRAINDICATIONS

Do not use CRYSVITA with oral phosphate and active vitamin D analogs.
Do not initiate CRYSVITA treatment if serum phosphorus is within or above the normal range for age.
CRYSVITA is contraindicated in patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism.
WARNINGS AND PRECAUTIONS
Hypersensitivity

Hypersensitivity reactions (e.g. rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment.
Hyperphosphatemia and Risk of Nephrocalcinosis

Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient’s serum phosphorus levels.
Injection Site Reactions

Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment.
ADVERSE REACTIONS
Pediatric Patients

The most common adverse reactions (more than 10%) in pediatric XLH patients are: headache, injection site reaction, vomiting, pyrexia, pain in extremity, vitamin D decreased, rash, toothache, myalgia, tooth abscess, and dizziness.
Adult Patients

The most common adverse reactions (more than 5% and in at least 2 patients more than placebo) in adult XLH patients are: back pain, headache, tooth infection, restless leg syndrome, vitamin D decreased, dizziness, constipation, blood phosphorus increased.
Spinal stenosis is prevalent in adults with XLH and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression.
USE IN SPECIFIC POPULATIONS

There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Ultragenyx Adverse Event reporting line at 1-888-756-8657.
There is no information regarding the presence of CRYSVITA in human milk, or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition.

DCprime Initiates Research Collaboration with the University Medical Center of Groningen on Relapse Vaccine Approach for Ovarian Cancer

On September 10, 2019 DCprime, the front-runner in the field of relapse vaccines, reported the initiation of a research collaboration with the Department of Obstetrics and Gynecology at the University Medical Center of Groningen (UMCG) lead by Professor Hans Nijman MD PhD (Press release, DCPrime, SEP 10, 2019, View Source [SID1234539427]). The purpose of the collaboration is to design a novel relapse vaccine approach for ovarian cancer and to prepare for a clinical trial in ovarian cancer patients.

"We see significant therapeutic promise of our relapse vaccine in ovarian cancer and the researchers at the Department of Obstetrics & Gynecology at UMCG are recognized experts in this field. We are extremely fortunate to have their expertise and guidance as we tackle this new indication and begin evaluating our relapse vaccine approach in solid tumors," commented Jeroen Rovers MD PhD, CMO of DCprime.

"Ovarian cancer affects 1 in 80 women, with an average 70% chance of tumor recurrence following initial treatment. We look forward to collaborate with DCprime to improve outcomes for patients with ovarian cancer," commented Professor Nijman.
DCprime’s lead product DCP-001 is a relapse vaccine currently studied in an international Phase II trial in AML patients who are ineligible for hematopoietic stem cell transplantations. Next to broadening its pipeline in blood cancers, DCprime is now including ovarian cancer as the first potential solid tumor relapse vaccine indication. Together with the experts in ovarian cancer at UMCG, DCprime will define an optimal vaccination strategy and determine the overall clinical trial design

BerGenBio to Present at H.C. Wainwright 21st Annual Global Life Sciences Conference

On September 10, 2019 BerGenBio ASA (OSE:BGBIO), a clinical-stage biopharmaceutical company developing novel, selective AXL kinase inhibitors for multiple cancer indications, reported that Richard Godfrey, BergenBio’s chief executive officer, will present at the H.C. Wainwright 21st Annual Global Life Sciences Conference today, September 10, 2019 (Press release, BerGenBio, SEP 10, 2019, View Source [SID1234539426]).

Date: Tuesday, September 10, 2019
Time: 5.15pm CEST
Location: Lotte New York Palace Hotel, New York
The presentation slides will be available on www.bergenbio.com in the investor section at the time of the presentation.