On September 25, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation (ODD) to CLR 131, the company’s lead Phospholipid Drug Conjugate (PDC) product candidate, for the treatment of pediatric osteosarcoma, a rare pediatric cancer (Press release, Cellectar Biosciences, SEP 25, 2018, View Source [SID1234530174]). CLR 131 also received Rare Pediatric Disease Designation for osteosarcoma, as announced by the company on September 17, 2018.

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"Osteosarcoma is the most common type of primary bone cancer occurring most frequently in children. Currently, there are no commercially available drugs for pediatric sarcoma, including osteosarcoma," said John Friend, M.D., chief medical officer of Cellectar. "This orphan designation for osteosarcoma is the fourth such designation granted by the FDA to CLR 131 for the treatment of rare pediatric cancers in the last six months, and we look forward to evaluating CLR 131 in these deadly and underserved diseases."

The FDA grants ODD to therapies targeting conditions that affect fewer than 200,000 people in the U.S. The designation provides seven-year market exclusivity, increased engagement and assistance from the FDA, tax credits for certain research, research grants and a waiver of the New Drug Application user fee. In 2018 the FDA also granted CLR 131 orphan drug and rare pediatric disease designations for the treatments of neuroblastoma, rhabdomyosarcoma and Ewing’s sarcoma.

Cellectar plans to initiate a Phase 1 clinical study evaluating CLR 131 for the treatment of pediatric patients with osteosarcoma, Ewing’s sarcoma, rhabdomyosarcoma, neuroblastoma, high-grade glioma and lymphomas. The trial is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of CLR 131 in pediatric patients with these cancer types. Further details about the trial can be found at clinicaltrials.gov using the identifier number NCT03478462.

About Osteosarcoma

Osteosarcoma derives from bone forming mesenchymal, or connective tissue, cells and is the most commonly diagnosed primary bone malignancy among children and adolescents. The incidence is about 4.4 cases per 1 million per year in children younger than 24 years. While there is a 70% cure rate among patients with localized disease, 5-year overall survival rates are approximately 20% for among patients who develop metastatic disease. Additionally, among patients who experience disease progression or recurrence survival for is less than 30%.

About CLR 131

CLR 131 is Cellectar’s investigational radioiodinated PDC therapy that exploits the tumor-targeting properties of the company’s proprietary phospholipid ether (PLE) and PLE analogs to selectively deliver radiation to malignant tumor cells, thus minimizing radiation exposure to normal tissues. CLR 131 is in a Phase 2 clinical study in R/R MM and a range of B-cell malignancies and a Phase 1b clinical study in patients with R/R MM exploring fractionated dosing. The objective of the multicenter, open-label, Phase 1b dose-escalation study is the characterization of safety and tolerability of CLR 131 in patients with R/R MM. Patients in Cohorts 1-4 received single doses of CLR 131 ranging from 12.5 mCi/m2 to 31.25 mCi/m2 as well as a fractionated dose of 15.625 mCi/m2 given twice over seven days in Cohort 5. All study doses and regimens have been deemed safe and well tolerated by an independent Data Monitoring Committee. The company is currently initiating a Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma, and is planning a second Phase 1 study in combination with external beam radiation for head and neck cancer.

On September 25, 2018, Aduro Biotech, Inc. ("we" or the "Company")reported it received written notices of termination from Janssen Biotech, Inc. ("Janssen") for its Research and License Agreements pertaining to the Company’s proprietary attenuated strains of Listeria for treatment of lung and prostate cancers (Filing, 8-K, Aduro Biotech, SEPT 25, 2018, View Source [SID1234529939]). Specifically, Janssen delivered notice for the following agreements (the "Janssen Agreements"): (i) the Research and License Agreement, dated as of October 13, 2014, as amended by that certain Amendment to Research and License Agreements, dated as of November 11, 2015 (the "Amendment"); (ii) the Research and License Agreement, dated as of May 27, 2014, as amended by the Amendment; and (iii) the GVAX Prostate License Agreement, dated as of May 27, 2014. The terminations are effective December 24, 2018.

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Under the terms of the Janssen Agreements, the Company granted Janssen an exclusive, worldwide license to research, develop, manufacture, use, sell and otherwise exploit products containing ADU-214, ADU-741 and GVAX Prostate for any and all uses. Additionally, the Company granted Janssen exclusive rights to develop products utilizing the Company’s proprietary attenuated strains of Listeria for treatment of lung and prostate cancers. The Company previously received upfront license fees and milestone payments upon completion of various development activities and was eligible to receive future contingent payments based on development, regulatory and commercial milestones as well as royalties on any net sales of licensed products by Janssen under each of the Janssen Agreements. Pursuant to the terms of the Janssen Agreements, upon Janssen’s termination, the Company regains worldwide rights for the development and commercialization of products containing ADU-214, ADU-741 and GVAX Prostate for any and all uses. In addition, Janssen will have certain obligations as set forth in the Janssen Agreements, including (i) immediately ceasing its use of any Company intellectual property and (ii) promptly returning or destroying any materials related to the development or manufacturing of the products containing ADU-214, ADU-741 and GVAX Prostate.

The foregoing descriptions of the Janssen Agreements do not purport to be complete and are qualified in their entirety by the full text of the agreements. The Janssen Agreements were filed as Exhibits 10.18, 10.19 and 10.20 to the Company’s Registration Statement on Form S-1, filed with the Securities and Exchange Commission on March 11, 2015, and the Amendment was filed as Exhibit 10.41 to the Company’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission on March 8, 2016.

On September 25, 2018 Akari Therapeutics, Plc (NASDAQ:AKTX) ("Akari" or "the Company"), a biopharmaceutical company focused on the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement and or leukotriene systems are implicated, reported that the Company’s management will present an overview of the Company and host investor meetings at the 2018 Cantor Fitzgerald Global Healthcare Conference (Press release, Akari Therapeutics, SEP 25, 2018, View Source [SID1234529897]).

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Date: Monday, October 1, 2018

Time: 7:45 a.m. ET

Place: InterContinental New York Barclay Hotel, New York City

A live webcast of the presentation can be accessed by visiting ‘Events’ in the Investors Section on the Company’s website at www.akaritx.com. An archived replay of the webcast will be available for 60 days on the Company’s website after the conference.

On September 25, 2018 Portola Pharmaceuticals (Nasdaq: PTLA) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to cerdulatinib, an investigational, oral Syk/JAK inhibitor for the treatment of peripheral T-cell lymphoma (PTCL) (Press release, Portola Pharmaceuticals, SEP 25, 2018, View Source;p=RssLanding&cat=news&id=2368929 [SID1234529770]).

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"We are pleased that the FDA has granted cerdulatinib Orphan Drug Designation, as it recognizes its potential to provide a significant clinical benefit to a group of patients with limited treatment options," said John Curnutte, M.D., Ph.D., Portola’s interim co-president and head of research and development. "We look forward to presenting additional data from the Phase 2a trial at a scientific congress early next year and to continuing discussions with the FDA regarding next steps for the development of cerdulatinib, including the potential for an accelerated approval pathway."

The FDA’s Office of Orphan Products Development grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA market application fees.

About PTCL
PTCL is a group of rare and often fast-growing lymphomas classified as a subtype of non-Hodgkin’s lymphoma. There are approximately 6,000 to 10,000 cases of PTCL annually in the United States. The three most common types of PTCL are anaplastic large cell lymphoma (ALCL), angioimmunoblastic T-cell lymphoma (AITL), and PTCL not otherwise specified (PTCL-NOS), but many other rarer types exist. PTCL is most often treated with a combination of chemotherapies and the options for patients that fail front-line therapy are limited.

About Cerdulatinib
Cerdulatinib is an investigational oral, dual spleen tyrosine kinase (Syk) and janus kinase (JAK) inhibitor that uniquely inhibits two key cell signaling pathways implicated in certain hematologic malignancies and autoimmune diseases. There is a strong rationale for inhibiting both Syk (B-cell receptor pathway) and JAK (cytokine receptors) in B-cell malignancies where both targets have been shown to promote cancer cell growth and survival. In addition, pre-clinical data suggest an important role for Syk and JAK in peripheral T-cell lymphoma (PTCL) tumor survival.

In addition to PTCL, cerdulatinib is being evaluated in an ongoing Phase 2a study among patients with other specific subtypes of B-cell and T-cell Non-Hodgkin Lymphoma (NHL), including relapsed/refractory follicular lymphoma (FL) and chronic lymphocytic lymphoma/small lymphocytic lymphoma (CLL/SLL). The Company reported new data from this study in June at both the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper). Cerdulatinib demonstrated broad clinical activity including an objective response rate of 47 percent in all patients, and was generally well tolerated. Additionally, seven of the 20 patients in the PTCL cohort achieved a complete response at the time of presentation.

On September 25, 2018 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immuno-oncology biopharmaceutical company focused on the acquisition, development and commercialization of novel treatments for patients with solid tumor cancers, reported that positive preliminary safety and efficacy data from an ongoing Phase 1/2 clinical trial of CK-101 were presented yesterday in an oral presentation at the International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer in Toronto (Press release, Checkpoint Therapeutics, SEP 25, 2018, View Source [SID1234529735]). The oral presentation included further details on, and updates from, the dataset announced previously. CK-101 is a third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) being evaluated in advanced non-small cell lung cancer (NSCLC).

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"The oral presentation included exciting updates to the data released in the abstract, including intracranial disease responses to treatment with CK-101 in patients with brain metastases present at baseline indicating that CK-101 may cross the blood-brain barrier to reach metastases in the central nervous system, as well as an additional partial response post-data cutoff in a T790M mutation-positive NSCLC patient that failed previous TKI therapy," said James F. Oliviero, President and Chief Executive Officer of Checkpoint Therapeutics. "Based on these data, we believe CK-101 has the potential to be an effective and differentiated treatment option in a potential $6 billion market currently dominated by one approved therapy."

Highlights from the Oral Presentation
• CK-101 was well-tolerated across multiple dose groups
– Most adverse events were Grade 1-2
– Maximum-tolerated dose was not defined; no reported dose-limiting toxicities or treatmentrelated
serious adverse events
– No events of interstitial lung disease, pneumonitis, QTc prolongation, cardiomyopathy, nail
toxicities, stomatitis or hyperglycemia, which are notable observed side effects of marketed
TKI therapies
• CK-101 demonstrates preliminary activity in EGFR mutation-positive NSCLC
– 75% (6 of 8) objective response rate (ORR) in treatment-naïve patients
– 100% (19 of 19) disease control rate (DCR), including 84% (16 of 19) of patients with target
lesion reductions versus baseline
– 60% (3 of 5) of patients with baseline brain metastases had intracranial disease response
• Enrollment in the trial is ongoing to identify the optimal dose to maximize therapeutic effect,
following which a Phase 3 trial is planned to initiate in 2019 in treatment-naïve EGFR mutationpositive
NSCLC patients.

The first-in-human, multicenter trial is evaluating CK-101 in NSCLC patients with EGFR mutations and other advanced malignancies (NCT02926768). Following dose escalation ranging from 100 mg to 1,200 mg/day in patients with any solid tumor where targeted EGFR was deemed reasonable, a first doseexpansion cohort was enrolled at 400 mg twice daily in patients with a confirmed diagnosis of either (1) EGFR mutation-positive advanced or metastatic NSCLC without prior exposure to EGFR-TKI therapy, or (2) T790M-positive advanced or metastatic NSCLC with disease progression on previous EGFR-TKI therapy. There was no limit on the number of prior lines of systemic therapy patients received prior to entering the trial.

A copy of the oral presentation slides is available on the Publications page in the Pipeline section of
Checkpoint’s website, www.checkpointtx.com.

About CK-101
CK-101 (also known as RX518) is an oral, third-generation, irreversible kinase inhibitor against selective mutations in the EGFR gene. Activating mutations in the tyrosine kinase domain of EGFR, such as L858R and exon 19 deletion, are found in approximately 20 percent of patients with advanced non-small cell lung cancer (NSCLC).

Compared to chemotherapy, first-generation EGFR inhibitors significantly improved objective response rate and progression-free survival in previously untreated NSCLC patients carrying EGFR mutations. However, tumor progression could develop due to resistance mutations, often within months of treatment with first-generation EGFR inhibitors. The EGFR T790M "gatekeeper" mutation is the most common resistance mutation found in patients treated with first-generation EGFR inhibitors. The mutation decreases the affinity of first-generation inhibitors to EGFR kinase domain, rendering the drugs ineffective. Second-generation EGFR inhibitors have improved potency against the T790M mutation, but have not provided meaningful benefits in NSCLC patients due to toxicity from also inhibiting wild-type EGFR. Third-generation EGFR inhibitors are designed to be highly selective against both EGFR-TKIsensitizing and resistance mutations, with minimal activity on wild-type EGFR, thereby improving tolerability and safety profiles.

Checkpoint Therapeutics is developing CK-101 for the treatment of NSCLC patients carrying the susceptible EGFR mutations. These include the EGFR T790M mutation in second-line NSCLC patients, as well as the EGFR L858R and exon 19 deletion mutations in first-line NSCLC patients. Checkpoint holds an exclusive worldwide license (except with respect to certain Asian countries) to CK‐101, which it acquired from NeuPharma, Inc., in 2015.