AVEO Oncology Announces Submission of New Drug Application to U.S. FDA for Tivozanib in Patients with Relapsed or Refractory Renal Cell Carcinoma

On March 31, 2020 AVEO Oncology (NASDAQ: AVEO) reported that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for tivozanib, the Company’s vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI), as a treatment for relapsed or refractory renal cell carcinoma (RCC) (Press release, AVEO, MAR 31, 2020, View Source [SID1234556051]).

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"NDA submission is a distinguishing milestone for any development stage biotechnology company, and our tivozanib NDA is an important step in our goal of providing an effective and more tolerable therapeutic option to patients with relapsed or refractory RCC," said Michael Bailey, president and chief executive officer. "The TIVO-3 study provides valuable insight into the potential sequencing of therapy following earlier TKI and immunotherapy treatment, an area of significant need for kidney cancer patients whose disease has relapsed or become refractory to multiple lines of therapy. All of us at AVEO offer our continued gratitude to the patients, caregivers, and investigators who participated in our clinical trials. We look forward to working closely with the FDA during their review process and remain hopeful that the study’s overall survival (OS) hazard ratio (HR) will continue to favor tivozanib at the time of the final readout, expected by June 2020."

The NDA submission is based on the pivotal active comparator-controlled Phase 3 study, TIVO-3, comparing tivozanib to sorafenib in 3rd and 4th line RCC patients. The application is supported by three additional trials, including an active comparator-controlled supportive Phase 3 study, TIVO-1, comparing tivozanib to sorafenib, and two Phase 2 studies, Study 902, the open-label, crossover clinical study of tivozanib for patients who progressed on sorafenib in TIVO-1, as well as placebo-controlled Study 201 in first line RCC patients.

A final OS analysis of the TIVO-3 study will be conducted in the second quarter based on a May 1, 2020 data cutoff date. AVEO expects to report results from the final OS analysis by June 2020. The FDA and the Company agreed that if, during the review, the final analysis yields an OS HR above 1.00, the Company will withdraw its NDA.

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) discovered by Kyowa Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union, the United Kingdom, Norway, New Zealand and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib is being studied in the TIVO-3 trial, which is supporting a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for relapsed/refractory RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC4. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers.

Takeda Receives Positive CHMP Opinion for ADCETRIS® (brentuximab vedotin) in Combination with CHP (Cyclophosphamide, Doxorubicin and Prednisone) for Treatment of Adult Patients with Previously Untreated Systemic Anaplastic Large Cell Lymphoma

On March 31, 2020 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) granted a positive opinion for the extension of the marketing authorization of ADCETRIS (brentuximab vedotin) and recommended its approval in combination with CHP (cyclophosphamide, doxorubicin, prednisone) as a treatment for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL) (Press release, Takeda, MAR 31, 2020, View Source [SID1234556050]). The positive CHMP opinion is based on the results of the Phase 3 ECHELON-2 study evaluating ADCETRIS in combination with CHP to a standard care, CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone), in previously untreated patients with CD30+ peripheral T-cell lymphoma (PTCL), including the subtype sALCL. ADCETRIS is an antibody-drug conjugate (ADC) directed at CD30, which is expressed on the surface of several types of PTCL, including sALCL.

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"There have been no significant treatment advancements for PTCL over the last few decades. Historically there have been a lack of randomized clinical studies in this setting, making it a challenge to establish an optimal therapy for these patients," said Eva Domingo-Domenech, MD, Institut Català d’Oncologia – Hospitalet, Hospital Duran i Reynals. "Outcomes with currently available therapies are often poor, and there is an urgent need for new treatment options. If approved for adult patients with previously untreated sALCL, ADCETRIS may offer an important option for European patients."

"The positive CHMP opinion marks an important step forward for the European sALCL community," said Christopher Arendt, Head, Oncology Therapeutic Area Unit, Takeda. "ECHELON-2 showed that ADCETRIS plus CHP demonstrated a significant improvement in progression-free survival and overall survival while maintaining a safety profile comparable to the standard of care of CHOP. We look forward to the European Commission’s review of this positive opinion and hope to bring ADCETRIS to eligible patients in need."

The opinion for ADCETRIS will now be reviewed by the European Commission (EC) for Commission Decision. ADCETRIS is currently not approved as a therapy for frontline sALCL in Europe.

The ECHELON-2 study met its primary endpoint with ADCETRIS plus CHP demonstrating a statistically significant improvement in progression-free survival (PFS) as assessed by an Independent Review Committee (hazard ratio [HR]=0.71; p-value=0.0110), 29 percent improvement in PFS. The safety profile of ADCETRIS plus CHP in the ECHELON-2 trial was comparable to CHOP and consistent with the established safety profile of ADCETRIS in combination with chemotherapy.

About the ECHELON-2 Trial
ECHELON-2 is a randomized, double-blind, placebo-controlled Phase 3 trial and is investigating ADCETRIS plus CHP (cyclophosphamide, doxorubicin, prednisone) versus CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone) as frontline therapy in patients with CD30-expressing peripheral T-cell lymphoma, also known as mature T-cell lymphoma. The primary endpoint is progression-free survival (PFS) per Independent Review Facility assessment, with events defined as progression, death, or receipt of chemotherapy for residual or progressive disease. Secondary endpoints include PFS in patients with systemic anaplastic large cell lymphoma (sALCL), complete remission rate, overall survival and objective response rate, in addition to safety. The multi-center trial was conducted at sites across North America, Europe and Asia and enrolled 452 patients. The majority of patient enrolled had sALCL.

About T-Cell Lymphomas
There are more than 100 subtypes of non-Hodgkin lymphomas which are broadly divided into two major groups: B-cell lymphomas, which develop from abnormal B-lymphocytes, and T-cell lymphomas, which develop from abnormal T-lymphocytes. There are many different forms of T-cell lymphomas, some of which are extremely rare. T-cell lymphomas can be aggressive (fast-growing) or indolent (slow-growing). PTCL accounts for approximately 10 percent of non-Hodgkin lymphoma cases in the U.S. and Europe and may be as high as 24 percent in parts of Asia.

About ADCETRIS
ADCETRIS is an antibody-drug conjugate (ADC) comprising an anti-CD30 monoclonal antibody attached by a protease-cleavable linker to a microtubule disrupting agent, monomethyl auristatin E (MMAE), utilizing Seattle Genetics’ proprietary technology. The ADC employs a linker system that is designed to be stable in the bloodstream but to release MMAE upon internalization into CD30-positive tumor cells.

ADCETRIS injection for intravenous infusion has received FDA approval for six indications in adult patients with: (1) previously untreated systemic anaplastic large cell lymphoma (sALCL) or other CD30-expressing peripheral T-cell lymphomas (PTCL), including angioimmunoblastic T-cell lymphoma and PTCL not otherwise specified, in combination with cyclophosphamide, doxorubicin, and prednisone, (2) previously untreated Stage III or IV classical Hodgkin lymphoma (cHL), in combination with doxorubicin, vinblastine, and dacarbazine, (3) cHL at high risk of relapse or progression as post-autologous hematopoietic stem cell transplantation (auto-HSCT) consolidation, (4) cHL after failure of auto-HSCT or failure of at least two prior multi-agent chemotherapy regimens in patients who are not auto-HSCT candidates, (5) sALCL after failure of at least one prior multi-agent chemotherapy regimen, and (6) primary cutaneous anaplastic large cell lymphoma (pcALCL) or CD30-expressing mycosis fungoides (MF) who have received prior systemic therapy.

Health Canada granted ADCETRIS approval with conditions for relapsed or refractory Hodgkin lymphoma and sALCL in 2013, and non-conditional approval for post-autologous stem cell transplantation (ASCT) consolidation treatment of Hodgkin lymphoma patients at increased risk of relapse or progression in 2017, adults with pcALCL or CD30-expressing MF who have had prior systemic therapy in 2018, for previously untreated Stage IV Hodgkin lymphoma in combination with doxorubicin, vinblastine, and dacarbazine in 2019 and for previously untreated adult patients with sALCL, peripheral T-cell lymphoma-not otherwise specified (PTCL-NOS) or angioimmunoblastic T-cell lymphoma (AITL), whose tumors express CD30, in combination with cyclophosphamide, doxorubicin, prednisone in 2019.

ADCETRIS received conditional marketing authorization from the European Commission in October 2012. The approved indications in Europe are: (1) for the treatment of adult patients with previously untreated CD30-positive Stage IV Hodgkin lymphoma in combination with doxorubicin, vinblastine and dacarbazine (AVD), (2) for the treatment of adult patients with CD30-positive Hodgkin lymphoma at increased risk of relapse or progression following ASCT, (3) for the treatment of adult patients with relapsed or refractory CD30-positive Hodgkin lymphoma following ASCT, or following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option, (4) for the treatment of adult patients with relapsed or refractory sALCL and (5) for the treatment of adult patients with CD30-positive cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

In Japan, ADCETRIS received its first approval in January 2014 for relapsed or refractory Hodgkin lymphoma and ALCL, and untreated Hodgkin lymphoma in combination with doxorubicin, vinblastine, and dacarbazine in September 2018, and Peripheral T-cell lymphomas in December 2019. In December 2019, ADCETRIS obtained additional dosage & administration for the treatment of relapsed or refractory Hodgkin lymphoma and Peripheral T-cell lymphomas in pediatric. The current wording of approved indication in Japan package insert is for the treatment of patients with CD30 positive: Hodgkin lymphoma and Peripheral T-cell lymphomas.

ADCETRIS has received marketing authorization by regulatory authorities in more than 70 countries/ regions for relapsed or refractory Hodgkin lymphoma and sALCL. See important safety information below.

ADCETRIS is being evaluated broadly in more than 70 clinical trials, including a Phase 3 study in first-line Hodgkin lymphoma (ECHELON-1) and another Phase 3 study in first-line CD30-positive peripheral T-cell lymphomas (ECHELON-2), as well as trials in many additional types of CD30-positive malignancies.

Seattle Genetics and Takeda are jointly developing ADCETRIS. Under the terms of the collaboration agreement, Seattle Genetics has U.S. and Canadian commercialization rights and Takeda has rights to commercialize ADCETRIS in the rest of the world. Seattle Genetics and Takeda are funding joint development costs for ADCETRIS on a 50:50 basis, except in Japan where Takeda is solely responsible for development costs.

ADCETRIS (brentuximab vedotin) Important Safety Information (European Union)
Please refer to Summary of Product Characteristics (SmPC) before prescribing.

CONTRAINDICATIONS

ADCETRIS is contraindicated for patients with hypersensitivity to brentuximab vedotin and its excipients. In addition, combined use of ADCETRIS with bleomycin causes pulmonary toxicity.

SPECIAL WARNINGS & PRECAUTIONS

Progressive multifocal leukoencephalopathy (PML): John Cunningham virus (JCV) reactivation resulting in progressive multifocal leukoencephalopathy (PML) and death can occur in patients treated with ADCETRIS. PML has been reported in patients who received ADCETRIS after receiving multiple prior chemotherapy regimens. PML is a rare demyelinating disease of the central nervous system that results from reactivation of latent JCV and is often fatal.

Closely monitor patients for new or worsening neurological, cognitive, or behavioral signs or symptoms, which may be suggestive of PML. Suggested evaluation of PML includes neurology consultation, gadolinium-enhanced magnetic resonance imaging of the brain, and cerebrospinal fluid analysis for JCV DNA by polymerase chain reaction or a brain biopsy with evidence of JCV. A negative JCV PCR does not exclude PML. Additional follow up and evaluation may be warranted if no alternative diagnosis can be established. Hold dosing for any suspected case of PML and permanently discontinue ADCETRIS if a diagnosis of PML is confirmed.

Be alert to PML symptoms that the patient may not notice (e.g., cognitive, neurological, or psychiatric symptoms).

Pancreatitis: Acute pancreatitis has been observed in patients treated with ADCETRIS. Fatal outcomes have been reported. Closely monitor patients for new or worsening abdominal pain, which may be suggestive of acute pancreatitis. Patient evaluation may include physical examination, laboratory evaluation for serum amylase and serum lipase, and abdominal imaging, such as ultrasound and other appropriate diagnostic measures. Hold ADCETRIS for any suspected case of acute pancreatitis. ADCETRIS should be discontinued if a diagnosis of acute pancreatitis is confirmed.

Pulmonary Toxicity: Cases of pulmonary toxicity, some with fatal outcomes, including pneumonitis, interstitial lung disease, and acute respiratory distress syndrome (ARDS), have been reported in patients receiving ADCETRIS. Although a causal association with ADCETRIS has not been established, the risk of pulmonary toxicity cannot be ruled out. Promptly evaluate and treat new or worsening pulmonary symptoms (e.g., cough, dyspnoea) appropriately. Consider holding dosing during evaluation and until symptomatic improvement.

Serious infections and opportunistic infections: Serious infections such as pneumonia, staphylococcal bacteremia, sepsis/septic shock (including fatal outcomes), and herpes zoster, cytomegalovirus (CMV) (reactivation) and opportunistic infections such as Pneumocystis jiroveci pneumonia and oral candidiasis have been reported in patients treated with ADCETRIS. Patients should be carefully monitored patients during treatment for the emergence of possible serious and opportunistic infections.

Infusion-related reactions (IRR): Immediate and delayed IRR, as well as anaphylaxis, have been reported with ADCETRIS. Carefully monitor patients during and after an infusion. If anaphylaxis occurs, immediately and permanently discontinue administration of ADCETRIS and administer appropriate medical therapy. If an IRR occurs, interrupt the infusion and institute appropriate medical management. The infusion may be restarted at a slower rate after symptom resolution. Patients who have experienced a prior IRR should be premedicated for subsequent infusions. IRRs are more frequent and more severe in patients with antibodies to ADCETRIS.

Tumor lysis syndrome (TLS): TLS has been reported with ADCETRIS. Patients with rapidly proliferating tumor and high tumor burden are at risk of TLS. Monitor these patients closely and manage according to best medical practice.

Peripheral neuropathy (PN): ADCETRIS treatment may cause PN, both sensory and motor. ADCETRIS-induced PN is typically an effect of cumulative exposure to ADCETRIS and is reversible in most cases. Monitor patients for symptoms of neuropathy, such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain, or weakness. Patients experiencing new or worsening PN may require a delay and a dose reduction or discontinuation of ADCETRIS.

Hematological toxicities: Grade 3 or Grade 4 anemia, thrombocytopenia, and prolonged (equal to or greater than one week) Grade 3 or Grade 4 neutropenia can occur with ADCETRIS. Monitor complete blood counts prior to administration of each dose.

Febrile neutropenia: Febrile neutropenia has been reported with ADCETRIS. Complete blood counts should be monitored prior to administration of each dose of treatment. Closely monitor patients for fever and manage according to best medical practice if febrile neutropenia develops.

When ADCETRIS is administered in combination with AVD, primary prophylaxis with G-CSF is recommended for all patients beginning with the first dose.

Stevens-Johnson syndrome (SJS): SJS and toxic epidermal necrolysis (TEN) have been reported with ADCETRIS. Fatal outcomes have been reported. Discontinue treatment with ADCETRIS if SJS or TEN occurs and administer appropriate medical therapy.

Gastrointestinal (GI) Complications: GI complications, some with fatal outcomes, including intestinal obstruction, ileus, enterocolitis, neutropenic colitis, erosion, ulcer, perforation and haemorrhage, have been reported with ADCETRIS. Promptly evaluate and treat patients if new or worsening GI symptoms occur.

Hepatotoxicity: Elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) have been reported with ADCETRIS. Serious cases of hepatotoxicity, including fatal outcomes, have also occurred. Pre-existing liver disease, comorbidities, and concomitant medications may also increase the risk. Test liver function prior to treatment initiation and routinely monitor during treatment. Patients experiencing hepatotoxicity may require a delay, dose modification, or discontinuation of ADCETRIS.

Hyperglycemia: Hyperglycemia has been reported during trials in patients with an elevated body mass index (BMI) with or without a history of diabetes mellitus. Closely monitor serum glucose for patients who experiences an event of hyperglycemia. Administer anti-diabetic treatment as appropriate.

Renal and Hepatic Impairment: There is limited experience in patients with renal and hepatic impairment. Available data indicate that MMAE clearance might be affected by severe renal impairment, hepatic impairment, and by low serum albumin concentrations.

CD30+ CTCL: The size of the treatment effect in CD30 + CTCL subtypes other than mycosis fungoides (MF) and primary cutaneous anaplastic large cell lymphoma (pcALCL) is not clear due to lack of high level evidence. In two single arm phase II studies of ADCETRIS, disease activity has been shown in the subtypes Sézary syndrome (SS), lymphomatoid papulosis (LyP) and mixed CTCL histology. These data suggest that efficacy and safety can be extrapolated to other CTCL CD30+ subtypes. Carefully consider the benefit-risk per patient and use with caution in other CD30+ CTCL patient types.

Sodium content in excipients: This medicinal product contains 13.2 mg sodium per vial, equivalent to 0.7% of the WHO recommended maximum daily intake of 2 g sodium for an adult.

INTERACTIONS
Patients who are receiving a strong CYP3A4 and P-gp inhibitor, concomitantly with ADCETRIS may have an increased risk of neutropenia. If neutropenia develops, refer to dosing recommendations for neutropenia (see SmPC section 4.2). Co-administration of ADCETRIS with a CYP3A4 inducer did not alter the plasma exposure of ADCETRIS, but it appeared to reduce plasma concentrations of MMAE metabolites that could be assayed. ADCETRIS is not expected to alter the exposure to drugs that are metabolized by CYP3A4 enzymes.

PREGNANCY: Advise women of childbearing potential to use two methods of effective contraception during treatment with ADCETRIS and until 6 months after treatment. There are no data from the use of ADCETRIS in pregnant women, although studies in animals have shown reproductive toxicity. Do not use ADCETRIS during pregnancy unless the benefit to the mother outweighs the potential risks to the fetus.

LACTATION (breast-feeding): There are no data as to whether ADCETRIS or its metabolites are excreted in human milk, therefore a risk to the newborn/infant cannot be excluded. With the potential risk, a decision should be made whether to discontinue breast-feeding or discontinue/abstain from therapy with ADCETRIS.

FERTILITY: In nonclinical studies, ADCETRIS treatment has resulted in testicular toxicity, and may alter male fertility. Advise men being treated with ADCETRIS not to father a child during treatment and for up to 6 months following the last dose.

Effects on ability to drive and use machines: ADCETRIS may have a moderate influence on the ability to drive and use machines.

UNDESIRABLE EFFECTS

Monotherapy: The most frequent adverse reactions (≥10%) were infections, peripheral sensory neuropathy, nausea, fatigue, diarrhoea, pyrexia, upper respiratory tract infection, neutropenia, rash, cough, vomiting, arthralgia, peripheral motor neuropathy, infusion-related reactions, pruritus, constipation, dyspnoea, weight decreased, myalgia and abdominal pain. Serious adverse drug reactions occurred in 12% of patients. The frequency of unique serious adverse drug reactions was ≤1%. Adverse events led to treatment discontinuation in 24% of patients.

Combination Therapy: In the study of ADCETRIS as combination therapy with AVD in 662 patients with previously untreated advanced HL, the most common adverse reactions (≥ 10%) were: neutropenia, nausea, constipation, vomiting, fatigue, peripheral sensory neuropathy, diarrhoea, pyrexia, alopecia, peripheral motor neuropathy, decreased weight, abdominal pain, anaemia, stomatitis, febrile neutropenia, bone pain, insomnia, decreased appetite, cough, headache, arthralgia, back pain, dyspnoea, myalgia, upper respiratory tract infection, alanine aminotransferase increased. Serious adverse reactions occurred in 36% of patients. Serious adverse reactions occurring in ≥ 3% of patients included febrile neutropenia (17%), pyrexia (6%), and neutropenia (3%). Adverse events led to treatment discontinuation in 13% of patients.

ADCETRIS (brentuximab vedotin) U.S. Important Safety Information

BOXED WARNING
PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY (PML): JC virus infection resulting in PML and death can occur in ADCETRIS-treated patients.

Contraindication
ADCETRIS concomitant with bleomycin due to pulmonary toxicity (e.g., interstitial infiltration and/or inflammation).

Warnings and Precautions

Peripheral neuropathy (PN): ADCETRIS causes PN that is predominantly sensory. Cases of motor PN have also been reported. ADCETRIS-induced PN is cumulative. Monitor for symptoms such as hypoesthesia, hyperesthesia, paresthesia, discomfort, a burning sensation, neuropathic pain, or weakness. Institute dose modifications accordingly.
Anaphylaxis and infusion reactions: Infusion-related reactions (IRR), including anaphylaxis, have occurred with ADCETRIS. Monitor patients during infusion. If an IRR occurs, interrupt the infusion and institute appropriate medical management. If anaphylaxis occurs, immediately and permanently discontinue the infusion and administer appropriate medical therapy. Premedicate patients with a prior IRR before subsequent infusions. Premedication may include acetaminophen, an antihistamine, and a corticosteroid.
Hematologic toxicities: Fatal and serious cases of febrile neutropenia have been reported with ADCETRIS. Prolonged (≥1 week) severe neutropenia and Grade 3 or 4 thrombocytopenia or anemia can occur with ADCETRIS. Administer G-CSF primary prophylaxis beginning with Cycle 1 for patients who receive ADCETRIS in combination with chemotherapy for previously untreated Stage III/IV cHL or previously untreated PTCL. Monitor complete blood counts prior to each ADCETRIS dose. Monitor more frequently for patients with Grade 3 or 4 neutropenia. Monitor patients for fever. If Grade 3 or 4 neutropenia develops, consider dose delays, reductions, discontinuation, or G-CSF prophylaxis with subsequent doses.
Serious infections and opportunistic infections: Infections such as pneumonia, bacteremia, and sepsis or septic shock (including fatal outcomes) have been reported in ADCETRIS-treated patients. Closely monitor patients during treatment for bacterial, fungal, or viral infections.
Tumor lysis syndrome: Closely monitor patients with rapidly proliferating tumor and high tumor burden.
Increased toxicity in the presence of severe renal impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with severe renal impairment compared to patients with normal renal function. Avoid use in patients with severe renal impairment.
Increased toxicity in the presence of moderate or severe hepatic impairment: The frequency of ≥Grade 3 adverse reactions and deaths was greater in patients with moderate or severe hepatic impairment compared to patients with normal hepatic function. Avoid use in patients with moderate or severe hepatic impairment.
Hepatotoxicity: Fatal and serious cases have occurred in ADCETRIS-treated patients. Cases were consistent with hepatocellular injury, including elevations of transaminases and/or bilirubin, and occurred after the first ADCETRIS dose or rechallenge. Preexisting liver disease, elevated baseline liver enzymes, and concomitant medications may increase the risk. Monitor liver enzymes and bilirubin. Patients with new, worsening, or recurrent hepatotoxicity may require a delay, change in dose, or discontinuation of ADCETRIS.
PML: Fatal cases of JC virus infection resulting in PML and death have been reported in ADCETRIS-treated patients. First onset of symptoms occurred at various times from initiation of ADCETRIS therapy, with some cases occurring within 3 months of initial exposure. In addition to ADCETRIS therapy, other possible contributory factors include prior therapies and underlying disease that may cause immunosuppression. Consider PML diagnosis in patients with new-onset signs and symptoms of central nervous system abnormalities. Hold ADCETRIS if PML is suspected and discontinue ADCETRIS if PML is confirmed.
Pulmonary toxicity: Fatal and serious events of noninfectious pulmonary toxicity including pneumonitis, interstitial lung disease, and acute respiratory distress syndrome have been reported. Monitor patients for signs and symptoms, including cough and dyspnea. In the event of new or worsening pulmonary symptoms, hold ADCETRIS dosing during evaluation and until symptomatic improvement.
Serious dermatologic reactions: Fatal and serious cases of Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) have been reported with ADCETRIS. If SJS or TEN occurs, discontinue ADCETRIS and administer appropriate medical therapy.
Gastrointestinal (GI) complications: Fatal and serious cases of acute pancreatitis have been reported. Other fatal and serious GI complications include perforation, hemorrhage, erosion, ulcer, intestinal obstruction, enterocolitis, neutropenic colitis, and ileus. Lymphoma with preexisting GI involvement may increase the risk of perforation. In the event of new or worsening GI symptoms, including severe abdominal pain, perform a prompt diagnostic evaluation and treat appropriately.
Hyperglycemia: Serious cases, such as new-onset hyperglycemia, exacerbation of preexisting diabetes mellitus, and ketoacidosis (including fatal outcomes) have been reported with ADCETRIS. Hyperglycemia occurred more frequently in patients with high body mass index or diabetes. Monitor serum glucose and if hyperglycemia develops, administer antihyperglycemic medications as clinically indicated.
Embryo-fetal toxicity: Based on the mechanism of action and animal studies, ADCETRIS can cause fetal harm. Advise females of reproductive potential of the potential risk to the fetus, and to avoid pregnancy during ADCETRIS treatment and for at least 6 months after the final dose of ADCETRIS.
Most Common (≥20% in any study) Adverse Reactions: Peripheral neuropathy, fatigue, nausea, diarrhea, neutropenia, upper respiratory tract infection, pyrexia, constipation, vomiting, alopecia, decreased weight, abdominal pain, anemia, stomatitis, lymphopenia and mucositis.

Drug Interactions
Concomitant use of strong CYP3A4 inhibitors or inducers has the potential to affect the exposure to monomethyl auristatin E (MMAE).

Use in Specific Populations
Moderate or severe hepatic impairment or severe renal impairment: MMAE exposure and adverse reactions are increased. Avoid use.

Advise males with female sexual partners of reproductive potential to use effective contraception during ADCETRIS treatment and for at least 6 months after the final dose of ADCETRIS.

Advise patients to report pregnancy immediately and avoid breastfeeding while receiving ADCETRIS.

Oncology Venture establishes a convertible note program of 100 million SEK

On March 31, 2020 Oncology Venture A/S ("OV" or the "Company") reported that is has established a convertible note program of 100 Million SEK (Press release, Oncology Venture, MAR 31, 2020, View Source [SID1234556049]). The issuance of the convertible bonds is in the full control of Oncology Venture.

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The convertible note agreement is with Negma Group LTD and Park Partners GP (the "Investor") in order to support Oncology Venture’s development and commercialization of its prioritized pipeline of cancer drugs. If Oncology Venture fully utilizes the convertible note program, the company will have sufficient financing to fund its planned activities for 2020.

"I am really happy that we have been able to establish such a flexible and relatively simple financing, where Oncology Venture is in full control of the facility and can solely decide when to exercise. The convertible note program also gives the company the necessary liquidity, so we can focus on bringing our 3 prioritized development programs forward in 2020 to the planned value inflection points," said Steve Carchedi, CEO.

"We are excited to enter into this agreement with Oncology Venture, as we have carefully analysed the investment case and have found both the current valuation and timing to constitute a great opportunity. We are convinced that Oncology Venture will maintain its current fast-phased momentum on its path towards commercialization, and we are proud to fuel the company’s important mission to develop precision treatments for cancer patients," say in a joint statement Elaf Gassam, Chairman of Negma Group and Aurora Lidman, Executive Sales Scandinavia of Park Partners GP.

The main conditions and structure of the program are:

The convertible note program runs for 24 months, during which time Oncology Venture can solely decide to call in 10 tranches of 10 million SEK against issuing convertible notes to the Investor.
The Investors will have the right to convert their convertible notes within a 12-month period following the registration of the notes with the Danish Business Authority. In case of an event of default, the Investor will have the right to request the reimbursement of the convertible notes in cash and/or or refuse to subscribe for additional tranches.
The convertible notes are a zero coupon note and will be issued at a subscription price corresponding to their par value (i.e. SEK 100,000).
The conversion price will be determined as 95% of the lowest closing volume weighted average (VWAP) share price of the 7 consecutive trading days prior the receipt of a conversion request.
No collateral is attached to the convertible notes.
The costs for Oncology Venture are 10 % of the total commitment of SEK 100 million, excluding legal and administrative costs.
The issuance of shares in connection with the convertible notes will require an authorization from the shareholders of Oncology Venture planned to be resolved at the Annual general meeting on April 22, 2020.

AIM ImmunoTech Reports 2019 Year-end Financial Results

On March 31, 2020 AIM ImmunoTech (NYSE American:AIM) reported financial results for the fiscal year ended December 31, 2019, and provides a business update (Press release, AIM ImmunoTech, MAR 31, 2020, View Source;id=188690&p=2174742&I=1206939-c7Z3G6f3m8 [SID1234556048]).

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2019 Financial Highlights

As of December 31, 2019, AIM had cash, cash equivalents and marketable securities of $8.778 million, compared with $1.825 million as of December 31, 2018.

Research and development expenses for 2019 were $4.651 million, compared with $4.778 million for 2018. General and administrative expenses for 2019 were $7.039 million, compared with $6.201 million for 2018.

The net loss from continuing operations for 2019 was $9.533 million, or $2.62 per share, compared with $9.827 million, or $9.77 per share, for 2018.

Please refer to the full 10-K for complete details.

Update on COVID-19 Pandemic Response

Since the outbreak of SARS-CoV-2 – which causes the disease COVID-19 – AIM has been actively engaged in determining whether its drug Ampligen could be an effective treatment for this virus. The company believe that prior studies of Ampligen in SARS-CoV-1 animal experimentation may predict similar protective effects against the new virus as both a protective prophylaxis and an early-onset therapy.

AIM has reached several significant COVID-19 milestones over the past two months:

AIM filed provisional patent applications to secure the company’s intellectual property before seeking domestic and international research partners.
Japan’s National Institute of Infectious Diseases (NIID) agreed to experiment with Ampligen as a potential treatment for COVID-19. The testing and research will be conducted by laboratories at the NIID and the University of Tokyo. AIM will report immediately upon receipt of results.
AIM engaged ChinaGoAbroad (CGA) to facilitate pre-clinical and clinical trials of Ampligen in the People’s Republic of China (PRC).
AIM is in discussions with GP-Pharm in Argentina to advance Ampligen for potential use against COVID-19. Ampligen is an approved drug in Argentina for use against myalgic encephalomyelitis/chronic fatigue syndrome.
AIM is in discussions in The Netherlands – where Ampligen was recently used to treat pancreatic cancer patients – to explore expedited pre-clinical and clinical trials of Ampligen.
AIM is currently targeting the United States, Europe, Asia and Argentina for new clinical trials and, as necessary steps before clinical trials could begin, the company is in the process of soliciting one or more sites and qualified principal investigators in one or more countries. Protocols for potential clinical trial are in the final stages of development. AIM intends, as a next step, to as rapidly as possible seek Institutional Review Board approvals and governmental authorizations to commence the clinical trials of Ampligen as both a protective prophylaxis and an early-onset therapy.

Update on Cancer Clinical Trials/Programs

Six Ampligen clinical trials are currently underway at university cancer centers testing whether tumor microenvironments can be reprogrammed to increase the effectiveness of cancer immunotherapy, including checkpoint inhibitors. The company will promptly update stockholders and the market as more information on these studies becomes available. Cancer patients are especially at risk with COVID-19. AIM recognizes that all cancer centers, like all medical facilities, must make the pandemic their priority. Therefore, there is the potential for delays in clinical trial enrollment and reporting in ongoing studies in cancer patients as a consequence of the COVID-19 medical emergency

Phio Announces Positive New Data Validating the INTASYL™ Immunotherapy Platform’s Ability to Develop Novel Compounds Capable of Inhibiting Cancer Tumor Growth

On March 31, 2020 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported data from various animal studies validating the potential of Phio’s INTASYL technology as a cancer immunotherapy platform for developing novel compounds (Press release, Phio Pharmaceuticals, MAR 31, 2020, View Source [SID1234556045]). The preclinical findings, which will be presented in further detail at upcoming scientific conferences, demonstrate that the INTASYL-enabled compounds can effectively be used to reduce immunosuppression in the tumor microenvironment (TME), which is one of the key challenges for many other immunotherapy platforms to achieve an adequate therapeutic effect in solid tumors.

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Phio scientists conducted several animal studies with a mouse version of PH-762 (mPH-762) and with PH-894 in a validated mouse model of cancer (murine hepatocellular carcinoma model with Hepa1-6 cells). These studies show that a local administration of mPH-762 or PH-894 through intra-tumoral injection resulted in potent anti-tumoral effects. The treated animals showed a complete and statistically significant (p <0.0001) inhibition of tumor growth, whereas placebo treated animals displayed exponential tumor growth.

"These results demonstrate that local administration of INTASYL-enabled compounds successfully infiltrate solid tumors, and significantly impact the tumor microenvironment, resulting in reduced tumor growth by activating the immune response in animal models of solid tumors," said Dr. Simon Fricker, Vice President of Research of Phio Pharmaceuticals. "Our findings show that with INTASYL we can efficiently and selectively target intracellular proteins such as BRD4, which is a well described therapeutic target that is challenging to attack with other available therapeutic platforms."

PH-762 is designed to elicit checkpoint blockade by inhibiting PD-1 receptor expression in T cells and has shown to silence the expression of checkpoint molecule PD-1 in target human T cells in a potent and durable manner. This is compared to the currently available treatment options for PD-1 inhibition, which include systemic administration of monoclonal antibody therapy.

"Only a minority of patients achieve a durable objective response with these monoclonal antibody therapies and there is a high risk of systemic immune-related toxicities, which is a major limiting factor to their clinical use. Intra-tumoral therapeutics, such as PH-762, could result in an optimized benefit/risk ratio, with good antitumor response as well as reduced systemic immune-related toxicities, that would be highly interesting, especially in early stages of the diseases," said Professor Caroline Robert, MD, PhD, head of the Dermatology Unit at the Gustave Roussy Institute which focuses on skin cancer research.

PH-894 is an INTASYL-enabled compound silencing the expression of BRD4, a regulator of gene expression impacting cell differentiation. PH-894 has shown in previous studies to improve T cell function and persistence by differentiating T cells into an effector memory phenotype.

Professor Rolf Kiessling, MD, PhD, Professor of Experimental Oncology at the Karolinska Institutet and member of Phio’s Scientific Advisory Board, stated: "BRD4 is increasingly recognized as an important target for cancer therapy, but selective inhibition of this protein thus far has been challenging. In contrast to small molecule drugs, PH-894 can selectively inhibit BRD4 without impacting closely related proteins, due to the precise and selective silencing of protein expression possible with the INTASYL platform."

"These exciting new preclinical results build upon our animal data with PH-804, an INTASYL compound designed to silence the expression of the immune exhaustion target TIGIT in various immune cells resulting in them becoming weaponized, and support the continued development of our pipeline of therapeutic compounds," said Dr. Gerrit Dispersyn, President and CEO of Phio Pharmaceuticals. "These results, along with our development efforts with INTASYL in the field of adoptive cellular therapy, show the promise of our INTASYL platform in various treatment approaches of hard-to-treat cancers, such as solid tumors. We believe our technology has a bright future as a means to improve adoptive cellular therapy, but also as a direct therapeutic platform."