On May 20, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that expanded tumor response data and overall survival data from the Phase 2a portion of the ongoing Phase 1/2a study of VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate in recurrent glioblastoma (GBM) patients, will be presented at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place June 4-8 (Press release, VBI Vaccines, MAY 20, 2021, View Source [SID1234580391]).

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The e-poster presentation will highlight patient-specific tumor response data and improvement in overall survival (OS) compared to historical controls across both study arms:

Study arm 1 : VBI-1901 + granulocyte-macrophage colony-stimulating factor (GM-CSF)
6-month and 12-month OS : 80% (n=8/10) and 60% (n=6/10), respectively
2 partial responses (≥ 50% tumor reduction) and 2 stable disease observations – 40% disease control rate
1 patient still on protocol
Study arm 2 : VBI-1901 + GSK’s AS01B adjuvant system
6-month OS : 89% (n=8/9) – 12-month OS not yet reached
5 stable disease observations – 50% disease control rate
1 patient still on protocol
With few options for recurrent GBM patients, historical control data have demonstrated overall survival to be ~60% at 6-months and ~30% at 12-months after treatment with a monotherapy.1

Presentation details and schedule are as follows:

Abstract #: 2047
Abstract Title: Evaluation of GM-CSF and AS01B adjuvants in a Phase 1/2a trial of a therapeutic CMV vaccine (VBI-1901) against recurrent glioblastoma (GBM)
Presenter: Patrick Y. Wen, M.D., Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute, Professor of Neurology at Harvard Medical School, and investigator of VBI’s Phase 1/2a study of VBI-1901
Session: Poster Session: Central Nervous System Tumors
Poster Access: June 4-8, 2021

The e-poster presentation will include a 5-minute pre-recorded presentation from Dr. Wen, available On Demand to meeting attendees from 9 AM ET on Friday, June 4.

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 38 patients with recurrent GBM:

Phase 1 (Part A)
Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) in recurrent GBM patients with any number of prior recurrences.
This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg.
Enrollment completed in December 2018.
Phase 2a (Part B)
Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase.
This phase is a two-arm study, enrolling 10 first-recurrent GBM patients in each vaccinated group, assessing 10.0 µg of VBI-1901 in combination with either GM-CSF or GSK’s proprietary AS01 adjuvant system as immunomodulatory adjuvants.
Enrollment of the 10 patients in each adjuvant group is complete.
VBI-1901 is administered intradermally when adjuvanted with GM-CSF and intramuscularly when adjuvanted with GSK’s AS01 adjuvant system. Patients in both phases of the study receive the vaccine immunotherapeutic every four weeks until tumor progression.

About VBI-1901 and GBM

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 12,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

On May 20, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that it and its investigator sponsors will present four new abstracts at the virtual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting from June 4 – June 8, 2021 and five abstracts at the virtual 26th Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) (EHA2021) from June 9 –16, 2021 (Press release, Jazz Pharmaceuticals, MAY 20, 2021, View Source [SID1234580407]). Research findings to be presented include new data on Zepzelca (lurbinectedin), Vyxeos/Vyxeos Liposomal (daunorubicin and cytarabine), also known as JZP351 (formerly known as CPX-351), and Defitelio (defibrotide sodium).

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"The unmet needs of patients facing unique challenges and difficult odds drive our research and development objectives, and it’s critical for us to continue enhancing our understanding of our existing therapies so that the greatest number of patients can benefit from our innovative medicines," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development and chief medical officer of Jazz Pharmaceuticals. "We are excited to build upon our growing oncology portfolio and make meaningful strides for more people living with rare or complex hematologic and oncologic diseases."

Highlights from Jazz and its investigational sponsors at the congresses will include:

A poster presentation at ASCO (Free ASCO Whitepaper) featuring results from the Phase 1/2 study evaluating the combination of lurbinectedin plus irinotecan in women with endometrial carcinoma.
A poster presentation featuring preliminary results from V-FAST, a Phase 1b clinical trial evaluating JZP351 in combination with one of three targeted agents (venetoclax, midostaurin or enasidenib) in adults with previously untreated acute myeloid leukemia (AML) who are considered fit for intensive chemotherapy. These data will be presented at both ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper).
All ASCO (Free ASCO Whitepaper) virtual poster presentations and poster discussion presentations will be available on-demand to registered participants for 180 days beginning June 4, 2021. The Jazz-supported and investigational sponsor presentations and publications covering lurbinectedin and JZP351 (formerly known as CPX-351) at the ASCO (Free ASCO Whitepaper) Annual Meeting are:

Lurbinectedin Presentations

Presentation Title

Author

Poster Number / Abstract Link

Phase 1/2 Study of Lurbinectedin in
Combination with Irinotecan in Patients
with Endometrial Carcinoma

Sanchez-
Simon I, et
al.

Poster Number: 5586

Abstract Link

Treatment Patterns of Patients with
Small Cell Lung Cancer in Second and
Third-Line Therapy Settings

Prince P, et
al.

Publication only

Overall Survival of Small Cell Lung
Cancer Patients Initiating Anti-Cancer
Treatment

Estrin A, et
al.

Publication only

JZP351 (formerly known as CPX-351) Poster Presentation

Presentation Title

Author

Poster Number / Abstract Link

Preliminary Results of V-FAST, a Phase
1B Master Trial to Investigate CPX-351
Combined with Targeted Agents in Newly
Diagnosed AML

Pullarkat V,
et al.

Poster Number: 7026

Abstract Link

The poster presentations and publications covering JZP351 (formerly known as CPX-351) and defibrotide sodium at EHA (Free EHA Whitepaper)2021 virtual congress are:

JZP351 (formerly known as CPX-351) Presentations

Presentation Title

Author

Abstract Code / Abstract Link

Preliminary Results of V-FAST, a Phase
1B Master Trial to Investigate CPX-351
Combined with Targeted Agents in Newly Diagnosed
AML

Pullarkat V,
et al.

Poster Number: EP442

Abstract Link

Rapid Genomic Profiling of AML Patients
with Myelodysplasia-related Cytogenetic
Aberrations (investigator sponsored trial)

Dolnik A, et
al.

Poster Number: EP402

Abstract Link

Pilot Study of CPX-351 in Adults with
Relapsed/Refractory Acute
Lymphoblastic Leukemia (investigator
sponsored trial)

Shah BD,
et al.

Publication only

Defibrotide Sodium Poster Presentations

Presentation Title

Author

Abstract Code / Abstract Link

Incidence of Veno-Occlusive
Disease/Sinusoidal Obstruction
Syndrome (VOD/SOS) After
Hematopoietic Cell Transplantation (HCT)
in England Based on Healthcare
Resource Group Codes and Hospital
Episode Statistics© Data

Angus J, et
al.

Poster Number: EP1258

Abstract Link

EBMT Pass Registry Outcomes of
Defibrotide-Treated Patients by Timing of
Onset of VOD/SOS After HCT

Mohty M,
et al.

Poster Number: EP1238

Abstract Link

About Vyxeos/Vyxeos Liposomal (daunorubicin and cytarabine), also known as JZP 351
Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor.

In the U.S., Vyxeos is indicated for the treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older.1

More information about Vyxeos in the United States, including Full Prescribing Information, BOXED Warning and Medication Guide, is available here.

In Europe, Vyxeos Liposomal (daunorubicin/cytarabine) is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). Backed by a robust clinical development program including Phase 3 data, Vyxeos is currently approved in more than 30 countries, and Jazz continues to work with regulatory authorities worldwide to bring this innovative therapy to appropriate patients.

The full Summary of Product Characteristics of Vyxeos Liposomal in Europe is available here.

Important Safety Information for VYXEOS/VYXEOS LIPOSOMAL
WARNING: VYXEOS has different dosage recommendations from other medications that contain daunorubicin and/or cytarabine. Do not substitute VYXEOS for other daunorubicin and/or cytarabine-containing products.

VYXEOS should not be given to patients who have a history of serious allergic reaction to daunorubicin, cytarabine, or any of its ingredients.

VYXEOS can cause a severe decrease in blood cells (red and white blood cells and cells that prevent bleeding, called platelets) which can result in serious infection or bleeding and possibly lead to death. Your doctor will monitor your blood counts during treatment with VYXEOS. Patients should tell the doctor about new onset fever or symptoms of infection or if they notice signs of bruising or bleeding.

VYXEOS can cause heart-related side effects. Tell your doctor about any history of heart disease, radiation to the chest, or previous chemotherapy. Inform your doctor if you develop symptoms of heart failure such as:

shortness of breath or trouble breathing
swelling or fluid retention, especially in the feet, ankles, or legs
unusual tiredness
VYXEOS may cause allergic reactions including anaphylaxis. Seek immediate medical attention if you develop signs and symptoms of anaphylaxis such as:

trouble breathing
severe itching
skin rash or hives
swelling of the face, lips, mouth, or tongue
VYXEOS contains copper and may cause copper overload in patients with Wilson’s disease or other copper-processing disorders.

VYXEOS can damage the skin if it leaks out of the vein. Tell your doctor right away if you experience symptoms of burning, stinging, or blisters and skin sores at the injection site.

VYXEOS can harm your unborn baby. Inform your doctor if you are pregnant, planning to become pregnant, or nursing. Do not breastfeed while receiving VYXEOS. Females and males of reproductive potential should use effective contraception during treatment and for 6 months following the last dose of VYXEOS.

The most common side effects were bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

About Zepzelca (lurbinectedin)
Disclaimer: All information about Zepzelca is intended for a U.S.-audience only.
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and eventual cell death.2

Zepzelca for injection 4 mg is a prescription medicine used to treat adults with a kind of lung cancer called small cell lung cancer that has spread to other parts of the body (metastatic) and who have received treatment with chemotherapy that contains platinum, and it did not work or is no longer working. Zepzelca is approved based on response rate and how long the response lasted. Additional studies will further evaluate the benefit of Zepzelca for this use.

Important Safety Information for ZEPZELCA

Before receiving ZEPZELCA, tell your healthcare provider about all of your medical conditions, including if you:

have liver or kidney problems.
are pregnant or plan to become pregnant. ZEPZELCA can harm your unborn baby.
Females who are able to become pregnant:
Your healthcare provider should do a pregnancy test before you start treatment with ZEPZELCA.
You should use effective birth control (contraception) during treatment with and for 6 months after your final dose of ZEPZELCA.
Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with ZEPZELCA.
Males with female partners who are able to become pregnant should use effective birth control during treatment with and for 4 months after your final dose of ZEPZELCA.

are breastfeeding or plan to breastfeed. It is not known if ZEPZELCA passes into your breastmilk. Do not breastfeed during treatment with ZEPZELCA and for 2 weeks after your final dose of ZEPZELCA. Talk to your healthcare provider about the best way to feed your baby during treatment with ZEPZELCA.
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Certain other medicines may affect how ZEPZELCA works.

What should I avoid while using ZEPZELCA?

Avoid eating or drinking grapefruit, or products that contain grapefruit juice during treatment with ZEPZELCA.

ZEPZELCA can cause serious side effects, including:

Low blood cell counts. Low blood counts including low neutrophil counts (neutropenia) and low platelet counts (thrombocytopenia) are common with ZEPZELCA, and can also be severe. Some people with low white blood cell counts may get fever, or an infection throughout the body (sepsis), that can cause death. Your healthcare provider should do blood tests before you receive each treatment with ZEPZELCA to check your blood cell counts.
Tell your healthcare provider right away if you develop:
fever or any other signs of infection
unusual bruising or bleeding
tiredness
pale colored skin
Liver problems. Increased liver function tests are common with ZEPZELCA, and can also be severe. Your healthcare provider should do blood tests to check your liver function before you start and during treatment with ZEPZELCA.
Tell your healthcare provider right away if you develop symptoms of liver problems including:
loss of appetite 
nausea or vomiting 
pain on the right side of your stomach area (abdomen)
Your healthcare provider may temporarily stop treatment, lower your dose, or permanently stop ZEPZELCA if you develop low blood cell counts or liver problems during treatment with ZEPZELCA.

The most common side effects of ZEPZELCA include:

tiredness
low white and red blood cell counts
increased kidney function blood test (creatinine)
increased liver function blood tests
increased blood sugar (glucose)
nausea
decreased appetite
muscle and joint (musculoskeletal) pain
low level of albumin in the blood
constipation
trouble breathing
low levels of sodium and magnesium in the blood
vomiting
cough
diarrhea
These are not all of the possible side effects of ZEPZELCA.

Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Jazz Pharmaceuticals at 1-800-520-5568.

More information about Zepzelca, including Full Prescribing Information and Patient Information, is available here.

ZEPZELCA is a trademark of PharmaMar, S.A. used by Jazz Pharmaceuticals under license.

About Defitelio (defibrotide sodium)
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received U.S. Food and Drug Administration (FDA) marketing approval on March 30, 2016, and it is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication. Defitelio is not approved for the prevention of VOD.

Please see full Prescribing Information for Defitelio in the United States.

In Europe, defibrotide is marketed under the name Defitelio ▼ (defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients after HSCT therapy. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC.
(View Source)

The full Summary of Product Characteristics of Defitelio in Europe is available here.

Important Safety Information for Defitelio
Defitelio should not be given to patients who are:

Currently taking anticoagulants or fibrinolytics
Allergic to Defitelio or any of its ingredients
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.

Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.

The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.

On May 20, 2021 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported that the Japan Patent Office has allowed the patent application titled "Systems and Methods for Producing Synthetic Hypericin" (Press release, Soligenix, MAY 20, 2021, View Source [SID1234580424]). The allowed claims are directed to unique, proprietary methods to produce a novel, highly purified form of synthetic hypericin, and are similar to those previously allowed in the United States (U.S.). Synthetic hypericin is the active pharmaceutical ingredient in HyBryte (SGX301), the Company’s photodynamic therapy, for which positive primary endpoint results in a pivotal Phase 3 study for the treatment of cutaneous T-cell lymphoma (CTCL) were recently announced. This new patent is the first allowed in Japan covering the proprietary methods developed by the Company and further expands the comprehensive HyBryte patent estate, which includes protection on the composition of the purified synthetic hypericin, methods of synthesis and therapeutic methods of use in both CTCL and psoriasis, and is being pursued worldwide.

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HyBryte (synthetic hypericin) is a novel first-in-class photodynamic therapy for first-line treatment of early stage CTCL. In the recently completed pivotal Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial, HyBryte achieved a statistically significant treatment response rate (p=0.04) in the primary endpoint after just 6 weeks (Cycle 1) of therapy when compared to placebo. This positive treatment response continued to significantly improve with extended HyBryte treatment in the open-label treatment cycles after 12 weeks (Cycle 2) and 18 weeks (Cycle 3) total treatment, reinforcing the positive HyBryte primary endpoint treatment response demonstrated in Cycle 1. In addition, HyBryte has demonstrated a statistically significant response in both patch and plaque lesions through 12 weeks of treatment (Cycle 2), highlighting the unique benefit of using visible light with its deeper skin penetration. HyBryte was well tolerated throughout the study and no mutagenic risks have been identified, unlike other second-line or off-label treatments, including other phototherapies, which utilize ultraviolet light. The Company believes HyBryte has compelling competitive advantages over existing therapies for early stage CTCL and represents a significant commercial opportunity.

"This recently issued patent continues to expand, strengthen and protect our synthetic hypericin patent estate," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "With the support of key patient advocacy organizations, such as the Cutaneous Lymphoma Foundation, and key opinion leaders, we are moving towards marketing approval and commercialization of HyBryte, with the initial focus on the U.S. market, with partnership opportunities being explored to leverage ex-U.S. markets."

About Cutaneous T-Cell Lymphoma (CTCL)

CTCL is a class of non-Hodgkin’s lymphoma (NHL), a type of cancer of the white blood cells that are an integral part of the immune system. Unlike most NHLs which generally involve B-cell lymphocytes (involved in producing antibodies), CTCL is caused by an expansion of malignant T-cell lymphocytes (involved in cell-mediated immunity) normally programmed to migrate to the skin. These malignant cells migrate to the skin where they form various lesions, typically beginning as patches and may progress to raised plaques and tumors. Mortality is related to the stage of CTCL, with median survival generally ranging from about 12 years in the early stages to only 2.5 years when the disease has advanced. There is currently no cure for CTCL. Typically, CTCL lesions are treated and regress but usually return either in the same part of the body or in new areas.

CTCL constitutes a rare group of NHLs, occurring in about 4% of the approximate 700,000 individuals living with the disease. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of CTCL that it affects over 25,000 individuals in the U.S., with approximately 3,000 new cases seen annually.

About HyBryte

HyBryte (SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later which triggers apoptosis of the cell. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte has received orphan drug and fast track designations from the U.S. Food and Drug Administration (FDA), as well as orphan designation from the European Medicines Agency (EMA) and Promising Innovative Medicine (PIM) and "Innovation Passport" under the Innovative Licensing and Access Pathway (ILAP) from the Medicines and Healthcare Products Regulatory Agency (MHRA) of the United Kingdom.

The Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte treatment in the first cycle was safe and well tolerated.

In the second open-label treatment cycle (Cycle 2), all patients received HyBryte treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte continued to be safe and well tolerated. Additional analyses also indicated that HyBryte is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.

The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte throughout all 3 cycles of treatment, 49% of them demonstrated a treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte continued to be well tolerated despite extended and increased use of the product to treat multiple lesions. Follow-up visits were completed in Q4 2020, and the clinical study report to support the NDA is in the process of being finalized.

Overall safety of HyBryte is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. Its mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.

The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.

On May 20, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that the first data on HB-201/HB-202 alternating 2-vector therapy and expanded data on HB-201 will be featured as an oral presentation at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place June 4-8, 2021 (Press release, Hookipa Biotech, MAY 20, 2021, View Source [SID1234580342]). HB-201 and HB-202, novel arenaviral therapeutics and HOOKIPA’s lead oncology candidates, are being evaluated in an ongoing, first-in-human Phase 1/2 clinical trial (NCT04180215) for the treatment of advanced Human Papillomavirus 16-positive (HPV16+) cancers. The oral presentation will highlight safety, tolerability, immunogenicity and preliminary anti-tumor activity data from about 40 evaluable patients as of March 31.

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"We’re thrilled that our novel arenaviral immunotherapeutics for HPV16+ cancers, HB-201 and HB-202, are being featured at ASCO (Free ASCO Whitepaper). We believe our selection as an oral presentation speaks to the strength of the data and the potential of our arenaviral therapeutics to redefine success in cancer treatment," said Joern Aldag, Chief Executive Officer at HOOKIPA. "Following the preliminary HB-201 data shared in December and our translational data presentation at AACR (Free AACR Whitepaper), we’re excited to share expanded data from more patients and additional doses, including first results on HB-201/HB-202 alternating 2-vector therapy, as well as detailed safety, tolerability, translational data and preliminary anti-tumor activity results. We believe these data are very encouraging for such an early-stage program."

The abstract, outlined below, is available on the ASCO (Free ASCO Whitepaper) website:

First report of the safety/tolerability and preliminary anti-tumor activity of HB-201 and HB-202, an arenavirus-based cancer immunotherapy, in patients with HPV16+ cancers
Abstract # 2502, oral presentation
Monday, June 7, 3:00 – 6:00pm EDT
Presenter: Alan L. Ho, M.D., Ph.D., Memorial Sloan Kettering Cancer Center
Investor Event
At the conclusion of the planned ASCO (Free ASCO Whitepaper) conference events on June 7, 2021, HOOKIPA will host a live webcast event "Advancing Novel Immunotherapies: HOOKIPA ASCO (Free ASCO Whitepaper) Data Review" at 6:30 p.m. EDT. Joern Aldag, Chief Executive Officer, and Igor Matushansky, Chief Medical Officer, will provide an overview of the ASCO (Free ASCO Whitepaper) oral data and future plans for HOOKIPA’s oncology program. Dmitriy Zamarin, M.D., Ph.D., Translational Research Director in Gynecologic Medical Oncology at Memorial Sloan Kettering Cancer Center and co-investigator in this study, will also offer commentary on the significance and implications of the translational findings. The webcast and the presentation will be available within the Investors & Media section of HOOKIPA’s website at View Source To participate in a live Q&A at the end of the prepared remarks, please register here. An archived replay will be accessible for 30 days following the event.

About HB-201/HB-202
HB-201 and HB-202 are HOOKIPA’s lead oncology candidates engineered with the company’s proprietary replicating arenaviral vector platform. Each single-vector compound uses a different arenavirus backbone (LCMV for HB-201 and PICV for HB-202), while expressing the same antigen, an E7E6 fusion protein derived from HPV16. In pre-clinical studies, alternating administration of HB-201 and HB-202 resulted in a ten-fold increase in immune response and better disease control than either compound alone.

About the trial
This Phase 1/2 clinical trial is an open-label trial exploring different dose levels and dosing schedules in individuals with treatment-refractory HPV16+ cancers who progressed on standard of care, including check point inhibitors. The primary endpoint of the Phase 1 is a recommended Phase 2 dose based on safety and tolerability. Secondary endpoints include anti-tumor activity as defined by RECIST 1.1 and immunogenicity.

The trial is evaluating HB-201 as a single-vector monotherapy, as an alternating two-vector therapy with HB-202, and in combination with a PD-1 inhibitor. Participants receive HB-201/HB-202 intravenously or, for patients with an accessible lesion, the first dose can be delivered via intratumoral injection followed by intravenous dosing. Dosing every three weeks and every two weeks is being explored, as well as different dose levels.

About Human Papillomavirus
Human Papillomavirus, or HPV, is estimated to cause about 5 percent of the worldwide burden of cancers. This includes approximately 99 percent of cases in cervical, up to 60 percent of head and neck, 70 percent of vaginal and 88 percent of anal cancers.

The majority of these cancers are caused by the HPV serotype 16. Most infections with HPV are cleared from the body with no lasting consequences. However, in some cases, HPV DNA becomes integrated into chromosomal DNA. When host cells take up this DNA, they express the HPV E6 and E7 proteins. This uptake can potentially lead to cancer since expression of these proteins leads to alterations in cell cycle control, which in turn predisposes these cells to become cancerous.

On May 20, 2021 Numab Therapeutics AG, a clinical stage biopharmaceutical company developing next-generation multispecific antibody-based immunotherapies for cancer and inflammation, reported the closing of a CHF 100 million (approximately USD 110 million) Series C financing (Press release, Numab, MAY 20, 2021, View Source [SID1234580359]). The financing was co-led by new investors Novo Holdings and HBM Partners, with participation from additional new investors Forbion via its Forbion Growth Opportunities Fund, Cormorant Asset Management, BVF Partners L.P., RTW Investments L.P., funds and accounts under the management of BlackRock, Octagon Capital Advisors, and existing investors. Concurrent with the financing, Nanna Lüneborg from Novo Ventures, Matthias Fehr from HBM Partners, and Carlo Incerti from Forbion have been appointed to Numab’s Board of Directors.

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Proceeds will support acceleration and expansion of the clinical development of Numab’s lead program NM21-1480 into multiple cancer indications, and the advancement of the company’s pipeline of multi-specific antibodies in oncology and inflammation, into clinical trials.

"We are thrilled to be supported by such a strong group of investors who share our vision for the company’s future," said David Urech, Ph.D., Founder and Chief Executive Officer of Numab Therapeutics. "Combining PD-L1 blockade with tumor localized 4-1BB co-stimulation in a single molecule emerges as an attractive next-generation therapeutic strategy in solid tumors, and Numab’s lead compound NM21-1480 has best-in-class potential. The financing will help us to maximize the value of this asset by significantly expanding the clinical development program and accelerating toward phase 2 proof-of-concept."

"We are impressed by the biological insights that went into the engineering of NM21-1480 and by the innovative pipeline that Numab has established. We believe that Numab’s Lambda-capTM and MATCHTM technologies can provide treatment options with favorable benefit-to-risk profiles," said Matthias Fehr, Head Private Equity at HBM Partners.

"Backing companies built on clearly differentiated science in areas of major unmet medical need is at the heart of Novo Holdings’ investment strategy. We are thus thrilled to co-lead Numab’s Series C financing to support the company at a pivotal stage in growth," commented Nanna Lüneborg, Partner at Novo Ventures.