Adaptive Biotechnologies to Present at the 37th Annual J.P. Morgan Healthcare Conference

On January 3, 2019 Adaptive Biotechnologies reported that Chad Robins, chief executive officer and co-founder, will present at the 37th Annual J.P. Morgan Healthcare Conference on January 8, 2019 at 11:30 am PT (2:30 pm ET) in San Francisco (Press release, Adaptive Biotechnologies, JAN 3, 2019, View Source [SID1234532413]). Robins will also participate in a panel presentation, Reinventing the Disease Continuum Through Precision Health, on January 7, 2019 at 5:00 pm PT (8:00 pm ET) to discuss the impact of immune profiling on the future of immune-driven medicine.

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A live webcast of the presentation and Q&A session can be accessed from the Newsroom under Events on the Adaptive website at adaptivebiotech.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 90 days.

Sutro to Present at 37th Annual J.P. Morgan Healthcare Conference

On January 3, 2019 Sutro Biopharma, Inc. (NASDAQ: STRO) reported that Bill Newell, Chief Executive Officer, will present at the 37th Annual J.P. Morgan Healthcare Conference on Thursday, Jan. 10, 2019 at 11:30 a.m. PT / 2:30 p.m. ET at the Westin St. Francis Hotel in San Francisco (Press release, Sutro Biopharma, JAN 3, 2019, View Source [SID1234532429]).

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A live webcast of the presentation will be accessible through the Events and Presentations page of the Investor Relations section of the company’s website at www.sutrobio.com. A replay of the webcast will be available for approximately 30 days following the event.

Cydan Announces Formation of Tiburio Therapeutics Inc. to Develop Treatments for Rare Neuroendocrine Tumors and Endocrine Diseases

On January 3, 2019 Cydan reported the launch of Tiburio Therapeutics Inc. (Tiburio), a private biopharmaceutical company focused on developing treatments for rare neuroendocrine tumors and rare endocrine diseases (Press release, Cydan Development, JAN 3, 2019, View Source [SID1234553930]). Tiburio will advance two compounds licensed from Ipsen for the treatment of non-functioning pituitary adenoma (NFPA) (TBR-760) and for additional rare endocrine diseases (TBR-065). As part of the launch, Tiburio raised a $31M Series A financing that will fund the company’s lead compound, TBR-760, through human proof-of-concept for the treatment of NFPA and further clinical assessment of TBR-065 as a treatment for rare endocrine diseases. In conjunction with the Tiburio financing, Abraham N. Ceesay has been appointed chief executive officer of the company. Tiburio is the third orphan drug company launched by Cydan, an orphan drug accelerator dedicated to creating therapies to improve the lives of patients living with orphan diseases.

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"Patients suffering from rare neuroendocrine tumors and endocrine diseases represent a significantly underserved patient population due to the lack of effective treatment options," said Abraham N. Ceesay, chief executive officer of Tiburio. "TBR-760 and TBR-065 represent potential breakthroughs in the treatment of debilitating neuroendocrine diseases and we, at Tiburio, are intently focused on advancing these compounds for the benefit of patients. We will immediately begin Phase 2 enabling studies with TBR-760 and anticipate enrolling patients with NFPA in a Phase 2 study in the 2nd half of 2019."

The development of NFPA, which is a non-metastatic tumor in the pituitary gland in the brain, can result in life-altering and potentially life-threatening consequences for patients. Currently there are no approved therapies to treat these tumors and patients must undergo transsphenoidal surgery and/or radiation to remove or shrink the tumor. TBR-760 is a dopamine-somatostatin chimeric molecule that inhibits NFPA cell proliferation and has the potential to shrink or halt tumor growth. In prior clinical trials, TBR-760 has been shown to have a favorable safety profile.

"The agreement with Cydan to license these two innovative compounds to Tiburio expands upon the work we’ve done to advance them through Phase 2 trials, providing a foundation for their safety profile and mechanism of action," said Alexandre LeBeaut, MD, executive vice president, research & development and chief scientific officer, Ipsen. "The Cydan team’s expertise makes them a natural choice to partner with to ensure these programs can have impact for patients in need."

"Nonfunctioning pituitary adenoma can have a significant and lasting impact on patients, often requiring multiple surgeries to control tumor size, as well as radiation therapy. These invasive neurosurgeries are associated with high-risk complications," said Shi Yin Foo, MD, PhD, MMSc, chief medical officer of Cydan and acting chief medical officer of Tiburio. "There are no approved therapies and the current treatment options for these patients are invasive, risky, and often ineffective in the longer term. We can do better for patients and believe TBR-760 represents a potentially game-changing therapy for patients."

"TBR-760’s unique mechanism of action offers an exciting potential treatment for patients living with NFPA," said Chris Adams, co-founder and chief executive officer of Cydan. "Following an extensive review of the prior TBR-760 preclinical and clinical data, the Cydan team launched our third company, Tiburio, to execute a rapid clinical and regulatory program to bring this drug to patients in need."

Tiburio was granted an exclusive world-wide license to both TBR-760 and TBR-065. Ipsen will receive a minority ownership in the company as well as development and commercial milestone payments and royalties on sales. Tiburio is responsible for all future development and commercialization costs for both compounds. Additional financial terms were not disclosed.

Investment in the $31M Series A came from Cydan’s syndicate of leading life sciences investors, New Enterprise Associates, Longitude Capital, Lundbeckfonden Ventures, and Alexandria Venture Investments.

About NFPA

The pituitary gland, a master regulator of endocrine function, is located below the base of the brain. A nonfunctioning pituitary adenoma (NFPA) is a non-metastatic pituitary tumor that arise from gonadotroph cells in the pituitary. Patients often present with symptoms related to the size of the NFPA tumor, including headaches, vision problems, changes in milk production, weight change, menstrual changes, fatigue, sleep disruption, and depression or cognitive/behavioral changes.

Current treatment is limited to transsphenoidal surgery (TSS), which is performed through the nose and sinus passage, and/or radiation to halt the growth of these tumors. However, these two treatment options come with serious and high-risk consequences. In addition to these interventions designed to remove or reduce the size of the tumors, patients often require a complex set of medications to manage the endocrine disruption and other symptoms caused by the tumors and side effects of TSS and radiation.

In the U.S. there are approximately 5,000 new cases of NFPA that require TSS each year and 40-50% of these tumors will regrow within the first 5 years after surgery. Additionally, there are approximately 50,000 patients living with NFPA who may benefit from therapy to control tumor growth.

About Abraham Ceesay, CEO Tiburio

Mr. Ceesay has over 17 years of experience in leading biopharmaceutical companies and commercializing innovative therapeutic products. Prior to joining Tiburio, he served as chief operating officer at scPharmaceuticals where he developed and led all operational and commercial aspects of the company. Mr. Ceesay was integral in raising more than $140 million in private and public capital as well as closing the company’s initial public offering. Prior to joining scPharmaceuticals, he served as vice president, Sales, Marketing, and Commercial Operations at Keryx Biopharmaceuticals and spent four years at Ironwood Pharmaceuticals as Vice President of Marketing, and held responsibility for the management of the U.S. P&L, leadership of the Linzess brand team and co-promotion collaboration with Forest Laboratories/Allergan. Previously he was at Genzyme/Sanofi, initially as a field sales specialist and ultimately as the director, Renal Global Marketing, in which capacity he led the global launch of Renvela and held global marketing responsibility for the company’s renal franchise (Renagel, Renvela, Hectorol). Mr. Ceesay serves on the Board of Advisors for Life Science Cares and the Board of Directors for Food for Free. He holds a bachelor’s degree from Ithaca College, and a Master of Business Administration from Suffolk University’s Sawyer School of Management.

Sesen Bio Announces Positive Preliminary 12-Month Data from Registration Phase 3 VISTA Trial of Vicinium for Non-Muscle Invasive Bladder Cancer

On January 3, 2019 Sesen Bio, Inc. (Nasdaq: SESN), a late-stage clinical company advancing targeted fusion protein therapeutics for the treatment of cancer, reported positive preliminary efficacy data for the primary endpoint of its ongoing Phase 3 registration trial, the VISTA Trial, of Vicinium for the treatment of patients with high-grade non-muscle invasive bladder cancer (NMIBC) who have been previously treated with bacillus Calmette-Guérin (BCG) and deemed BCG-unresponsive (Press release, Eleven Biotherapeutics, JAN 3, 2019, View Source [SID1234532380]). The data reported show clinically meaningful complete response rates in evaluable Carcinoma in situ patients at three, six, nine and 12 months of follow-up in the trial consistent with the data in the completed Phase 1 and Phase 2 clinical trials. Importantly, Vicinium continues to be generally well-tolerated in treated patients.

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"Non-muscle invasive bladder cancer is a very prevalent cancer that can progress to become incurable. The usual treatment for patients who relapse or become refractory to BCG, today’s standard-of-care, is complete bladder removal or radical cystectomy," said Michael A.S. Jewett, M.D., Professor of Surgery, Division of Urology, University of Toronto. "Removing the bladder is a potentially morbid and complex surgery with potential for side effects that can drastically reduce a patient’s quality of life. In fact, many patients choose not to undergo bladder removal. I am very encouraged by the data generated to-date with intravesical Vicinium as an alternative after BCG failure. Based on the strength of the clinical activity observed, and the consistently favorable safety and tolerability, I believe that Vicinium has the potential to change the treatment outcome for patients."

VISTATrial Design
The Phase 3 VISTA Trial is a single-arm, multi-center clinical trial designed to support the approval of Vicinium for the treatment of patients with high-grade, BCG-unresponsive NMIBC. The trial enrolled a total of 133 patients across three cohorts based on histology and time to disease recurrence after adequate BCG:

Cohort 1 (n=86): Carcinoma in situ patients with or without papillary disease whose cancer was determined to be refractory or recurred within six months of their last course of adequate BCG
Cohort 2 (n=7): Carcinoma in situ patients with or without papillary disease whose cancer was determined to be refractory or recurred after six months, but less than 12 months, after their last course of adequate BCG
Cohort 3 (n=40): patients with papillary disease without Carcinoma in situ whose cancer was determined to be refractory or recurred within six months of their last course of adequate BCG
The data reported build upon preliminary three-month data presented from a subset of patients in May 2018 and are for the primary endpoint of the VISTA Trial, which is the complete response rate and duration of response in patients in Cohort 1. The company also reported data from Cohort 2, separately and pooled with Cohort 1, based on final U.S. Food and Drug Administration guidance on treatment of BCG-unresponsive Carcinoma in situ patients (defined as patients with recurrent Carcinoma in situ within 12 months of adequate BCG therapy)1.

The patient population in Cohort 3 represents an opportunity for future label expansion, and the company plans to report efficacy data from this cohort, as well as the secondary endpoints in the VISTA Trial, in mid-2019.

Preliminary Efficacy Results in Carcinoma in situ Patients

Cohort 1 (n=86)


Time point Evaluable Patients Complete Response Rate (95% Confidence Interval)
3-months n=86 37% (27%, 48%)
6-months n=85 25% (16%, 35%)
9-months n=84 18% (10%, 28%)
12-months n=81 14% (7%, 23%)

Cohort 2 (n=7)


Time point Evaluable Patients Complete Response Rate (95% Confidence Interval)
3-months n=7 57% (18%, 90%)
6-months n=7 57% (18%, 90%)
9-months n=7 43% (10%, 82%)
12-months n=7 14% (0%, 58%)

Pooled Cohorts 1 and 2 (n=93)

Time point Evaluable Patients Complete Response Rate (95% Confidence Interval)
3-months n=93 39% (29%, 49%)
6-months n=92 27% (18%, 37%)
9-months n=91 20% (12%, 29%)
12-months n=88 14% (7%, 23%)

Notably, the interim Phase 3 complete response rates in pooled patients from Cohorts 1 and 2 are in-line with the complete response rates in pooled patients in the completed Phase 2 clinical trial.

Preliminary Phase 3 CRR vs Phase 2 CRR

Time point Phase 3 Pooled CRR
(95% Confidence Intervals)

Phase 2 Pooled CRR
(95% Confidence Interval)

3-months 39% (29%, 49%) 40% (26%, 56%)
6-months 27% (18%, 37%) 27% (15%, 42%)
9-months 20% (12%, 29%) 18% (8%, 32%)
12-months 14% (7%, 23%) 16% (7%, 30%)

The company also reported an update on the durability of responses in the VISTA Trial. While the median has not yet been reached, the preliminary data show that Vicinium treatment resulted in a prolonged duration of response in many patients. This is particularly notable given that, in order for patients to remain on study, they have to have achieved a complete response at each assessment time period. These findings suggest that Vicinium has the potential to benefit patients by delaying the time to a radical cystectomy, a secondary endpoint that will be measured and reported in mid-2019.

Preliminary Safety Results
Vicinium continues to be well-tolerated by patients treated in the VISTA Trial. As of the December 3, 2018 data cut off, in patients across all three cohorts (n=133), 78 percent of adverse events were Grade 1 or 2. The most commonly reported treatment-related adverse events were dysuria (13%), hematuria (12%) and urinary tract infection (11%) – all of which are consistent with the profile of bladder cancer patients and the use of catheterization for treatment delivery. These adverse events were determined to be manageable and reversible, and only five patients discontinued treatment due to an adverse event. Serious adverse events, regardless of treatment attribution, were reported in 14 percent of patients. There were four treatment-related SAEs reported in three patients including acute renal injury (Grade 3), pyrexia (Grade 2), cholestatic hepatitis (Grade 4) and renal failure (Grade 5).

"We are very pleased with these preliminary data, which are consistent with the data in our completed Phase 2 clinical trial of Vicinium for the treatment of high-grade NMIBC, and further support our belief that Vicinium has the potential to change how patients are treated after BCG," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "The design of the VISTA Trial aligns with FDA’s guidance for NMIBC drug development, and the findings are highly encouraging, demonstrating that treatment with Vicinium results in clinically meaningful efficacy and favorable safety and tolerability. Overall, the data reinforce our belief that Vicinium is positioned to provide a valuable benefit to patients by treating their disease with long-term responses and extending their time to face such a decision as removing their bladder. 2019 is set to be a transformational year for Sesen Bio, and we look forward to advancing the VISTA Trial and assessing the full 12-month data from all patients later this year."

The VISTA Trial completed enrollment in the second quarter of 2018, and complete 12-month efficacy data from all patients in the clinical trial are expected to be reported at a medical meeting in mid-2019.

Conference Call Information
To participate in the conference call, please dial (844) 831-3025 (domestic) or (315) 625-6887 (international) and refer to conference ID 4263106. The webcast can be accessed in the Investor Relations section of the company’s website at www.sesenbio.com. The replay of the webcast will be available in the investor section of the company’s website at www.sesenbio.com for 60 days following the call.

About the VISTA Clinical Trial
The VISTA Trial is an open-label, multicenter, single-arm Phase 3 clinical trial evaluating the efficacy and tolerability of Vicinium in patients with high-grade non-muscle invasive bladder cancer (NMIBC) that is Carcinoma in situ, which is cancer found on the inner lining of the bladder that has not spread into muscle or other tissue and/or papillary, which is cancer that has grown from the bladder lining out into the bladder but has not spread into muscle or other tissue, who have been previously treated with bacillus Calmette-Guérin (BCG). The primary endpoint of the trial is the complete response rate in patients with Carcinoma in situ with or without papillary disease. Patients in the trial receive locally administered Vicinium twice a week for six weeks, followed by once-weekly treatment for another six weeks, then treatment every other week for up to two years. Twelve-month data from all patients in the VISTA Trial are anticipated in mid-2019. To learn more about the Phase 3 VISTA Trial, please visit www.clinicaltrials.gov and search the identifier NCT02449239.

About Vicinium
Vicinium, a locally-administered fusion protein, is Sesen Bio’s lead product candidate being developed for the treatment of high-grade non-muscle invasive bladder cancer (NMIBC). Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A (ETA). Vicinium is constructed with a stable, genetically engineered peptide tether to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently conducting the Phase 3 VISTA Trial, designed to support the registration of Vicinium for the treatment of high-grade NMIBC in patients who have previously received two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Complete twelve-month data from the trial are anticipated in mid-2019. Additionally, Sesen Bio believes that Vicinium’s cancer cell-killing properties promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

About Non-Muscle Invasive Bladder Cancer
Bladder cancer is the sixth most commonly diagnosed cancer in the United States, and approximately 80 percent of patients have non-muscle invasive bladder cancer (NMIBC). In NMIBC, cancer cells are in the lining of the bladder or have grown into the lumen of the bladder but have not spread into muscle or other tissue. NMIBC primarily affects men and is associated with carcinogen exposure. Initial treatment includes surgical resection; however, there is a high rate of recurrence and more than 60 percent of all patients diagnosed with NMIBC will receive bacillus Calmette-Guérin (BCG) immunotherapy. While BCG is effective in many patients, challenges with tolerability have been observed and many patients will experience recurrence of disease. If BCG is not effective or a patient can longer receive BCG, the recommended option for treatment is radical cystectomy, the complete removal of the bladder.

Genmab to Present at the 37th Annual J.P. Morgan Healthcare Conference

On January 3, 2019 Genmab A/S (Nasdaq Copenhagen: GEN) reported that its CEO, Jan van de Winkel, Ph.D., will present a company update at the 37th Annual J.P. Morgan Healthcare Conference in San Francisco at 3:30 PM PST on January 9, 2019 (12:30 AM CET, January 10) (Press release, Genmab, JAN 3, 2019, View Source [SID1234532397]). A webcast of the presentation will be available on Genmab’s website at View Source

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