On March 31, 2021 Provectus (OTCQB: PVCT) reported that Melanoma Research had published results from an investigator-led, single-center study of Australian in-transit melanoma (ITM) patients who received intralesional (aka intratumoral) PV-10 under a Provectus-sponsored expanded access (aka compassionate use) program (EAP) (Press release, Provectus Biopharmaceuticals, MAR 31, 2021, View Source [SID1234577446]).

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The Melanoma Research article, entitled "Treatment of in-transit melanoma metastases using intralesional PV-10," detailed the experience of investigators at Melanoma Institute Australia (MIA; formerly the Sydney Melanoma Unit) in Sydney, Australia who treated 48 patients from 2008 to 2016.

The article may be accessed at: View Source (subscription required).

Key highlights of the Melanoma Research publication:

Baseline characteristics: 58% women; median age of 75.1 years (range 21.5-93.5),
Median of 5 lesions treated with PV-10 (interquartile range [IQR] 2-7),
Median of 1 PV-10 treatment (IQR 1-2),
46% complete response (CR) and 86% overall response rate (ORR) in patients, and
12- and 24-month overall survival (OS) rates of 77% and 56%, respectively.
This is the third publication about single-agent PV-10 treatment of ITM lesions under Provectus’ EAP (which has treated about 200 patients to date) and the fifth publication overall. Previous publications describing EAPs led by other major Australian medical institutions and the Company’s Phase 1 and 2 clinical trials at Australian and U.S. sites include:

"Intralesional PV-10 for the treatment of in-transit melanoma metastases – Results of a prospective, non-randomized, single center study" (Read et al. J Surg Oncol 2018), which describes the EAP at Princess Alexandra Hospital in Brisbane: 45 patients treated from 2008 to 2015; 42% patient CR (86% ORR); median of 1 PV-10 treatment (range 1-4),

"Intralesional PV-10 for in-transit melanoma – A single-center experience" (Lippey et al. J Surg Oncol 2016; subscription required), which describes the EAP at Peter MacCallum Cancer Centre in Melbourne: 19 patients treated from 2010 to 2014; 26% patient CR (52% ORR); most patients received 1 PV-10 treatment,

"Phase 2 Study of Intralesional PV-10 in Refractory Metastatic Melanoma" (Thompson et al. Ann Surg Oncol 2015): 28 (out of 80) patients with all known lesions treated; median of 1 PV-10 treatment (range 1-4); 50% patient CR (71% ORR), and

"Chemoablation of metastatic melanoma using intralesional Rose Bengal" (Thompson et al. Mel Res 2008; subscription required): 11 (out of 20) patients treated in the Phase 1 trial; 1 PV-10 treatment; 27% patient CR (54% ORR).
Dominic Rodrigues, Vice Chair of the Company’s Board of Directors, said, "Patients who present with in-transit melanoma lesions suffer from a rare disease that is a serious, life-threatening condition with no approved therapies. No drug therapies provide the rapid, sustained reduction of tumor burden, with low toxicity, that investigational drug product PV-10 does. Injection of lesions with PV-10 is globally recognized as a treatment option for in-transit melanoma patients in clinical practice guidelines, including those for Australiaa, Canadab, Europec, and the U.S.d"

Mr. Rodrigues added "Given the high percentage of in-transit melanoma lesions that achieve complete response from typically one or two PV-10 injections, and its established safety profile, we believe there is a role for PV-10 in the treatment of in-transit melanoma lesions for those patients who are not candidates for systemic drug therapies."

About PV-10

Intralesional (IL) administration of PV-10 for the treatment of solid tumor cancers can yield immunogenic cell death within hours of tumor injection, and induce tumor-specific reactivity in circulating T cells within days.1,2,3 This IL PV-10-induced functional T cell response may be enhanced and boosted in combination with immune checkpoint blockade (CB).4 In CB-refractory advanced cutaneous melanoma, IL PV-10 may restore disease-specific T cell function, which may also be prognostic of clinical response. IL PV-10 has been administered to over 450 patients with cancers of the skin and of the liver. It is administered under visual, tactile or ultrasound guidance to superficial malignancies, and under CT or ultrasound guidance to tumors of the liver.

About Rose Bengal Disodium

RBD is 4,5,6,7-tetrachloro-2′,4′,5′,7′-tetraiodofluorescein disodium, a halogenated xanthene and Provectus’ proprietary lead molecule. Provectus’ current Good Manufacturing Practices (cGMP) RBD is a proprietary pharmaceutical-grade drug substance produced by the Company’s quality-by-design (QbD) manufacturing process to exacting regulatory standards that avoids the formation of uncontrolled impurities currently present in commercial-grade rose bengal. Provectus’ RBD and cGMP RBD manufacturing process are protected by composition of matter and manufacturing patents as well as trade secrets.

An IL formulation (i.e., by direct injection) of cGMP RBD drug substance, cGMP PV-10, is being developed as an autolytic immunotherapy drug product for solid tumor cancers.

IL PV-10 is also undergoing preclinical study for relapsed and refractory pediatric solid tumor cancers, such as neuroblastoma, Ewing sarcoma, rhabdomyosarcoma, and osteosarcoma.5,6

A topical formulation of cGMP RBD drug substance, PH-10, is being developed as a clinical-stage immuno-dermatology drug product for inflammatory dermatoses, such as atopic dermatitis and psoriasis. RBD can modulate multiple interleukin and interferon pathways and key cytokine disease drivers.7

Oral formulations of cGMP RBD are undergoing preclinical study for relapsed and refractory pediatric blood cancers, such as acute lymphocytic leukemia and acute myelomonocytic leukemia.8,9

Oral formulations of cGMP RBD are also undergoing preclinical study as prophylactic and therapeutic treatments for high-risk adult solid tumor cancers, such as head and neck, breast, pancreatic, liver, and colorectal cancers.

Different formulations of cGMP RBD are also undergoing preclinical study as potential treatments for multi-drug resistant (MDR) bacteria, such as Gram-negative bacteria.

Topical formulations of cGMP RBD are also undergoing preclinical study as potential treatments for diseases of the eye, such as infectious keratitis

Tumor Cell Lysosomes as the Seminal Cancer Drug Target

Lysosomes are the central organelles for intracellular degradation of biological materials, and nearly all types of eukaryotic cells have them. Discovered by Christian de Duve, MD in 1955, lysosomes are linked to several biological processes, including cell death and immune response. In 1959, de Duve described them as ‘suicide bags’ because their rupture causes cell death and tissue autolysis. He was awarded the Nobel Prize in 1974 for discovering and characterizing lysosomes, which are also linked to each of the three primary cell death pathways: apoptosis, autophagy, and necrosis.

Building on the Discovery, Exploration, and Characterization of Lysosomes

Cancer cells, particularly advanced cancer cells, are very dependent on effective lysosomal functioning.10 Cancer progression and metastasis are associated with lysosomal compartment changes11,12, which are closely correlated (among other things) with invasive growth, angiogenesis, and drug resistance13.

RBD selectively accumulates in the lysosomes of cancer cells upon contact, disrupting the lysosomes and causing the cells to die. Provectus2,14, external collaborators5, and other researchers15,16,17 have independently shown that RBD triggers each of the three primary cell death pathways: apoptosis, autophagy, and necrosis.

Cancer Cell Autolytic Death via RBD: RBD-induced autolytic cell death, or death by self-digestion, in Hepa1-6 murine hepatocellular carcinoma (HCC) cells can be viewed in this Provectus video of the process (ethidium homodimer 1 [ED-1] stains DNA, but is excluded from intact nuclei; lysosensor green [LSG] stains intact lysosomes; the video is provided in 30-second frames, with a duration of approximately one hour). Exposure to RBD triggers the disruption of lysosomes, followed by nucleus failure and autolytic cell death. Identical responses have been shown by the Company in HTB-133 human breast carcinoma (which can be viewed in this Provectus video of the process, with a duration of approximately two hours) and H69Ar human multidrug-resistant small cell lung carcinoma. Cancer cell autolytic cell death was reproduced by research collaborators in neuroblastoma cells to show that lysosomes are disrupted upon exposure to RBD.5

Tumor Autolytic Death via RBD: RBD causes acute autolytic destruction of injected tumors (via autolytic cell death), mediating the release of danger-associated molecular pattern molecules (DAMPs) and tumor antigens; release of these signaling factors may initiate an immunologic cascade where local response by the innate immune system may facilitate systemic anti-tumor immunity by the adaptive immune system. The DAMP release-mediated adaptive immune response activates lymphocytes, including CD8+ T cells, CD4+ T cells, and NKT cells, based on clinical and preclinical experience in multiple tumor types. Mediated immune signaling pathways may include an effect on STING, which plays an important role in innate immunity.9

Orphan Drug Designations (ODDs)

ODD status has been granted to RBD by the U.S. Food and Drug Administration for metastatic melanoma in 2006, hepatocellular carcinoma in 2011, neuroblastoma in 2018, and ocular melanoma (including uveal melanoma) in 2019.

Intellectual Property

Provectus’ IP includes a family of US and international (a number of countries in Asia, Europe, and North America) patents that protect the process by which cGMP RBD and related halogenated xanthenes are produced, avoiding the formation of previously unknown impurities that exist in commercial-grade rose bengal in uncontrolled amounts. The requirement to control these impurities is in accordance with International Council on Harmonisation (ICH) guidelines for the manufacturing of an injectable pharmaceutical. US patent numbers are 8,530,675, 9,273,022, and 9,422,260, with expirations ranging from 2030 to 2031.

The Company’s IP also includes a family of US and international (a number of countries in Asia, Europe, and North America) patents that protect the combination of RBD and CB (e.g., anti-CTLA-4, anti-PD-1, and anti-PD-L1 agents) for the treatment of a range of solid tumor cancers. US patent numbers are 9,107,887, 9,808,524, 9,839,688, and 10,471,144, with expirations ranging from 2032 to 2035; US patent application numbers include 20200138942 (i.e., 16/678,133), which has been allowed.

On March 31, 2021 Cancer Genetics, Inc. (the "Company" or "CGI") (Nasdaq: CGIX), an emerging leader in novel drug discovery techniques, and StemoniX, Inc. ("StemoniX"), a company empowering the discovery of new medicines through the convergence of novel human biology and software technologies, reported their recently approved transaction has closed, and in connection with the merger, Cancer Genetics, Inc. was renamed Vyant Bio, Inc. ("Vyant Bio") effective March 30, 2021 (Press release, Cancer Genetics, MAR 31, 2021, View Source [SID1234577445]). StemoniX will operate as a wholly-owned subsidiary of the Company.

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Vyant Bio will be traded on the Nasdaq under the symbol VYNT beginning on March 31, 2021. The name and ticker change will align the Company’s strategic focus on the creation of a leading biotechnology and drug discovery platform business.

Vyant Bio is now positioned to integrate human-powered scientific and technology-based systems with years of preclinical experience to de-risk and accelerate discovery and development of preclinical and clinical pipelines for biopharma partners as well as for the Company’s proprietary pipeline. The merger of the two companies represents a bold new chapter in drug discovery, creating a unique platform using in vivo, in vitro, and in silico technologies to identify repurposed and novel therapeutics to fight diseases in neurology, oncology, and cardiology.

The merger has attracted highly experienced board and management team members who share a vision of creating world-class capabilities. Management teams from both companies will join forces for Vyant Bio, led by health science veteran Jay Roberts, who will serve as Chief Executive Officer, innovation thought-leader Ping Yeh, Vyant Bio’s new Chief Innovation Officer, and Andrew LaFrence, incoming Chief Financial Officer, an accomplished public company financial executive and former KPMG audit partner.

"Vyant Bio will now jumpstart an exciting clinical pipeline of therapeutics from its Drug Discovery Engine for purposes of out-licensing to partners worldwide," stated Jay Roberts. "We worked tirelessly throughout 2020 and Q1 2021 to identify and complete this merger with StemoniX and are very excited to bring the best of these two companies together in a shared culture and vision for the future – to create safer and more effective therapeutics and meaningful shareholder value."

"We are very pleased to be announcing the new name and branding initiative. Vyant Bio was created as it represents a vital, vibrant, innovative new force in drug discovery, derived from the French words "vie" and "avant" – together, they represent our mission to transform lives with new treatments derived from leading-edge science and technology. We believe the combined companies create a new path for innovation, with a human-powered approach that will de-risk and accelerate decision making to more rapidly bring important therapeutics to patients," said Ping Yeh.

Under the terms of the merger agreement, the Company will issue an aggregate of 17,977,272 shares of its common stock to the former holders of StemoniX common stock, preferred stock, convertible notes and certain warrants. It will also issue options to purchase an aggregate of 893,179 shares of Common Stock to the holders of StemoniX options and warrants expiring in 2026 to purchase 143,890 shares of Common Stock to the holder of a StemoniX warrant.

Immediately after the merger, there were approximately 28,984,458 million shares of Common Stock of the Company outstanding.

H.C. Wainwright & Co. acted as financial advisor to Cancer Genetics, Inc., and Lowenstein Sandler LLP served as legal counsel to Cancer Genetics. Roth Capital Partners and Northland Capital Markets acted as financial advisors to StemoniX, and Taft Stettinius & Hollister LLP served as legal counsel to StemoniX.

A Current Report on Form 8-K containing more detailed information regarding the merger transaction will be filed with the Securities and Exchange Commission.

On March 31, 2021 Istari Oncology, Inc., a clinical-stage biotechnology company, reported that the first patient was dosed in the LUMINOS-102 phase 2 clinical trial, which will assess the safety and efficacy of PVSRIPO alone or in combination with a programmed death receptor-1/ligand 1 (PD-1/L1) inhibitor in patients with melanoma who are resistant to these checkpoint therapies (Press release, Istari Oncology, MAR 31, 2021, View Source [SID1234577443]).

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PVSRIPO is a novel viral immunotherapy that activates the innate and adaptive immune system to stimulate the production of a functional, systemic anticancer CD8+ T cell response. Following positive phase 1 results,1 the phase 2 trial will further explore PVSRIPO’s impact on this population of patients in severe need of additional therapeutic options.

"Anti-PD-1/L1 therapies have been a major advancement in melanoma treatment, however, many patients develop resistance or never respond in the first place," said Matt Stober, President and Chief Executive Officer at Istari Oncology. "We are very optimistic about the prospects for the phase 2 trial. PVSRIPO monotherapy has already shown clinical activity in this population, and its mechanism is synergistic with anti-PD-1/L1 therapies, so we believe the combination may provide even more benefit."

"As the use of anti-PD-1/L1 therapies has grown, so too has the need for new treatments as patients experience primary or acquired resistance and must resort to therapeutic approaches with a high incidence of serious adverse events," said Garrett Nichols, MD, MS, Chief Medical Officer at Istari Oncology. "Our goal is to address this unmet need, and LUMINOS-102 will be a critical step in determining whether PVSRIPO can rekindle antitumor responses in the PD-1/L1 refractory population without adding any significant toxicity."

LUMINOS-102 is an open-label, phase 2, multicenter randomized trial (NCT04577807) in patients with melanoma that have progressed on anti–PD1/L1 therapy and will characterize the safety, tolerability and initial efficacy of PVSRIPO intratumoral injection alone (Arm 1) and in combination with a PD-1 inhibitor (Arm 2). An interim analysis is planned once 20 patients have been randomized and treated for 3 months. Patient outcomes, including objective response rates (by RECIST criteria), durability of responses, progression free survival and overall survival will be measured over a 24-month time frame.

LUMINOS-102 builds upon a successful phase 1 study of PVSRIPO monotherapy in anti–PD-1 refractory advanced melanoma presented by Dr. Georgia Beasley and colleagues at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2020 Annual Meeting and in-press for an upcoming issue of the Journal for ImmunoTherapy of Cancer (JITC). In the phase 1 study, the overall response rate in subjects who received three single intratumoral injections 3 weeks apart (the maximum number administered in the study) was 67% (4/6), suggesting PVSRIPO was able to initiate or rekindle responses in patients who have failed anti–PD-1 therapy. Responses were observed in both injected and noninjected tumors, suggestive of an abscopal response. No serious adverse events or dose-limiting toxicities were observed.

"Being based on the poliovirus vaccine, our team was intrigued by the potential for PVSRIPO to leverage an immunological recall response to fight cancer in patients who have been vaccinated against polio. This and other unique mechanisms as well as the responses seen in the phase 1 trial encouraged us to get involved in LUMINOS-102," noted Ding Wang, MD, ?Director of the Phase 1 Program and Associate Director of Clinical Trials Office at Henry Ford Cancer Institute, Detroit, MI. "LUMINOS-102 will build on these data and further evaluate the ability of PVSRIPO to generate a systemic immune response, important for patients with unresectable anti–PD-1 refractory melanoma. We’re proud to be the first site to treat a participant and looking forward to continuing enrollment."

The LUMINOS-102 phase 2 trial will be conducted across more than 20 research sites across the US including Henry Ford Cancer Institute.

For more information about Istari Oncology and their ongoing clinical trials, visit istarioncology.com.

About PVSRIPO
PVSRIPO is an investigational immunotherapy based on the live attenuated Sabin type 1 poliovirus vaccine that has been genetically modified for safety. PVSRIPO has a distinct target (the poliovirus receptor, CD155), which is expressed on virtually all solid tumors and antigen-presenting cells. Via CD155, PVSRIPO targets tumors with two primary mechanisms: 1) direct damage to and killing of cancerous cells; and 2) engaging innate and adaptive antitumor immune responses via nonlethal infection of antigen presenting cells in the tumor, which stimulates a specific signaling pathway resulting in a sustained, robust type-I/III interferon-dominant response, with minimal release of unwanted cytokines. Its effects are potentiated by prior vaccination against poliovirus. PVSRIPO has been granted Breakthrough Therapy Designation and Orphan Status by the FDA in recurrent glioblastoma. PVSRIPO has also been granted Orphan Status by the FDA for advanced melanoma.

About Melanoma
There are estimated to be over 12,000 new and recurrent cases of advanced, unresectable melanoma diagnosed in the U.S. each year, and around 7,000 deaths. While immune checkpoint inhibitors have dramatically improved the outlook for advanced melanoma patients today, most patients treated with these immunotherapies are either primary nonresponders or eventually develop immune-refractory progressive disease and require additional therapy.

On March 31, 2021 Galectin Therapeutics Inc. (NASDAQ: GALT), the leading developer of therapeutics that target galectin proteins,reported financial results and provided a business update for the year ended December 31, 2020 (Press release, Galectin Therapeutics, MAR 31, 2021, View Source [SID1234577441]). These results are included in the Company’s Annual Report on Form 10-K, which has been filed with the U.S. Securities and Exchange Commission and is available at www.sec.gov.

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Joel Lewis, Chief Executive Officer and President of Galectin Therapeutics, said, "I am very encouraged by the progress achieved in fiscal 2020, and remain extremely optimistic for 2021. Given the current challenging environment, I am proud of our success, highlighted by site activations and ongoing enrollment of our innovative NAVIGATE study. This global program continues to be the only active late-stage trial of patients with compensated NASH cirrhosis, where the medical need is greatest and with a clinically meaningful endpoint. Our concurrent hepatic impairment study will also provide important information on belapectin tolerance, safety and exposure in advanced cirrhotic patients.

Over the course of the last year at the Board’s direction we have taken aggressive steps to strengthen our organization, adding Pol Boudes as Chief Medical Officer, as well as Mr. Richard Zordani and Dr. Elissa Schwartz to our Board of Directors. These changes informed my decision to accept the role of Chief Executive Officer, and they afforded me the confidence to receive 80% of my compensation in the

form of Galectin stock. Additionally, I believe this breadth of talent reinvigorated Galectin and placed the Company in a position to monetize our assets. This has served to strengthen my commitment to my compensation strategy, which aligns my interests with all shareholders.

More recently, the peer-reviewed publication of a well-recognized mouse model has shown that the combination of belapectin, a galectin-3 inhibitor, with immunotherapy reprograms the tumor microenvironment. This favors anti-tumor immunity, results in better anti-tumor activity, and most importantly, brings further rationale for our ongoing cancer trial combining belapectin with Keytruda, a potent PD-1 inhibitor. Providence Cancer Institute is currently conducting the study and preliminary results suggest improved activity and, potentially, improved tolerance of this regimen.

I am extremely confident in our science, our team, and our progress," concluded Lewis. The upcoming year will be dedicated to advancing our trial in NASH cirrhosis and supporting investigations of belapectin’s safety and efficacy in other indications, such as the ongoing cancer trial in conjunction with the Providence Cancer Institute. I also want to recognize the outstanding efforts of our entire team, who persevered through the challenges precipitated by COVID-19 in the interest of developing a therapy for NASH cirrhosis, a critical, unmet medical need. Let me once again thank the investigators and patients participating in our NAVIGATE trial, where a positive outcome would be very clinically relevant for patients with NASH cirrhosis."

Richard E. Uihlein, Chairman of the Board, added, "I want to echo Joel’s sentiment and thank Pol, Jack and our entire team for their dedication throughout

this past year, especially their commitment to initiating our exciting NAVIGATE trial under less than optimal circumstances due to the global pandemic. Joel has proven to be the leader we all expected, and I am pleased with the progress he has achieved since assuming the role and confident in his ability to unlock the value of our proprietary compound, belapectin. Peer-reviewed research, such as that recently published in OncoImmunology, clearly confirms our basic scientific premise regarding belapectin’s anti-inflammatory characteristics in a broad range of fibrosis as well as its ability to potentially enhance the efficacy of cancer therapies. As such, the NAVIGATE trial represents an opportunity to further demonstrate the anti-inflammatory activity of belapectin, which would open up vast new opportunities to investigate other indications and establish our compound as a foundation for a platform technology."

NAVIGATE Trial Update

The NAVIGATE trial uses a seamless, adaptive design to confirm dose selection and reaffirm the observed efficacy of belapectin to prevent the development of esophageal varices in the NASH-CX trial. Pre-planned adaptations will inform the larger Phase 3 trial component.

Key clinical study milestones:

First patient randomized August 2020

130+ sites, 12 countries in North America, Europe, Asia and Australia

Phase 2b part to Interim Analysis will be ~315 patients

Recruiting period for phase 2b portion now expected to conclude around the end of 2021 due to COVID-19 impact on recruitment

Key inclusion criteria – NASH cirrhosis (baseline or historical liver biopsy), clinical sign of portal hypertension, no esophageal varices (esophago-gastro endoscopy)

Interim analysis expected late 2023

Peer-reviewed publication, Scientific Presentations and Conferences

OncoImmunology published a peer-reviewed article describing how belapectin, a potent galectin-3 inhibitor, in combination with an anti-OX40 (CD134) monoclonal antibody, reduces tumor progression compared to either agent alone. The paper, titled "Galectin-3 inhibition with belapectin combined with anti-OX40 therapy reprograms the tumor microenvironment to favor anti-tumor immunity," describes results from a collaboration between Galectin Therapeutics and Providence Cancer Institute highlighting the mechanism of action of the combination which is explained by a reduction in myeloid-derived suppressor cell infiltration and function coupled to an increase in T-cell effector function. For many years, galectin-3 has been known to play a key role in the control of tumor-induced immunosuppression. Galectin-3 acts to maintain tumor growth, in part, by supporting the generation of suppressive macrophages and inhibiting T cell function. This creates an attractive rationale for the use of a galectin-3 inhibitor, such as belapectin, to improve anti-tumor activities of multiple cancer therapies.

Financial Results

For the year ended December 31, 2020, the Company reported a net loss applicable to common stockholders of $23.6 million, or ($0.41) per share, compared to a net loss applicable to common stockholders of $20.2 million, or ($0.39) per share for the full year 2019. The increase is largely due to an increase in research and development expenses related to our NAVIGATE clinical trial, partially offset by a non-cash, one-time warrant modification charge of $6.6 million in 2019.

Research and development expense for 2020 was $18.0 million compared with $7.5 million for 2019. The increase was primarily due to costs related to our NAVIGATE clinical trial, along with preparations and some preclinical activities incurred in support of the clinical program, such as development and reproductive toxicity studies, clinical supplies and other supportive activities. General and administrative expenses for 2020 were $5.5 million, down from $6.0 million for the full year 2019, primarily due to decreases in legal, investor relations and non-cash stock-based compensation expenses partially offset by an increase in insurance expenses.

As of December 31, 2020, the Company had $27.1 million of cash and cash equivalents. The Company also has a $10 million unsecured line of credit, under which no borrowings have been made to date. The Company believes it has sufficient cash, including availability under the line of credit, to fund currently planned operations and research and development activities through at least March 31, 2022.

The Company expects that it will require more cash to fund operations after March 31, 2022, and believes it will be able to obtain additional financing as needed. The currently planned operations include costs related to our adaptively designed NAVIGATE Phase 2b/3 clinical trial. Currently, we expect to require an additional approximately $45-$50 million to cover costs of the trial to reach the planned interim analysis estimated to occur in the second half of 2023 along with drug manufacturing and other scientific support activities and general and administrative costs and further amounts to complete the Phase 3 portion of the trial. However, there can be no assurance that we will be successful in obtaining such new financing or, if available, that such financing will be on terms favorable to us.

On March 31, 2021 BridgeBio Pharma, Inc. (Nasdaq: BBIO), through its affiliate QED Therapeutics, Inc., and Helsinn Group reported a global collaboration and licensing agreement (the "Agreement") to further develop and commercialize QED Therapeutics’ FGFR1-3 inhibitor, infigratinib, in oncology and all other indications except for skeletal dysplasias (including achondroplasia) (Press release, BridgeBio, MAR 31, 2021, View Source [SID1234577440]). Completion of the Agreement is subject to regulatory review and customary closing conditions, which are expected to occur in the second quarter of 2021.

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Infigratinib is an orally administered, ATP-competitive, tyrosine kinase inhibitor that is designed to inhibit FGFR, and being investigated for treatment of individuals with FGFR-driven conditions, including cholangiocarcinoma (bile duct cancer), urothelial carcinoma (urinary tract and bladder cancer), and other FGFR-driven cancers.

Under the terms of the Agreement, BridgeBio will retain all rights to infigratinib in skeletal dysplasia, including achondroplasia. Subject to U.S. Food and Drug Administration ("FDA") approval, QED and Helsinn will co-commercialize infigratinib in oncology indications in the U.S. and will share profits and losses on a 50:50 basis. Helsinn will have exclusive commercialization rights and lead commercialization for infigratinib in non-skeletal dysplasia indications outside of the U.S., excluding China, Hong Kong and Macau, which are covered by BridgeBio’s strategic development and commercialization collaboration with LianBio. Under the Agreement, BridgeBio will be eligible to receive more than $2 billion in upfront, regulatory and commercial milestones, as well as tiered royalties on adjusted net sales from Helsinn Group.

"We are privileged to partner with Helsinn as we strive to unlock the full potential of infigratinib for patients with FGFR-driven cancers," said BridgeBio CEO and founder Neil Kumar, Ph.D. "Helsinn has an impressive track record of advancing and commercializing oncology therapies around the globe. Our hope is that partnering with Helsinn will significantly strengthen our anticipated upcoming launch of infigratinib and our ongoing research into infigratinib’s potential across other cancer indications."

Riccardo Braglia, Helsinn Group Vice Chairman and CEO, commented, "As a leader in oncology therapeutics and supportive care, Helsinn is always looking to partner with high quality companies. The combination of BridgeBio and its lead oncology product candidate, infigratinib, fall into the strategic sweet spot of a quality company and product with which we look to work. We are highly excited by the potential positive impact this collaboration can deliver for patients around the world."

BridgeBio and Helsinn Group intend to pursue an ambitious co-development plan in oncology indications, including clinical investigation underway in first-line cholangiocarcinoma and adjuvant urothelial cancer. This plan will be underpinned by close collaboration among the parties, with the aim of developing new treatments for patients with FGFR-driven cancers. As infigratinib heads toward potential approval and commercialization in a range of oncology indications, Helsinn’s unique integrated licensing business model will enable its distribution to reach patients globally.

The FDA has accepted the New Drug Application ("NDA") for infigratinib for patients with previously-treated advanced cholangiocarcinoma ("CCA") harboring an FGFR2 gene fusion or rearrangement. The NDA has been granted Priority Review designation and is being reviewed under the Real-Time Oncology Review ("RTOR") pilot program, an initiative of the FDA’s Oncology Center of Excellence designed to expedite the delivery of safe and effective cancer treatments to patients. Additionally, infigratinib is currently under review in Australia and Canada under Project Orbis, an initiative of the FDA’s Oncology Center of Excellence that allows for concurrent submission and review of oncology drugs among participating international regulatory agencies.