On March 19, 2020 National Cancer Institute (NCI), part of the National Institutes of Health, reported that offers new insight into genetic alterations associated with osteosarcoma, the most common cancerous bone tumor of children and adolescents (Press release, US NIH, MAR 19, 2020, View Source [SID1234555739]). The researchers found that more people with osteosarcoma carry harmful, or likely harmful, variants in known cancer-susceptibility genes than people without osteosarcoma. This finding has implications for genetic testing of children with osteosarcoma, as well as their families.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The study was published March 19, 2020, in JAMA Oncology.

"With this study, we wanted to find out how many people with osteosarcoma may have been at high risk for it because of their genetics," said Lisa Mirabello, Ph.D., of NCI’s Division of Cancer Epidemiology and Genetics (DCEG), who led the research. "We not only learned that at least a quarter of the people in the study with osteosarcoma had a variant in a gene known to predispose someone to cancer, we also uncovered variants that had never before been associated with this cancer."

In the study, the researchers looked for harmful (or likely harmful) variants in 238 known cancer-susceptibility genes in DNA samples from 1,244 people with osteosarcoma and compared the frequency of such variants with that in people in a cancer-free control group. They identified a harmful or likely harmful variant in a known cancer-susceptibility gene in 28% of the people with osteosarcoma. By contrast, only 12% of people in the cancer-free control group had such a variant.

When the authors looked at a subset of 166 genes that are known to be inherited in an autosomal dominant fashion—that is, where inheriting one alteration from one parent is sufficient to increase cancer risk—they saw harmful or likely harmful variants in about 18% of the patients but only 5% of controls. And another 25% of the patients had a rare variant of uncertain significance that was predicted to be harmful.

Patients who had harmful variants were younger at age of osteosarcoma diagnosis, on average, than patients who did not (15.3 years versus 16.9 years). In addition, the youngest children in the study (ages 0-10 years) had the highest prevalence of harmful variants.

If a child is found to have a gene variant associated with osteosarcoma, the authors say that genetic testing may be warranted for the child’s family members, who may also carry the variant. Family members who have the variant could undergo screening for the cancers associated with that variant, potentially leading to earlier detection.

"The idea is, if you have a new patient with osteosarcoma, we found that a quarter of them have a [harmful] variant in a gene associated with increased risk of other cancers," said Sharon Savage, M.D., senior author of the study, also of DCEG. "Genetic testing of the patient likely makes sense, because they could be at risk of other cancers, and they might have family members who carry the variant who might be at risk of other cancers."

Because osteosarcoma is a rare cancer, the researchers assembled case samples from 10 international research centers, making this the largest collection to date of a pediatric solid cancer evaluated for cancer-susceptibility gene variants.

The researchers found harmful or likely harmful variants in several cancer-susceptibility genes that hadn’t been associated with osteosarcoma—or, indeed, pediatric cancers—before. Because the biological function for the variants newly found to be associated with osteosarcoma remain unknown, this could open new areas of research related to cancer susceptibility.

This study is part of a larger pediatric cancer susceptibility research program at NCI through which researchers are studying common and rare causes of osteosarcoma and other pediatric cancers. In earlier research, Dr. Mirabello found that TP53 gene variants, which are associated with the cancer predisposition disease Li-Fraumeni Syndrome, were more common than previously thought in children and adolescents with osteosarcoma. In the current study, TP53 gene variants had the highest frequency of the harmful or likely harmful cancer-susceptibility gene variants in children with osteosarcoma, another finding that Dr. Mirabello said warrants additional research.

On March 19, 2020 Vaxart, Inc., a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, reported financial results for the fourth quarter and full year ended December 31, 2019, and provided a corporate update (Press release, Vaxart, MAR 19, 2020, View Source [SID1234555707]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"SARS CoV-2, the coronavirus that causes COVID-19, is primarily transmitted by viral particles that enter through the nose, mouth or eyes, and cause a respiratory infection," said Wouter Latour, MD, chief executive officer of Vaxart Inc. "Unlike injectable vaccines, our oral vaccines have been shown to protect against respiratory infection based on mucosal immunity, the first line of defense for such infections. This could be essential for an effective vaccine that protects the population from COVID-19. In addition, the Vaxart vaccine would be administered using a room temperature-stable tablet, an enormous logistical advantage in large vaccination campaigns."

"This outbreak is a call to duty for all of us here at Vaxart and we are highly focused on the development of the COVID-19 vaccine," Dr. Latour continued. "Accordingly, we have put several vaccine programs on hold, including the norovirus vaccine program for which we recently successfully completed a Phase 1 study and for which we are actively seeking a development partner, as well as our therapeutic HPV vaccine program. The Janssen-partnered Universal Flu program is fully active and on track to be completed in the coming weeks."

All Vaxart vaccines are based on its oral Vector-Adjuvant platform, VAASTTM. In a recent clinical study published in the Lancet Infectious Diseases, the Company demonstrated that the Vaxart oral H1 influenza tablet vaccine primarily protected against infection based on mucosal immunity, in contrast to the injectable flu vaccine that protected primarily through systemic immunity.

In January, Vaxart initiated a program to develop a coronavirus vaccine candidate based on its VAASTTM platform and is currently producing multiple research grade COVID-19 vaccine candidates to be evaluated in a preclinical model. In addition, Vaxart announced this week that it had entered into an agreement with Emergent BioSolutions Inc. Per the agreement, development services are to begin immediately and upon Vaxart’s election, Emergent is expected to produce bulk cGMP vaccine in time for a Phase 1 clinical study to begin early in the second half of 2020.

Other Corporate Updates:

The Universal Influenza vaccine collaboration with Janssen is proceeding as planned and remains on schedule to provide results in 1H 2020, barring delays due to the Coronavirus outbreak.

Published results from the H1 seasonal influenza oral tablet vaccine challenge study in the Lancet Infectious Diseases. The study demonstrated that Vaxart’s oral tablet influenza vaccine generated a 39 percent reduction in clinical disease relative to placebo, compared to a 27 percent reduction by Fluzone. It also reduced infection rates by 47 percent, compared to 43 percent by Fluzone.

Restructured the manufacturing and process development departments resulting in, among other things, a reduction in headcount.

Raised approximately $9.8 million in net proceeds from the exercise of Warrants during the first quarter of 2020 to date.

Completed a registered direct offering in March 2020, through which Vaxart raised aggregate gross proceeds of $10.0 million from the sale of 4.0 Million shares of common stock and warrants to purchase 2.0 million shares of common stock that are exercisable for $2.50 per share.

Regained compliance with the continuing listing standards of The Nasdaq Capital Market.

Financial Results for the Three Months Ended December 31, 2019

Vaxart reported a net loss of $6.4 million for the fourth quarter of 2019 compared to $4.9 million for the fourth quarter of 2018. The principal reason for the increase was a charge of $4.9 million for restructuring, partially offset by an increase in revenue and a reduction in research and development expenditure.

Revenue for the quarter was $3.9 million compared to $1.8 million in the fourth quarter of 2018. The increase was mostly due to non-cash royalty revenue related to the sale of future royalties.

For the quarter ended December 31, 2019, royalty revenue from Inavir increased by $2.2 million, or 148%, compared to the quarter ended December 31, 2018.

Financial Results for the Full Year 2019

Vaxart reported a net loss of $18.6 million for 2019 compared to $18.3 million for 2018. The principal reason for the increase was the absence of a bargain purchase gain of $6.8 million, partially offset by an increase in revenue of $5.7 million.

Revenue for the year was $9.9 million compared to $4.2 million in 2018. The increase was mostly due to $3.5 million of 2018 revenue having been recorded in the pre-Merger period of January 1-February 13, 2018 and increased royalty revenue from Inavir in the fourth quarter of 2019.

Research and development expenses were $14.5 million for 2019 compared to $17.3 million for 2018. The decrease was mainly due to the absence of clinical trials costs for teslexivir and HHS BARDA contract costs and a reduction in pre-clinical research costs, partially offset by higher manufacturing and clinical trial costs incurred in Vaxart’s norovirus program.

General and administrative expenses were $6.2 million for the year compared to $6.7 million for 2018. The decrease was mainly due to reductions in legal fees and other costs associated with becoming a public company.

Other operating expenses were $4.9 million for 2019 due to restructuring costs related to the suspension of Vaxart’s manufacturing operations, compared to $2.0 million for 2018, which related to the impairment of intangible assets related to teslexivir and the sublease of Vaxart’s premises in Georgia.

Vaxart ended the year with cash and cash equivalents of $13.5 million compared to $11.5 million at December 31, 2018. The increase was primarily due to funds raised via issuances of equity totaling $19.8 million during 2019, partially offset by cash used in operations totaling $13.1 million and the repayment of debt for $3.8 million.

On March 19, 2020 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies, reported that it will report its fourth quarter and year-end 2019 financial results on Thursday, March 26, 2020 (Press release, IGM Biosciences, MAR 19, 2020, View Source [SID1234555696]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In connection with the earnings release, IGM’s management team will host a live conference call and webcast at 4:30 p.m. ET on Thursday, March 26, 2020, to discuss the Company’s financial results and provide a corporate update.

Conference ID: 2991398
Domestic Dial-in Number: (866) 649-1996
International Dial-in Number: (409) 217-8769
Live Webcast: View Source

A replay of the audio webcast will be available for 30 days on the Investors section of the Company’s website, www.igmbio.com.

On March 19, 2020 Concure Oncology, whose mission is to ease the burden for women facing early-stage breast cancer by providing an innovative new kind of radiation treatment option, has reported that it has secured $2.5 million in new capital, helping to give new hope to women dealing with difficult treatment for breast cancer (Press release, Concure Oncology, MAR 19, 2020, View Source;boosting-safe-effective-and-cost-efficient-breast-cancer-treatment-for-patients-301027053.html [SID1234555721]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This new round of funding provides a pathway for Concure to double its field sales organization and focus on training new clinicians across the country in making this treatment option available to women. It follows a year in which Concure added a nationally-recognized team of clinical experts in breast cancer treatment to its team. Concure is using its expertise to improve the next-generation device that is at the heart of its Breast Microseed Treatment.

"This new financing marks an exciting inflection point for the team at Concure Oncology as well as the patients that we treat, now and moving forward," said Kevin Kelley, Chief Financial Officer of Concure Oncology. "Combined with the team of clinical experts that we’ve brought on in the last year and recent major shifts in treatment and therapy, women dealing with breast cancer now should have a much more optimistic path forward."

Breast Microseed Treatment has been proven safe, effective and convenient in providing radiation therapy to patients with early-stage breast cancer. This technique, also known as low-dose-rate brachytherapy, is based on a similar method that has already been proven to successfully treat prostate cancer. What distinguishes this treatment from conventional radiation therapy is that it allows post-lumpectomy patients the chance to have a one-time, one-hour procedure, as opposed to the more burdensome conventional radiation that may last from three to six weeks and require daily visits to a radiation facility.

This form of treatment has been recognized as being as effective as traditional whole breast irradiation relative to local recurrence and overall survival rates, with excellent cosmetic outcomes as well as reported high patient satisfaction levels in peer-reviewed, published data. The procedure involves the placement of tiny, low dose-rate brachytherapy sources – or Microseeds – into the breast tissue, which release a safe but effective dose of radiation for 2-3 months to the immediate area surrounding the lumpectomy site. Once fully released, the seeds become inert, with no radioactivity remaining.

Anyone interested in clinical applications, sales or marketing roles at Concure should visit the company’s website for more information.

On March 19, 2020 Zai Lab Limited (NASDAQ: ZLAB), a China and U.S.-based innovative commercial stage biopharmaceutical company, reported financial results for the twelve months ended December 31, 2019 and corporate updates (Press release, Zai Laboratory, MAR 19, 2020, View Source [SID1234555708]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Coinciding with the celebration of our five-year anniversary, 2019 was a year of many important achievements for Zai Lab," said Dr. Samantha Du, Founder and Chief Executive Officer of Zai Lab. "Some key milestones achieved include the marketing approval obtained for ZEJULA in China and our submissions for regulatory approval in China for Optune and omadacyline in China. We also entered into licensing collaborations with Deciphera and Incyte, expanded our commercial platform in China, launched commercial activities for ZEJULA and Optune in Hong Kong and further enhanced our in-house R&D capabilities. We are proud of our global reputation as a biotech pioneer and a partner-of-choice in China, and we continued to deliver exceptional execution across global and local clinical development, regulatory affairs, business development and operational expansion. Despite the challenges caused by the coronavirus (COVID-19) in China and globally, we expect 2020 to be another pivotal year for us as we continue to advance our clinical pipeline and further demonstrate our commercial and discovery capabilities. The proceeds from our recent financing positions us well to execute our key strategic initiatives towards becoming a leading global biopharma company."

Key Product Highlights and Expected Milestones

Oncology

ZEJULA (niraparib)

ZEJULA is a highly potent and selective oral, once-daily small molecule poly (ADP-ribose) PARP 1/2 inhibitor.

In March 2020, Zai Lab announced that the China National Medical Products Administration (NMPA) has accepted its supplemental New Drug Application (sNDA) for ZEJULA for first-line maintenance treatment of patients with ovarian cancer.

Based on IQVIA data, ZEJULA was the leading PARP inhibitor with market share in Hong Kong of 71% by value for 2019. ZEJULA was also ranked amongst the top 5 oncology drugs launches in Hong Kong after 2014 behind Keytruda, Opdivo, Tecentriq and Tagrisso based on first full-year revenue.

In February 2020, Zai Lab dosed the first patient in the Phase 1b study of niraparib with MGD013, a first-in-class PD1/LAG-3 bispecific antibody, in advanced or metastatic gastric cancer.

In February 2020, Zai Lab terminated the Phase 3 study of niraparib as first-line maintenance treatment of patients with small-cell lung cancer due to changes in the treatment landscape in China.

In January 2020, Zai Lab launched ZEJULA in China.

In December 2019, Zai Lab announced that the China NMPA approved the NDA for ZEJULA as maintenance treatment of patients with recurrent ovarian cancer. This approval represents the fastest approval time by the NMPA in 2019 for a locally manufactured, category 1 oncology drug.

In November 2019, Zai Lab completed enrollment of the China Phase 3 PRIME study of ZEJULA in patients with first-line ovarian cancer.

Expected Milestones

Initiate registrational bridging trial for late-line ovarian cancer treatment in second half of 2020

Expect final clinical data from the NORA study, the pivotal trial for maintenance therapy of Chinese patients with recurrent ovarian cancer in first half of 2020

Continue to collaborate with our Partner GSK on additional indications and opportunities

Tumor Treating Fields

Tumor Treating Fields is a cancer therapy that uses electric fields tuned to specific frequencies to disrupt cell division, inhibiting tumor growth and potentially causing cancer cells to die.

In January 2020, Zai Lab announced that the first patient has been enrolled in a Phase 2 pilot clinical trial evaluating the safety and efficacy of Tumor Treating Fields in combination with chemotherapy as first-line treatment in patients with gastric adenocarcinoma.

In September 2019, the NMPA accepted the Marketing Authorization Application (MAA) of Optune, a Tumor Treating Fields delivery system for the treatment of glioblastoma (GBM).

Expected Milestones

Potential China GBM MAA approval with trial waiver in first half of 2020

Complete enrollment of the Phase 2 pilot clinical trial in first-line gastric adenocarcinoma in 2020

File MAA for malignant pleural mesothelioma in China

Partner Milestone: Novocure to announce interim results from Phase 3 pivotal LUNAR trial in patients with non-small cell lung cancer in second half of 2020

Ripretinib

Ripretinib is an investigational KIT and PDGFRα kinase switch control inhibitor in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors (GIST), systemic mastocytosis, and other cancers.

In November 2019, Zai Lab received the Clinical Trial Authorization (CTA) approval for the registrational bridging study of ripretinib in patients with advanced GIST. The previously announced positive top-line data from the pivotal Phase 3 INVICTUS clinical study of ripretinib in patients with advanced GIST will support the NDA package of ripretinib in China.

Expected Milestones

Submit NDA to the China NMPA for advanced GIST in 2020

Initiate bridging trial for second-line GIST in second half of 2020

Margetuximab

Margetuximab is an Fc-optimized monoclonal antibody that targets the human epidermal growth factor receptor 2 (HER2).

In February 2020, Zai Lab announced that the first patient has been dosed in the registrational bridging study of margetuximab, in combination with chemotherapy, for the treatment of patients with metastatic HER2-positive breast cancer.

In February 2020, Zai Lab received CTA approval for the Phase 2/3 MAHOGANY study, a clinical trial of margetuximab in combination with a checkpoint inhibitor, with or without chemotherapy, as first-line treatment for patients with advanced HER2-positive gastric cancer and gastroesophageal junction (GEJ) cancer.

Expected Milestones

Enroll first Chinese patient in second half of 2020 in the global Phase 2/3 MAHOGANY initiated by MacroGenics

Partner Milestone: MacroGenics expects final overall survival (OS) data from the Phase 3 SOPHIA study in HER2+ metastatic breast cancer by year end of 2020. MacroGenics also anticipates a Prescription Drug User Fee Act (PDUFA) date by the end of 2020

Infectious Disease

NUZYRA (Omadacycline)

NUZYRA is a once-daily oral and intravenous antibiotic for the treatment of adults with community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI).

In March 2020, Zai Lab entered into a contract sales agreement with Hanhui, a leading local pharmaceutical company with a strong commercial presence in antibiotics. The agreement allows us to leverage Hanhui’s existing infrastructure to optimize an anticipated future commercial launch of omadacycline in China given that omadacycline is a broad spectrum antibiotic in both the hospital and community setting.

In February 2020, Zai Lab announced that the NMPA has accepted its NDA with Category 1 new drug designation for NUZYRA for the treatment of CABP and ABSSSI.

In February 2020, Zai Lab submitted a request for Priority Review to the NMPA for patients developing CABP in the context of coronavirus infection.

Durlobactam (ETX2514)

Durlobactam is a beta-lactamase inhibitor which – in combination with sulbactam – provides unique activity against Acinetobacter organisms, including carbapenem-resistant strains (CRAB).

In March 2020, Zai Lab’s partner Entasis Therapeutics announced it expects top-line results of the global Phase 3 ATTACK (Acinetobacter Treatment Trial Against Colistin) registrational trial in early 2021.

Expected Milestones

Enroll first Chinese patient into the global Phase 3 ATTACK trial in first half of 2020

Other Upcoming Milestones

MGD013 – a first-in-class, bispecific PD-1 x LAG-3 DART molecule

Enroll first Chinese patient to the global Phase 1 basket trial in second half of 2020

INCMGA0012 – an anti-PD-1 monoclonal antibody

Initiate pivotal study in second-line MSI-high endometrial cancer in China in second half of 2020

Enroll first Chinese patient into the Incyte-sponsored global Phase 3 study of INCMGA0012 with platinum-based chemotherapy in first-line metastatic squamous and non-squamous non–small cell lung cancer in China in second half of 2020

FPA144 – a first-in-class antibody for tumors that overexpress FGFR2b

Zai Lab’s partner Five Prime Therapeutics expects futility results from the Phase 3 FIGHT trial in gastric and GEJ cancer in mid-2020

Discovery

Submit two global Investigational New Drug (IND) applications in 2020

Research facilities located in Menlo Park, California scheduled to open in mid-2020

Business Development

Continue to pursue bolt-on and transformational business development opportunities

Corporate Highlights

In January 2020, Zai Lab announced the closing of an underwritten public offering of 5,500,000 American depositary shares ("ADSs") at a price of $47.50 per ADS. In addition, the underwriters fully exercised their option to purchase an additional 500,000 ADSs at the public offering price. Total proceeds, net of underwriting fees and other offering expenses, were $281.3 million.

In January 2020, Zai Lab announced the appointment of Lonnie Moulder, formerly chief executive officer of TESARO, to its Board of Directors.

In December 2019, Zai Lab announced the appointment of Timothy Yap, M.D., Ph.D., to its Scientific Advisory Board.

Zai Lab continues to expand its U.S. presence to enhance internal drug discovery and business development, with the securing of a lease for a larger facility in San Francisco and the opening of a Boston office.

Zai Lab continues to expand and hire talented professionals. As of February 2020, Zai Lab employed 733 full-time employees, with 293 and 340 employees engaged in R&D and commercial activities, respectively.

Full-Year 2019 Financial Results

Revenues for the full year of 2019 were $13.0 million, compared to $0.1 million in 2018. Revenues for the period were comprised of $6.6 million in sales of ZEJULA in Hong Kong and Macau, and $6.4 million of in sales of Optune in Hong Kong.

R&D expenses were $142.2 million for 2019, compared to $120.3 million for 2018. The increase in R&D expenses was primarily attributable to ongoing and newly initiated late-stage clinical trials, payroll and payroll-related expenses from increased R&D headcount and expansion of research efforts to support internal development programs.

Selling, General & Administrative expenses were $70.2 million for 2019, compared to $21.6 million for 2018. The increase was primarily due to payroll and payroll-related expenses from increased commercial headcount and related costs as Zai Lab expanded its commercial operations in China.

For the full year of 2019, Zai Lab reported a net loss of $195.1 million, or net loss per share attributable to common stockholders of $3.03, compared to a net loss of $139.1 million, or net loss per share attributable to common stockholders of $2.64, for the full year of 2018.

As of December 31, 2019, cash and cash equivalents, short-term investment and restricted cash totaled $276.4 million. In addition, in January 2020, Zai Lab announced the closing of an underwritten public offering with total proceeds, net of underwriting fees and other offering expenses, of approximately $281.3 million.

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast today, March 19, 2020 at 8:30 a.m. EDT to review its financial results and provide a general business update.

The live webcast and a replay may be accessed by visiting the Company’s website at View Source Alternatively, please call (866) 5194004 (U.S.), +65 67135090 (International), 4006208038 (China), +852 30186771 (Hong Kong), or +44 2036214779 (United Kingdom) to listen to the live conference call. The conference ID number for the live call is 8093543. Telephone replay will be available shortly after the call. To access the replay, please call (855) 4525696 (U.S.). The conference ID number for the replay is 8093543.