On May 13, 2019 Xenetic Biosciences, Inc. (NASDAQ: XBIO) ("Xenetic" or the "Company"), a clinical-stage biopharmaceutical company focused on the discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics, reported its financial results for the quarter ended March 31, 2019 and provided a corporate update (Press release, Xenetic Biosciences, MAY 13, 2019, View Source [SID1234536226]).

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"We expect 2019 to be a transformative year for Xenetic. With the anticipated closing of our acquisition of XCART, our differentiated CAR T platform technology, on track for this quarter, I believe Xenetic will be well positioned to drive momentum in the innovation and development of new oncology therapeutics where there remains significant unmet need," commented Jeffrey Eisenberg, Chief Executive Officer of Xenetic. "We plan to focus our R&D efforts initially on leveraging the XCART platform to develop cell-based therapeutics for the treatment of B-cell Non-Hodgkin lymphomas. All of this marks a very important moment in the evolution of Xenetic, and I firmly believe the Company can become a significant player in the oncology space with this potentially transformative technology platform, providing therapeutic solutions for physicians and individuals fighting cancer and their families as well as driving value for our shareholders."

XCART Technology

On March 1, 2019, the Company entered into agreements to acquire a novel CAR T ("Chimeric Antigen Receptor T Cell") platform technology, called "XCART." XCART is a proximity-based screening platform capable of identifying CAR constructs that can target patient-specific tumor neoantigens, with a demonstrated proof of mechanism in B-cell Non-Hodgkin lymphomas. The XCART technology, developed by The Scripps Research Institute in collaboration with the Shemyakin-Ovchinnikov Institute of Bioorganic Chemistry, is believed to have the potential to significantly enhance the safety and efficacy of cell therapy for B-cell lymphomas by generating patient- and tumor-specific CAR T cells. The acquisition is subject to conditions typical for a transaction of this kind, including appropriate stockholder approvals, and is expected to close by the end of the second quarter.

The XCART technology platform was designed by its originators to utilize an established screening technique to identify peptide ligands that bind specifically to the unique B-cell receptor ("BCR") on the surface of an individual patient’s malignant tumor cells. The peptide is then inserted into the antigen-binding domain of a CAR, and a subsequent transduction/transfection process is used to engineer the patient’s T cells into a CAR T format which redirects the patient’s T cells to attack the tumor. Essentially, the XCART screening platform is the inverse of a typical CAR T screening protocol wherein libraries of highly specific antibody domains are screened against a given target. In the case of XCART screening, the target is itself an antibody domain, and hence highly specific by its nature. The XCART technology creates the possibility of personalized treatment of lymphomas utilizing a CAR with an antigen-binding domain that should only recognize, and only be recognized by, the unique BCR of a particular patient’s B-cell lymphoma.

An expected result for XCART is reduced off-tumor toxicities, such as B-cell aplasia. Xenetic’s clinical development program will seek to confirm the early preclinical results, and to demonstrate a more attractive safety profile than existing therapies.

Once the acquisition is consummated, the Company intends to pursue development efforts of the XCART technology as well as other development efforts in the area of CAR T therapy.

Summary of Financial Results for First Quarter 2019

Net loss for the three months ended March 31, 2019, was approximately $1.3 million compared to a net loss of approximately $1.8 million for the same period in 2018. Working capital (deficit) was approximately $1.2 million and $(0.4) million at March 31, 2019 and December 31, 2018, respectively. During the quarter ended March 31, 2019, the Company’s working capital increased by $1.6 million due to the issuance of common stock and warrants in our March 2019 offering resulting in $2.7 million of net proceeds to Xenetic. The Company ended the quarter with approximately $2.0 million of cash.

On May 13, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported that it will participate in the Bank of America Merrill Lynch Health Care Conference 2019 in Las Vegas, NV (Press release, Halozyme, MAY 13, 2019, View Source [SID1234536225]). Laurie Stelzer, senior vice president and chief financial officer, will provide an overview of the company on Thursday, May 16 at 12:20 p.m. ET / 9:20 a.m. PT.

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A live webcast of the presentation can be accessed through the "Investors" section of www.halozyme.com, and a recording will be made available for 90 days following each event. To access a live webcast, please visit Halozyme’s website approximately 15 minutes prior to the presentation to register and download any necessary audio software.

On May 13, 2019 Celgene Corporation (NASDAQ:CELG) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to POMALYST (pomalidomide) for the treatment of patients with human immunodeficiency virus (HIV)-positive Kaposi sarcoma who have previously received systemic chemotherapy, as well as patients with HIV‐negative Kaposi’s sarcoma (Press release, Celgene, MAY 13, 2019, View Source [SID1234536224]).

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Kaposi sarcoma is a multicentric tumor caused by Kaposi sarcoma-associated herpesvirus, also called human herpesvirus-8. Patients suffer multiple lesions on the skin and oral mucosa, and at times other organs such as the lungs or gastrointestinal mucosa. Kaposi sarcoma most commonly arises in persons infected with HIV. There is a substantial need for new treatments because there are no approved therapies for HIV-positive patients who are refractory to or intolerant of systemic chemotherapy. Although the use of combination anti-retroviral treatments (cART or HAART) has reduced the incidence of advanced Kaposi sarcoma in the United States, there are still nearly 2000 new cases each year. The disease is more highly prevalent in areas of the world where HIV treatments are less available, such as sub-Saharan Africa, and in some countries is the most common tumor in men overall.

"The encouraging news of the FDA Breakthrough Therapy designation for POMALYST in Kaposi sarcoma reflects the urgency in accelerating the development of therapies to address diseases of this type," said Jay Backstrom, M.D., Chief Medical Officer for Celgene. "We will continue to work closely with the agency to move this program forward for patients with this rare and serious cancer."

The Breakthrough Therapy designation was granted by the FDA on the basis of the results of a clinical study performed under a Cooperative Research and Development Agreement (CRADA) by a team led by Dr. Robert Yarchoan, of the HIV and AIDS Malignancy Branch within the Center for Cancer Research of the National Cancer Institutes (NCI). The results of that study, published in the Journal of Clinical Oncology (MN Polizzotto et al, JCO, 2016, 34, 4125-31), evaluated POMALYST in patients with Kaposi sarcoma, with or without HIV infection, many of whom had received prior cytotoxic chemotherapy.

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases.

Celgene plans to submit a supplemental New Drug Application for POMALYST in this disease area by the end of 2019.

Celgene also has two additional studies planned in this disease. In partnership with the AIDS Malignancy Consortium (AMC), a U.S. multicenter study will be performed to confirm and extend the results of the NCI study. The AMC is also sponsoring a second study in sub-Saharan Africa, where Kaposi sarcoma continues to be a serious problem. This program is a part of the Celgene Global Health effort to discover and develop new drugs for diseases that affect patients in the lower- and middle-income countries where health systems and medical resources are less advanced.

POMALYST is not approved for Kaposi sarcoma in any country.

About POMALYST

POMALYST is one of Celgene’s IMiD agents – proprietary small molecule, orally-available compounds for the treatment of some blood cancers. IMiD agents are hypothesized to have multiple mechanisms of action and have become a foundation of multiple myeloma research, with a growing number of studies exploring these compounds in different settings and diseases.

U.S. Safety Information

Indication

POMALYST (pomalidomide) is a thalidomide analogue indicated, in combination with dexamethasone, for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or within 60 days of completion of the last therapy.

Important Safety Information

WARNING: EMBRYO-FETAL TOXICITY and VENOUS AND ARTERIAL THROMBOEMBOLISM

Embryo-Fetal Toxicity

POMALYST is contraindicated in pregnancy. POMALYST is a thalidomide analogue. Thalidomide is a known human teratogen that causes severe birth defects or embryo-fetal death. In females of reproductive potential, obtain 2 negative pregnancy tests before starting POMALYST treatment.
Females of reproductive potential must use 2 forms of contraception or continuously abstain from heterosexual sex during and for 4 weeks after stopping POMALYST treatment.
POMALYST is only available through a restricted distribution program called POMALYST REMS .

Venous and Arterial Thromboembolism

Deep venous thrombosis (DVT), pulmonary embolism (PE), myocardial infarction, and stroke occur in patients with multiple myeloma treated with POMALYST. Prophylactic antithrombotic measures were employed in clinical trials. Thromboprophylaxis is recommended, and the choice of regimen should be based on assessment of the patient’s underlying risk factors.

CONTRAINDICATIONS

Pregnancy : POMALYST can cause fetal harm and is contraindicated in females who are pregnant. If POMALYST is used during pregnancy or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential risk to a fetus.
WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity & Females of Reproductive Potential: See Boxed WARNINGS
Males: Pomalidomide is present in the semen of patients receiving the drug. Males must always use a latex or synthetic condom during any sexual contact with females of reproductive potential while taking POMALYST and for up to 4 weeks after discontinuing POMALYST, even if they have undergone a successful vasectomy. Males must not donate sperm.
Blood Donation: Patients must not donate blood during treatment with POMALYST and for 4 weeks following discontinuation of POMALYST therapy because the blood might be given to a pregnant female patient whose fetus must not be exposed to POMALYST.
POMALYST REMS Program: See Boxed WARNINGS
Prescribers and pharmacies must be certified with the POMALYST REMS program by enrolling and complying with the REMS requirements; pharmacies must only dispense to patients who are authorized to receive POMALYST. Patients must sign a Patient-Physician Agreement Form and comply with REMS requirements; female patients of reproductive potential who are not pregnant must comply with the pregnancy testing and contraception requirements and males must comply with contraception requirements.
Further information about the POMALYST REMS program is available at www.CelgeneRiskManagement.com or by telephone at 1-888-423-5436.
Venous and Arterial Thromboembolism: See Boxed WARNINGS. Patients with known risk factors, including prior thrombosis, may be at greater risk, and actions should be taken to try to minimize all modifiable factors (e.g., hyperlipidemia, hypertension, smoking). Thromboprophylaxis is recommended, and the choice of regimen should be based on assessment of the patient’s underlying risk factors.
Increased Mortality with Pembrolizumab : In clinical trials in patients with multiple myeloma, the addition of pembrolizumab to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of patients with multiple myeloma with a PD-1 or PD-L1 blocking antibody in combination with a thalidomide analogue plus dexamethasone is not recommended outside of controlled clinical trials.
Hematologic Toxicity : Neutropenia (46%) was the most frequently reported Grade 3/4 adverse reaction in patients taking POMALYST in clinical trials, followed by anemia and thrombocytopenia. Monitor complete blood counts weekly for the first 8 weeks and monthly thereafter. Patients may require dose interruption and/or modification.
Hepatotoxicity : Hepatic failure, including fatal cases, has occurred in patients treated with POMALYST. Elevated levels of alanine aminotransferase and bilirubin have also been observed in patients treated with POMALYST. Monitor liver function tests monthly. Stop POMALYST upon elevation of liver enzymes. After return to baseline values, treatment at a lower dose may be considered.
Severe Cutaneous Reactions Including Hypersensitivity Reactions : Angioedema and severe cutaneous reactions including Stevens-Johnson Syndrome (SJS), toxic epidermal necrolysis (TEN), and drug reaction with eosinophilia and systemic symptoms (DRESS) have been reported. DRESS may present with a cutaneous reaction (such as rash or exfoliative dermatitis), eosinophilia, fever, and/or lymphadenopathy with systemic complications such as hepatitis, nephritis, pneumonitis, myocarditis, and/or pericarditis. Discontinue POMALYST for angioedema, skin exfoliation, bullae, or any other severe cutaneous reactions such as SJS, TEN or DRESS, and do not resume therapy.
Dizziness and Confusional State : In patients taking POMALYST in clinical trials, 14% experienced dizziness (1% Grade 3 or 4) and 7% a confusional state (3% Grade 3 or 4). Instruct patients to avoid situations where dizziness or confusional state may be a problem and not to take other medications that may cause dizziness or confusional state without adequate medical advice.
Neuropathy : In patients taking POMALYST in clinical trials, 18% experienced neuropathy (2% Grade 3 in one trial) and 12% peripheral neuropathy.
Second Primary Malignancies : Cases of acute myelogenous leukemia have been reported in patients receiving POMALYST as an investigational therapy outside of multiple myeloma.
Tumor Lysis Syndrome (TLS) : TLS may occur in patients treated with POMALYST. Patients at risk are those with high tumor burden prior to treatment. These patients should be monitored closely and appropriate precautions taken.
ADVERSE REACTIONS

The most common adverse reactions for POMALYST (≥30%) included fatigue and asthenia, neutropenia, anemia, constipation, nausea, diarrhea, dyspnea, upper-respiratory tract infections, back pain, and pyrexia.

In the phase III trial, nearly all patients treated with POMALYST + low-dose dex experienced at least one adverse reaction (99%). Adverse reactions (≥15% in the POMALYST + low-dose dex arm and ≥2% higher than control) included neutropenia (51.3%), fatigue and asthenia (46.7%), upper respiratory tract infection (31%), thrombocytopenia (29.7%), pyrexia (26.7%), dyspnea (25.3%), diarrhea (22%), constipation (21.7%), back pain (19.7%), cough (20%), pneumonia (19.3%), bone pain (18%), edema peripheral (17.3%), peripheral neuropathy (17.3%), muscle spasms (15.3%), and nausea (15%). Grade 3 or 4 adverse reactions (≥15% in the POMALYST + low-dose dex arm and ≥1% higher than control) included neutropenia (48.3%), thrombocytopenia (22%), and pneumonia (15.7%).

DRUG INTERACTIONS

Avoid concomitant use of POMALYST with strong inhibitors of CYP1A2. Consider alternative treatments. If a strong CYP1A2 inhibitor must be used, reduce POMALYST dose by 50%.

USE IN SPECIFIC POPULATIONS

Pregnancy: See Boxed WARNINGS. If pregnancy does occur during treatment, immediately discontinue the drug and refer patient to an obstetrician/gynecologist experienced in reproductive toxicity for further evaluation and counseling. There is a POMALYST pregnancy exposure registry that monitors pregnancy outcomes in females exposed to POMALYST during pregnancy as well as female partners of male patients who are exposed to POMALYST. This registry is also used to understand the root cause for the pregnancy. Report any suspected fetal exposure to POMALYST to the FDA via the MedWatch program at 1-800-FDA-1088 and also to Celgene Corporation at 1-888-423-5436.
Lactation : There is no information regarding the presence of pomalidomide in human milk, the effects of POMALYST on the breastfed child, or the effects of POMALYST on milk production. Pomalidomide was excreted in the milk of lactating rats. Because many drugs are excreted in human milk and because of the potential for adverse reactions in a breastfed child from POMALYST, advise women not to breastfeed during treatment with POMALYST.
Pediatric Use : Safety and effectiveness have not been established in pediatric patients.
Geriatric Use : No dosage adjustment is required for POMALYST based on age. Patients >65 years of age were more likely than patients ≤65 years of age to experience pneumonia.
Renal Impairment : Reduce POMALYST dose by 25% in patients with severe renal impairment requiring dialysis. Take dose of POMALYST following hemodialysis on hemodialysis days.
Hepatic Impairment : Reduce POMALYST dose by 25% in patients with mild to moderate hepatic impairment and 50% in patients with severe hepatic impairment.
Smoking Tobacco : Advise patients that smoking may reduce the efficacy of POMALYST. Cigarette smoking reduces the AUC of pomalidomide by 32% by CYP1A2 induction.
Please see full Prescribing Information , including Boxed WARNINGS.

About Celgene Global Health

Celgene Global Health (CGH) is a dedicated R&D unit of Celgene committed to discovering, developing and delivering novel drugs for Diseases of the Developing World (DDWs). Collaborating with non-profit and academic institutions around the globe, CGH has utilized the company’s library of more than 400,000 compounds to evaluate candidates for drug development for DDWs. More than ten discovery and development programs are ongoing in several disease areas such as malaria and tuberculosis. For more information, visit View Source

(Press release, Celgene, MAY 13, 2019, View Source [SID1234536224])

On May 13, 2019 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with an initial focus on myelodysplastic syndromes (MDS), reported that it has entered into a license agreement for the Greater China territory with HanX Biopharmaceuticals, Inc. (HanX), a China-based pharmaceutical company focused on the development, registration, and commercialization of novel oncology products in China, to develop and commercialize rigosertib, a novel and targeted anti-cancer compound currently in a Phase 3 study for the treatment of higher-risk MDS (HR-MDS) (Press release, Onconova, MAY 13, 2019, View Source [SID1234536223]).

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Under the terms of the agreement, Onconova has granted to HanX (i) an exclusive license to develop and commercialize rigosertib in Greater China and (ii) a non-exclusive license to manufacture rigosertib in Greater China. In exchange for these rights, HanX will make upfront payments totaling $6 million, $4 million of which are cash payments to Onconova, including a $2 million fee and an investment totaling $2 million in shares purchased at a premium to market. In addition, HanX will also place $2 million in escrow in local currency for rigosertib clinical development expenses in Greater China. Onconova could receive up to $45.5 million in regulatory, development. and sales-based milestone payments and tiered royalties up to double digits on net sales in Greater China. Onconova will initially supply the finished product for sale in the licensed territories. HanX will also support Onconova’s clinical trial initiatives in the territory

"We are excited about our expanded collaboration with HanX, which now includes our lead investigational drug candidate, rigosertib, currently being studied in higher-risk MDS patients," said Steven M. Fruchtman, M.D., President and Chief Executive Officer. "We have now partnered with HanX for the development of rigosertib in Greater China, in addition to our continued collaboration on the development of ON 123300, our novel CDK4/6 and ARK5 inhibitor. We plan for patients from China to be included in Onconova’s INSPIRE trial, and this additional source will support the goal of completing accrual to INSPIRE in the second half of 2019. In addition, we expect this agreement will broaden the exploration of additional indications for rigosertib, such as lung cancer. We look forward to presenting our data on rigosertib for the first time in China at the Acute Leukemia Forum being held in Shanghai on May 31, 2019."

Faming Zhang, Ph.D., Founder and Chairman of HanX, commented, "Our license agreement with Onconova for rigosertib is our second collaboration with Onconova following the IND enabling co-development of a novel CDK4/6 and ARK5 inhibitor program. Approximately 25-30 percent of all cancers involve a mutation of the Ras gene family. Rigosertib represents a novel mechanism of action to attenuate Ras signaling pathway and thus has the potential to affect a variety of cancers. Our pre-clinical testing shows that rigosertib acts synergistically in combination with HX-008, our PD-1 antibody which is now in phase II/III clinical trials in China, as well as being studied in solid tumor animal models. In collaboration with Onconova, our goal is to join the INSPIRE trial and the planned combination study of oral rigosertib with azacitidine for patients in Greater China with first line higher-risk MDS and also to initiate a proof-of-concept clinical study of the combination of rigosertib with our PD-1 inhibitor in NSCLC with Ras mutations."

Rigosertib is currently being evaluated in the global, pivotal Phase 3 INSPIRE clinical trial in patients who have failed or relapsed after receiving currently approved therapeutic options, with a goal of full accrual in the second half of 2019. Rigosertib is also being evaluated in an expanded Phase 2 combination study with azacitidine in HR-MDS patients. Onconova has a research collaboration agreement with the National Cancer Institute to study rigosertib in rare pediatric Ras driven cancers. Rigosertib has been granted orphan drug designation for MDS in the United States and Europe. Onconova is partnered for rigosertib with SymBio Pharmaceuticals for Japan and Korea and with Pint Pharma for Latin America.

On May 13, 2019 Sesen Bio (Nasdaq:SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of patients with cancer, reported operating results for the first quarter ended March 31, 2019 (Press release, Eleven Biotherapeutics, MAY 13, 2019, View Source [SID1234536222]). The Company also reported updated, preliminary primary and additional secondary endpoint data from the Company’s Phase 3 VISTA trial further supporting the strong benefit-risk profile of Viciniumfor the potential treatment in patients with high-risk,bacillus Calmette-Guérin(BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC). The updated preliminary Phase 3 clinical data, along with the Phase 2 clinical trial data, will serve as the basis for upcoming meetings with the U.S. Food and Drug Administration (FDA).

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"We are very encouraged by the most recent analysis of our 12-month Phase 3 VISTA trial data, which will be the basis for our meetings with the FDA in May and June," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "We believe these preliminary data, along with a closely matched Phase 2 data set, support a compelling benefit-risk profile, and give us confidence in the regulatory approvability and strong commercial viability of Vicinium. The huge unmet need for patients with NMIBC is widely acknowledged and has been exacerbated by the recurring global shortage of BCG. We will continue to work closely with the FDA in our effort to expeditiously bring a product to market that has the potential to save and improve the lives of patients with NMIBC."

Phase 3 VISTA Trial Progress and Updates

Updated, Preliminary VISTA Trial Data Reported in BCG-unresponsive NMIBC: In March, Sesen Bio announced updated preliminary data from its ongoing Phase 3 VISTA trial, a single-arm, multi-center clinical trial designed to support the approval of Vicinium for the treatment of patients with high-risk, BCG-unresponsive NMIBC. The trial completed registration in the second quarter of 2018, with a total of 133 patients across three cohorts based on histology and time to disease recurrence after adequate BCG treatment (at least two courses of BCG with at least five doses in the first course and two doses in the second course). Primary endpoints include complete response rate and duration of response for patients in Cohort 1. Secondary endpoints include time to disease recurrence for patients in Cohort 3, and time to cystectomy, progression-free survival, event-free survival, and overall survival for all patients across cohorts. As of the March 1, 2019 data cutoff, updated preliminary primary and secondary efficacy data for each of the trial cohorts were as follows:

Patients with Carcinoma in situ with or without papillary disease that was determined to be refractory or recurred less than 11 months, after their last course of adequate BCG

Duration of Response: The median duration of response for patients in Cohort 1 (n=86) is 287 days (95% CI, 127-NA), using the Kaplan-Meier method. Additional ad hoc analysis of pooled data for all patients with Carcinoma in situ (Cohorts 1 and 2, n=93) shows that among patients who achieved a complete response at 3 months, 54% had a complete response for a total of 12 months or longer after starting therapy, using the Kaplan-Meier method.
Time to Disease Recurrence: High-risk papillary (Ta or T1) NMIBC is associated with higher rates of progression and recurrence. Therefore, time to disease recurrence is a key secondary endpoint for patients with high-risk papillary-only NMIBC. The median time to disease recurrence for patients in Cohort 3 (n=40) is 402 days (95% CI, 170-NA), using the Kaplan-Meier method.
Time to Cystectomy: The FDA guidance states that the goal of therapy in patients with BCG-unresponsive NMIBC is to avoid cystectomy. Therefore, time to cystectomy is a key secondary endpoint in the VISTA trial. Across all 133 patients treated with Vicinium, >75% of patients are estimated to remain cystectomy-free at 2.5 years, using the Kaplan-Meier method. Additional ad hoc analysis of responders and non-responders for all patients shows that responders are approximately 15 times more likely to remain cystectomy-free at 2.5 years compared to non-responders.
Additional Secondary Endpoint Data from Phase 3 VISTA Trial Support a Growing Body of Evidence Demonstrating the Durable Anti-tumor Activity of Vicinium: Since the last data update reported in March, Sesen Bio has completed preliminary analyses of the remaining secondary endpoints in the VISTA trial, including progression-free survival, overall survival, and event-free survival, as measured across all patient cohorts. Reported data is as of the March 1, 2019 cutoff:
Progression-Free Survival: >85% of all 133 patients treated with Vicinium are estimated to remain progression-free at 2 years, using the Kaplan-Meier method. Progression-free is defined as the time from the date of first dose of study treatment to disease progression (e.g. T2 or more advanced disease) or death as a first event.
Event-Free Survival: 30% of all 133 patients treated with Vicinium are estimated to remain event-free at 12 months, using the Kaplan-Meier method. Event-free survival is defined as the time from the date of first dose of study treatment to disease recurrence, progression, or death as a first event.
Overall Survival: 91% of all 133 patients treated with Vicinium have an overall survival of >2.5 years, using the Kaplan-Meier method. Overall survival is defined as the time from the date of first dose of study treatment to death from any cause.
Vicinium Continues to be Well-tolerated by Patients Treated in the Phase 3 VISTA Trial: As of the March 1, 2019 data cut off, in patients across all cohorts (n=133), 78% 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 by the clinical investigators to be manageable and reversible, and only five patients (4%) discontinued treatment due to an adverse event. Serious adverse events, regardless of treatment attribution, were reported in 14% of patients. There were four treatment-related SAEs reported in three patients including acute kidney injury (Grade 3), pyrexia (Grade 2), cholestatic hepatitis (Grade 4) and renal failure (Grade 5). There were no age-related increases in adverse events observed in the Phase 3 VISTA trial.
Positive Data and Safety Monitoring Board (DSMB) Review of Phase 3 VISTA Trial Data: In March, the independent DSMB completed its ninth planned safety review for the Phase 3 VISTA trial and recommended the trial continue without modification.
Completion of Full-Scale GMP Manufacturing Run at FUJIFILMProvides Encouraging Preliminary Results, Supporting Analytical Comparability Plan to be Reviewed with the FDA: In October 2018, Sesen Bio entered into an agreement for the manufacturing process and technology transfer of Vicinium production with FUJIFILM Diosynth Biotechnologies U.S.A., Inc. (FUJIFILM). In April 2019, the first full, commercial-scale GMP run was completed at FUJIFILM. Preliminary indicators of success, including the bacterial growth and purification profiles, support FUJIFILM’S ability to produce the bulk drug substance form of Vicinium for commercial purposes if Sesen Bio receives regulatory approval to market Vicinium. Full quality release testing is underway, and results are expected to be completed in May 2019.
Updated Phase 3 VISTA Trial Data Along with Closely Matched Phase 2 Clinical Trial Data to Serve as Basis for Upcoming FDA Meetings:
Type C CMC Meeting Scheduled for May 20, 2019. In conjunction with the technology transfer of Vicinium production with FUJIFILM, the Company will seek alignment with the FDA on an analytical comparability plan that can be used to assess comparability between the supply used in clinical trials and the potential commercial supply produced by FUJIFILM.
Pre-BLA Meeting Scheduled for June 6, 2019: In concurrence with the FDA’s recommendation that the Company schedule a meeting in mid-2019, Sesen Bio has confirmed a meeting date with the FDA in June to discuss its intended registration strategy for Vicinium for the treatment of high-risk NMIBC.
First Quarter 2019 Financial Results

Cash Position: Cash and cash equivalents were $42.4 million as of March 31, 2019, compared to $50.4 million as of December 31, 2018, and $19.7 million as of March 31, 2018, the comparable period one year ago.
Revenue: No revenue was recorded for the three months ended March 31, 2019, nor for the same period in 2018.
R&D Expenses: Research and development (R&D) expenses for the first quarter of 2019 were $4.7 million compared to $3.3 million in R&D expenses for the same period in 2018. The increase was primarily due to $1.7 million in costs related to the ongoing manufacturing process and technology transfer with FUJIFILM, and increased internal and external staffing costs, partially offset by reduced expenses related to the Phase 3 VISTA trial.
G&A Expenses: General and administrative expenses for the first quarter of 2019 were $3.1 million compared to $2.0 million for the same period in 2018. The increase was primarily due to higher legal costs, an increase in professional fees and market research costs.
Net Loss: Net loss was $6.5 million, or $0.08 per share, for the first quarter of 2019, compared to $4.0 million, or $0.11 per share, for the first quarter of 2018.
Financial Guidance: Based on its current operating plans, Sesen Bio believes it will have capital sufficient to fund its current operating plan into 2020.
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 7176228. 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 as a monotherapy in patients with high-risk, bacillus Calmette-Guérin, or BCG, unresponsive non-muscle invasive bladder cancer (NMIBC). The primary endpoints of the trial are the complete response rate and the duration of response 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. 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-risk 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. 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-risk NMIBC in patients who have previously received a minimum of two courses of bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. 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.