On January 8, 2019 Adimab, LLC, the global leader in the discovery and optimization of fully human monoclonal and bispecific antibodies, reported that in 2018 it entered into agreements with 10 new companies (Press release, Adimab, JAN 8, 2019, View Source [SID1234532584]). In addition, Adimab announced the expansion of seven of its current partnerships and the achievement of 70 technical and development milestones across numerous collaborations.

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In the past ten years, Adimab has partnered with over 60 companies for the discovery of therapeutic IgGs and bispecific antibodies, resulting in more than 260 therapeutic programs derived from the Adimab Platform. In 2018, Adimab and its partners added 56 new therapeutic programs. New alliances for 2018 include collaborations with Biotheus, Boehringer Ingelheim, Cullinan Oncology, Cygnal Therapeutics, Pliant Therapeutics, REGiMMUNE, and Werewolf Therapeutics, among others. In addition, Adimab expanded its collaborations with Boehringer Ingelheim, Innovent Biologics, Mapp Biopharmaceutical, Sanofi, Surface Oncology, and Takeda.

"In 2018, we saw fantastic growth in the number of therapeutic programs coming from our platform," said Guy Van Meter, Senior Vice President of Business Development. "In addition to being the leader for antibody discovery, Adimab has become the go-to provider for bispecifics and protein engineering. Our partners really benefit from our deep domain expertise and get to access all our capabilities."

"In an industry that is used to exaggerated claims, we have proven time and time again that we deliver the highest quality therapeutic leads," added Tillman Gerngross, Chief Executive Officer and Co-Founder of Adimab. "To date more than 80% of our partners have expanded their partnership with Adimab, which is a meaningful indicator of the quality of our work."

Over the past five years, Adimab has worked with an increasing number of smaller companies, backed by leading venture capital firms. Following successful antibody discovery campaigns, many of these companies have gone on to partner their programs with larger pharmaceutical companies. Publicly announced transactions include: Tizona and Abbvie (2019), Dragonfly and Merck (2018), Tusk and Roche (2018), Scholar Rock and Gilead (2018), Alector and Abbvie (2017), Dragonfly and Celgene (2017), Mersana and Takeda (2016), Surface Oncology and Novartis (2016), Potenza and Astellas (2015, 2018), and Innovent and Lilly (2015).

"Roche was very thorough in their diligence, and the quality of the leads and the data from Adimab made the process much smoother," said Luc Dochez, former Chief Executive Officer of Tusk Therapeutics.

"Investors and partners invariably conduct extensive diligence on the structural and pharmacological properties of our drug leads," said Arnon Rosenthal, Co-Founder and Chief Executive Officer of Alector. "Adimab data and the support of the Adimab team were critical elements in developing compelling product candidates."

"Adimab has been a fantastic partner. The Adimab team is comprised of leading scientists, using state of the art technology, who have a sense of urgency towards their collaborators. I cannot praise them highly enough," said Bill Haney, Co-Founder and Chief Executive Officer of Dragonfly Therapeutics.

On January 8, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, reported topline results from the ongoing pivotal Phase 3 OLYMPUS clinical trial of UGN-101 (mitomycin gel) for instillation, an investigational mitomycin formulation for the non-surgical treatment of low-grade upper tract urothelial cancer (UTUC) (Press release, UroGen Pharma, JAN 8, 2019, View Source [SID1234532583]). This analysis showed that on an intent-to-treat basis, 57 percent of patients achieved a complete response (CR) rate at their primary disease evaluation (PDE, or the primary endpoint) which was conducted four to six weeks after completion of UGN-101 treatment. Importantly, all evaluated patients in CR remain disease free at six months.

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This international, multi-center trial completed enrollment with 71 patients in December 2018. Of the 71 patients enrolled in the trial, 61 patients have been evaluated for the primary endpoint which was a CR as defined as a negative ureteroscopic evaluation and a negative wash cytology. The remaining 10 patients are awaiting PDE evaluation.

"We are pleased to report that the CR and durability data remain consistent with the Interim Analysis presented in May 2018. These results continue to validate the potential of UGN-101 to shift the surgical treatment paradigm and benefit patients whose only alternative would be repetitive endoscopic surgical intervention or complete loss of a kidney," said Mark P. Schoenberg, M.D., Chief Medical Officer of UroGen. "The durability observed in the OLYMPUS study provides further evidence that the non-surgical treatment of LG UTUC with UGN-101 may result in clinically-meaningful, recurrence free survival. We are grateful to the patients, their families, and clinical investigators who have made this important study possible."

Approximately 45 percent of tumors treated were categorized as unresectable by surgery at baseline. Of the patients who achieved CR, UroGen now has six-month durability on half of these patients. Durability is a key secondary endpoint for the trial.

The safety profile of UGN-101 continues to be acceptable with most treatment-emergent adverse events characterized as mild or moderate and transient and in line with ureteral procedures. These included ureteral narrowing and hydronephrosis, urinary tract infection, flank pain and creatinine elevation.

UroGen intends to seek regulatory approval of UGN-101 in LG UTUC based on data from all 71 patients and initiated its rolling submission of the New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.

Seth Paul Lerner, M.D., FACS, Professor of Urology at Baylor College of Medicine in Houston, Texas served as Principal Investigator of the OLYMPUS trial.

About UGN-101

UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of mitomycin to urinary tract tissue, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters.

On January 8, 2019 Biond Biologics Ltd. ("Biond" or the "Company"), a privately-held Israeli biotechnology company, developing novel immunotherapies for cancer and autoimmune diseases, reported the closing of a $17 million Series B financing (Press release, Biond Biologics, JAN 8, 2019, View Source [SID1234532582]).

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Biond was founded in 2016 by the former scientific team of cCam Biotherapeutics, an immuno-oncology company which was fully acquired by Merck in July 2015. The former cCAM Biotherapeutics team was joined by additional veterans of the Israeli biotech industry, with the aim to build a strong, sustainable and innovative science-driven biotechnology company. The company’s vision and strategy are to bring innovative drugs to patients based on synergistic long-term collaborations with leading global companies in the immunotherapy field.

Biond intends to use the proceeds to move its lead drug candidate, BION-202, into clinical trials and to advance the preclinical development of BION-206 and its proprietary antibody cell-internalization technology.

The financing and due diligence were led by Israel Biotech Fund and Harel Insurance & Finance Group, with participation of Celgene Corporation, (NASDAQ: CELG), the Japanese-Israeli fund, SBI JI Innovation Fund and existing investors.

"We are excited to have such prominent investors as shareholders who are aligned with the company’s vision of delivering breakthrough immune-based solutions to patients with serious diseases," said Dr. Tehila Ben Moshe, Co-Founder and Chief Executive Officer of Biond Biologics. "After two years of intense and creative discovery work, we are ready to advance our first innovative drug candidate into clinical trials."

"We were very impressed by the innovative science discovered at Biond and by the quality and experience of its managerial and scientific team," said Dr. David Sidransky, M.D., Co-Founder and General Partner at Israel Biotech Fund and Biond board member. "Biond’s novel pipeline candidates have the potential of becoming paradigm shifting therapies in immuno-oncology."

"We are happy to support the outstanding management team at Biond and its cutting-edge pipeline and technology," stated at Harel Insurance & Finance. "This investment aligns with the group’s ongoing effort of investing in breakthrough and innovative technologies developed by companies with great growth potential."

On January 8, 2019 Moderna, Inc. (Nasdaq: MRNA), a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, reported recent updates to several of its immuno-oncology and rare disease programs and outlined its 2019-2020 corporate objectives (Press release, Moderna Therapeutics, JAN 8, 2019, View Source [SID1234532581]). Moderna has 21 mRNA development candidates in its pipeline, with 11 programs now in clinical development.

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"This year we are focused on making significant advances to our pipeline as we work to bring multiple programs into Phase 2 clinical trials, move programs within our rare disease portfolio toward the clinic and leverage our mRNA platform to create both new development candidates and potential new modalities where we believe there is an opportunity to develop therapies for a broad range of diseases," said Stéphane Bancel, Moderna’s Chief Executive Officer. "I am pleased with the continued progress of our pipeline, our ability to now manufacture mRNA for clinical development at our new site in Norwood and the relentless work of our team. I believe we are better positioned than we have ever been to deliver on the promise of our science to bring forward mRNA medicines that have the potential to improve the lives of patients."

Mr. Bancel will present a company overview and strategy update today at 10:30am PST at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco. A live webcast of today’s presentation can be found at investors.modernatx.com.

Detailed program updates:

Intratumoral Immuno-oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.

OX40L (mRNA-2416): Based on previously reported clinical observations in two patients with advanced ovarian carcinoma in its Phase 1 study, Moderna has submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) and to the study’s clinical research sites to commence a Phase 2 cohort of mRNA-2416 as a monotherapy in advanced ovarian carcinoma within its current Phase 1 study. Thus far, 28 patients have been dosed in the ongoing Phase 1 trial for mRNA-2416, an open-label, multicenter study of repeated intratumoral injections of mRNA-2416 in patients with advanced relapsed/refractory solid tumor malignancies and lymphomas. Initial data from the Phase 1 study were presented in a poster session at the Annual Meeting of the Society for Immunotherapyof Cancer in November 2018.
OX40L + IL23 + IL36γ (Triplet) (mRNA-2752):Moderna has dosed the first patient in the Phase 1 study of mRNA-2752, an intratumoral injection comprising three mRNAs encoding for OX40L + IL23 + IL36γ for the treatment of advanced or metastatic solid tumor malignancies or lymphoma. The open label, multi-center study is evaluating the safety and tolerability of mRNA-2752 as a single agent and in combination either with AstraZeneca’s durvalumab or tremelimumab, and will assess anti-tumor activity, protein expression in tumors and pharmacokinetics, and exploratory endpoints that include assessment of immunological response.
Cancer Vaccines: These programs focus on stimulating a patient’s immune system to tumor-related antigens to enable the immune system to elicit a more effective antitumor response.

Personalized Cancer Vaccine (PCV) (mRNA-4157): Moderna and Merck areplanning a randomized Phase 2 study comparing PCV and KEYTRUDA against KEYTRUDA alone. To date, interim Phase 1 PCV study data from 24 patients showed no dose limiting toxicities up to 0.39 mg (the third of four dose levels). Interim Phase 1 immunogenicity data have also been collected in certain patients dosed with mRNA-4157 as a monotherapy, and potential antigen-specific T cell responses have been detected. The Phase 1 study continues in the dose-escalation phase of the protocol.
KRAS vaccine (mRNA-5671): Merck will lead an open-label, multi-center, dose-escalation and dose-expansion Phase 1 study to evaluate the safety and tolerability of mRNA-5671 administered as an intramuscular injection both as a monotherapy and in combination with KEYTRUDA. KRAS is a frequently mutated oncogene in epithelial cancers, primarily in non-small cell lung, colorectal and pancreatic cancers. The IND for a KRAS vaccine was originally submitted by Moderna and included an mRNA for the membrane protein STimulator of INterferon Gene (STING) to help promote antitumor activity. That IND was transferred to Merck which now will move the program forward under the same IND with KRAS as the sole mRNA. Merck may choose to include STING mRNA in later clinical development of the KRAS vaccine.
Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.

Methylmalonic Acidemia (MMA) (mRNA-3704): An IND application has been submitted to the FDA for mRNA-3704, Moderna’s development candidate for MMA. If approved, this will be Moderna’s first rare disease program to advance into clinical trials. The Company plans to conduct an open-label, multi-center, dose escalation Phase 1/2 study of multiple ascending doses of mRNA-3704 in pediatric patients with isolated MMA due to MUT enzyme deficiency. The objectives of the study are to evaluate safety and tolerability. mRNA-3704 has received Rare Pediatric Disease Designation by the FDA and Orphan Drug Designation by both the FDA and the European Medicines Agency.
Propionic Acidemia (PA) (mRNA-3927): mRNA-3927 was granted Orphan Drug Designation by the FDA in December 2018 and Rare Pediatric Disease Designation by the FDA in January 2019. PA is a rare, life-threatening, inherited metabolic disorder due to a defect in the mitochondrial enzyme propionyl-CoA carboxylase, or PCC. It primarily affects the pediatric population and there is no approved therapy. Moderna is continuing to advance mRNA-3927 in pre-clinical studies. Moderna also continues to enroll patients in a global natural history study of MMA and PA (MaP Study) designed to identify and correlate clinical and biomarker endpoints for these disorders. This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to methylmalonyl-CoA mutase (MUT) deficiency or PA.
More than 760 subjects have been dosed with a therapeutic or vaccine candidate developed with Moderna’s mRNA technology.

Information about each program in Moderna’s pipeline, including those discussed in this press release, can be found on our investor relations page of our website www.modernatx.com.

Corporate Objectives:

Moderna shared its corporate objectives for 2019 – 2020, which include:

1. Generate human proof-of-concept data for multiple medicines
2. Execute on current development pipeline
3. Create new development candidates in existing modalities
4. Invent new modalities

Corporate Updates:

Continued growth across organization:Moderna ended 2018 with approximately 735 full time employees. The Company ended 2017 with 535 employees.

Continued strong cash position: We expect our cash, cash equivalents, and investments in marketable securities as of December 31, 2018 to be approximately $1.7 billion (unaudited), as compared to $902 million (audited) as of December 31, 2017. The year over year increase includes approximately $564 million (unaudited) in net proceeds from our initial public offering completed in December 2018, approximately $661 million in net proceeds from our preferred stock issuances in 2018, and a $13 million (unaudited) premium associated with the 2018 amended and restated personalized cancer vaccines agreement with Merck & Co.

On January 8, 2019 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported a corporate update on the Company’s clinical development of galinpepimut-S (GPS) and nelipepimut-S (NPS), which are both in late-stage development (Press release, Sellas Life Sciences, JAN 8, 2019, View Source [SID1234532580]).

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"In 2018, SELLAS achieved significant progress towards our corporate and clinical goals, as we advanced our immunotherapy pipeline and completed our first year as a public company," said Dr. Angelos M. Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "As we look to 2019, we are excited to initiate our pivotal Phase 3 program for GPS in acute myeloid leukemia (AML), and are in active discussions with the U.S. Food and Drug Administration (FDA) regarding the registration-enabling Phase 3 trial and potential consideration for accelerated approval and breakthrough designation for NPS to treat triple negative breast cancer. We also are exploring the potential of GPS in combination with pembrolizumab in additional tumor types in the context of a Phase 1/2 basket clinical study and look forward to continued progress across our broad pipeline in the year ahead."

Galinpepimut-S (GPS)

In November 2018, SELLAS aligned with the FDA on the clinical trial design and biostatistical plan for a Phase 3 registrational study for GPS in acute myeloid leukemia (AML). The planned Phase 3 registrational study will be a 1:1 randomized, open-label study comparing GPS in the maintenance setting to investigators’ choice of best available treatment (BAT) in adult AML patients who have achieved hematologic complete remission, with or without thrombocytopenia (CR2/CR2p), after second-line antileukemic therapy and who are deemed ineligible for or unable to undergo allogeneic stem-cell transplantation. This study will serve as the basis for a Biologics License Application (BLA) submission, subject to positive results, and SELLAS expects to start the Phase 3 trial in the second quarter of 2019.

The Phase 3 study is expected to enroll approximately 116 patients at approximately 50 clinical sites in the United States and Europe. The primary endpoint is overall survival (OS) and secondary endpoints include leukemia-free survival, antigen-specific T-cell immune response dynamics over time and rates of achievement of measurable residual disease (MRD) negativity. The study will have a planned interim safety and futility analysis after 80 events, expected to occur in the third quarter of 2020. GPS was previously granted Fast Track and Orphan Drug designations by the FDA for the treatment of AML.

In December 2018, SELLAS initiated enrollment of the Phase 1/2 open-label, non-comparative, multicenter, multi-arm study of GPS in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with selected WT1-positive advanced cancers, including both hematologic malignancies and solid tumors. This study, which is being conducted under a Clinical Trial Collaboration and Supply Agreement (CTSA) with Merck (known as MSD outside the United States and Canada), will assess the efficacy and safety of the combination, with exploratory long-term follow-up for overall survival and safety. The study will enroll approximately 90 patients at up to 20 centers in the United States. The initial tumor types to be treated will be acute myelogenous leukemia (AML) (patients unable to attain deeper morphological response than partial on hypomethylating agents and who are not eligible for allogeneic hematopoietic stem cell transplant) and ovarian cancer (second or third line), to be followed by triple negative breast cancer (second line), small cell lung cancer (second line), and colorectal cancer (third or fourth line).

Nelipepimut-S (NPS)

Based on promising Phase 2b data presented in 2018, SELLAS is currently in continuing active discussions with the FDA regarding the optimal development path for NPS in triple negative breast cancer (TNBC).

In the Phase 2b study of trastuzumab (Herceptin) +/- nelipepimut-S (NPS) in HER2 low-expressing breast cancer patient cohorts, trastuzumab + NPS demonstrated clinically and statistically significant efficacy in the TNBC cohort, with a p-value of 0.013 and a 75.2% reduction in risk of relapse or death. In October 2018, the Data Safety Monitoring Board (DSMB) unanimously concluded that the final analysis of the Phase 2b study data with a median follow-up of 26 months confirmed that TNBC patients should be the key target population for the development of trastuzumab + NPS in the adjuvant setting in early-stage HER2 1+/2+ breast cancer patients.

A preplanned secondary efficacy analysis across human leukocyte antigen (HLA) allele subgroups from the Phase 2b study confirmed the therapeutic potential of NPS in patients with early-stage TNBC in the adjuvant setting across HLA types A-02, -03, -24 and -26, which cover approximately 80-85% of the North American/European populations and 86-90% of Asian/Pacific basin populations.

Additional positive data from the Phase 2b study showed a clinically meaningful and statistically significant decrease in the number of clinically detectable relapses in the TNBC cohort with the combination of trastuzumab + NPS (7.5%) vs. trastuzumab alone (27.3%) (p=0.004). In addition, four pre-defined subgroups of TNBC patients in the trastuzumab + NPS arm demonstrated an average decrease of 84.2% in relative risk of relapse or death at 24 months (p=0.004-0.014).

In a Type C meeting with the FDA, SELLAS discussed several key points of the clinical and regulatory strategy for NPS in combination with trastuzumab for TNBC, including potential for accelerated approval; a registration-enabling Phase 3 trial design and biostatistical plan; and the potential for breakthrough designation. SELLAS expects a further meeting with the FDA in early 2019 regarding the potential for breakthrough designation as well as an additional meeting in the first quarter of 2019 to reach agreement for a final development program for NPS in TNBC.

An abstract summarizing the comprehensive set of results from the final analysis of the NPS + trastuzumab Phase 2b clinical study has been accepted for an oral presentation at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium in San Francisco, CA, on March 2, 2019 at 10:15 AM.

NPS was previously granted Fast Track designation by the FDA for the adjuvant treatment of patients with early stage breast cancer with low to intermediate HER2 expression following standard of care upfront therapy (surgery plus chemotherapy +/- radiotherapy).

Expected 2019 Clinical Milestones

Regulatory and development program with FDA for NPS in TNBC patients finalized in Q1 2019.
Phase 3 registration-enabling study of GPS in AML patients initiated in Q2 2019, with a planned interim analysis expected in Q3 2020.
Interim analysis of Phase 1/2 basket study of GPS with pembrolizumab in multiple tumor types in Q4 2019.
Herceptin and Keytruda are registered trademarks of Genentech, Inc. and Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, N.J., USA., respectively, and are not trademarks of SELLAS. The manufacturers of these brands are not affiliated with and do not endorse SELLAS or its products.