On March 3, 2020 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its full year 2019 financial results and provided an update on the company, including its progress with IPI-549, a first-in-class, oral immuno-oncology product candidate targeting immune-suppressive tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition (Press release, Infinity Pharmaceuticals, MAR 3, 2020, View Source [SID1234555120]).

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"2019 was a year of great execution at Infinity with tremendous progress in building a data-driven clinical program designed to clearly elucidate the activity of IPI-549 across a range of indications and innovative combination treatment regimens," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "This progress in 2019 included the initiation of Phase 2 MARIO-275 and MARIO-3 studies as well as the clinical collaboration with Arcus Biosciences. These new studies were informed by MARIO-1, our Phase 1 study that provided important early clinical and translational data demonstrating anti-cancer activity of IPI-549, on target reduction of myeloid derived suppressor cells, or MDSCs, and an excellent tolerability profile. These data laid the foundation for our upcoming milestone rich 2020 which will include enrollment completion for MARIO-275 in bladder cancer, and enrollment completion and initial data from MARIO-3 in front-line renal cell carcinoma and triple negative breast cancer. Importantly, our cash runway funds our trials through key data readouts, encompassing over 500 patients. We look forward to continued momentum as we establish MDSC and tumor macrophage-directed immunotherapies as important new options for patients currently unresponsive or refractory to available treatments."

Key 2019 Accomplishments and IPI-549 Program Guidance:

Financial:

In January 2020, completed a $20 million non-dilutive asset-backed financing with BVF Partners L.P. with sole recourse in potential royalty payments due on future sales of patidegib, a hedgehog pathway inhibitor discovered by Infinity and licensed to PellePharm in 2013. In addition, Infinity is eligible to receive from BVF an additional $5 million payment upon positive data from PellePharm’s Phase 3 trial in patients with Gorlin Syndrome. PellePharm completed enrollment of its Phase 3 trial in 2019.
Completed a royalty monetization with HealthCare Royalty Partners (HCR) for the right to receive certain royalty payments based on worldwide annual net sales of COPIKTRA (duvelisib), payable by Verastem. Under the agreement, HCR paid Infinity a $30 million upfront payment.
Extended cash runway with approximately $62 million cash at January 8, 2020 to fund all current IPI-549 trials to key data readouts in over 500 patients throughout 2020 and into mid-2021.
Clinical:

Initiated MARIO-275, the company’s ongoing global, randomized, controlled Phase 2 study in collaboration with Bristol-Myers Squibb, to evaluate IPI-549 in combination with Opdivo in platinum-refractory, I/O naïve patients with advanced urothelial cancer. Completion of enrollment is expected in 2020 with data mid-2021.
Initiated MARIO-3, the company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate IPI-549 in novel triple combination front-line therapies with Tecentriq and Abraxane in triple negative breast cancer (TNBC) and with Tecentriq and Avastin in renal cell cancer (RCC). Enrollment completion and initial data are expected in 2020.
Initiated a Phase 1b collaboration study with Arcus Biosciences, which is being conducted by Arcus, evaluating a checkpoint-inhibitor free, novel triple-combination regimen of IPI-549 + AB928 (dual adenosine receptor antagonist) + Doxil in advanced TNBC patients. Enrollment in the expansion cohort of up to 40 patients is ongoing.
Completed enrollment in MARIO-1, the company’s ongoing Phase 1/1b study of IPI-549 as a monotherapy and in combination with Opdivo in patients with advanced solid tumors. Additional data are expected in 2020.
Corporate:

Established Clinical Advisory Board with the following initial members:
Chair: Sam Agresta, MD, Infinity Board Member and Chief Medical Officer of Foghorn Therapeutics. Previously Dr. Agresta was responsible for the development and approval of several important drugs including of TIBSOVO and IDHIFA for patients with IDH1 and IDH2 mutation positive AML while at Agios and, while at Genentech, he played a key role in the global development of KADCYLA, which is approved for HER2 positive breast cancer. Dr. Agresta also played a key role in designing the Infinity trials now underway and is actively engaged with the company in advancing these trials.
Padmanee (Pam) Sharma, MD, PhD, co-leader of the MD Anderson Cancer Center’s immunotherapy platform and T.C. and Jeanette Hsu Endowed Chair in Cell Biology, Department of Genitourinary Medical Oncology. Dr. Sharma was the principal investigator on the BMS Checkmate 275 study that first demonstrated the association of high MDSCs to significantly lower overall survival in patients with UC and which provided the inspiration for our MARIO-275 study to which Dr Sharma will bring unique insight.
Toni Choueiri, MD, Director of the Lank Center for Genitourinary Oncology at Dana-Farber Cancer Institute/Brigham and Women’s Hospital and the Jerome and Nancy Kohlberg Chair and Professor of Medicine at Harvard Medical School and a recognized thought leader in the GU field who has done extensive work in the development of better treatments for patients with RCC and will bring great insight to the interpretation of our data from MARIO-3 in RCC and the development of next steps.
Michael Postow, MD, Co-Lead Melanoma Disease Management Team at Memorial Sloan Kettering Cancer Center and assistant professor of medicine at Weill Cornell Medical College. Dr. Postow treats patients with advanced melanoma and was the principal investigator of a clinical trial which led to FDA approval of the nivolumab + ipilimumab immunotherapy combination to treat melanoma. He is currently leading clinical trials testing new immunotherapy combinations in patients with advanced melanoma and will bring tremendous insight to potential future paths for IPI-549 in melanoma.
Jeff Kutok, MD, PhD, will be stepping down as Chief Scientific Officer at the end of March to lead drug discovery at another life science company and will assume the role as Chair of Infinity’s Scientific Advisory Board.
Seth Tasker, J.D. was promoted from General Counsel to Chief Business Officer.
Full Year 2019 Financial Results:

At December 31, 2019, Infinity had total cash, cash equivalents and available-for-sale securities of $42.4 million, compared to $58.6 million at December 31, 2018 and approximately $62 million at January 8, 2020.
Revenue during 2019 was $3.0 million, which primarily relates to the achievement of a $2.0 million milestone from PellePharm. Revenue during 2018 was $22.1 million, related to the amount received from Verastem for the approval by the FDA of COPIKTRA.
Research and development expense for 2019 was $27.1 million, compared to $19.8 million in 2018. The increase in R&D expense in 2019 compared to 2018 was primarily due to higher clinical development expenses for IPI-549.
General and administrative expense was $14.3 million for 2019, compared to $14.2 million for 2018.
Royalty expense for 2019 was $7.3 million, which primarily reflects Takeda’s share of the $30 million gross proceeds received from HCR for the monetization of COPIKTRA royalties in 2019, which was recognized as a liability in accordance with relevant accounting guidance. While recognized as a liability, the company is not obligated to repay the $30 million from HCR.
Net loss for 2019 was $47.1 million, or a basic and diluted loss per common share of $0.83, compared to a net loss of $11.3 million, or a basic and diluted loss per common share of $0.20 for 2018. The increase in net loss was mostly driven by the $22 million milestone received from Verastem in 2018 for the approval of COPIKTRA and the $7.3 million royalty expense to Takeda in 2019 for their share of the COPIKTRA royalty monetization with HCR.
Financial Outlook:

Infinity’s 2020 financial guidance remains unchanged:

Net Loss: Infinity expects net loss for 2020 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2020 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $15 million to $25 million.
Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities, including the $20.0 million received from BVF in January 2020, will be adequate to satisfy the company’s capital needs into 2H 2021. Infinity’s financial guidance does not include additional funding or business development activities or a potential $5 million milestone payment from BVF for positive patidegib Phase 3 data and any milestones from, or the sale of the company’s equity interest in, PellePharm.
Conference Call Information

Infinity will host a conference call today, March 3, 2020, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial (877) 316-5293 (domestic) and (631) 291-4526 (international) five minutes prior to start time. The conference ID number is 6177179. An archived version of the webcast will be available on Infinity’s website for 30 days.

About Infinity and IPI-549

Infinity is an innovative biopharmaceutical company dedicated to advancing novel medicines for people with cancer. Infinity is advancing IPI-549, a first-in-class, oral immuno-oncology development candidate that selectively inhibits PI3K-gamma, in multiple clinical studies. MARIO-1 is an ongoing Phase 1/1b study evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab) in approximately 225 patients with advanced solid tumors including patients refractory to anti-PD-1 therapy. MARIO-275 and MARIO-3 have recently initiated. MARIO-275 is a global, randomized, combination study of IPI-549 combined with Opdivo in I/O naïve urothelial cancer patients. MARIO-3 is the first IPI-549 combination study in front-line advanced cancer patients and is evaluating IPI-549 in combination with Tecentriq and Abraxane in front-line TNBC and in combination with Tecentriq and Avastin in front-line RCC. With the addition of MARIO-275 and MARIO-3 to the ongoing MARIO-1 study, Infinity will be evaluating IPI-549 in the anti-PD-1 refractory, I/O-naïve and front-line settings. For more information on Infinity, please refer to Infinity’s website at www.infi.com.

On March 3, 2020 Immunomic Therapeutics, Inc., a privately held clinical stage biotechnology company pioneering the study of nucleic acid immunotherapy platforms, reported that it successfully completed a pre-IND (Investigational New Drug) meeting with the U.S. Food and Drug Administration (FDA) regarding its First-In-Human (FIH) Phase I trial and development plans for ITI-1001 (Press release, Immunomic Therapeutics, MAR 3, 2020, View Source [SID1234555119]). The FDA addressed the Company’s questions and provided feedback on key components of the planned IND application for the candidate, ITI-1001, for the treatment of newly diagnosed Glioblastoma Multiforme (GBM).

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ITI-1001 is an investigational plasmid DNA vaccine therapy that leverages Immunomic’s proprietary UNITE platform to treat patients with newly diagnosed GBM. ITI-1001 is designed to target the pp65, IE-1, and gB viral antigens of Cytomegalovirus (CMV), expressed in GBM, but not in normal brain cells.

"We are pleased to have completed our pre-IND meeting with the FDA, which marks an important development milestone for Immunomic and the ITI-1001 program," said Dr. William Hearl, CEO of Immunomic Therapeutics. "We appreciate the guidance from the FDA as we move forward with our plans to initiate a Phase 1 trial for GBM in the US, which we hope will bring us closer to providing a therapeutic option to patients with this devastating disease."

In the planned First-In-Human (FIH) Phase I trial, Immunomic will evaluate the safety, tolerability, immunogenicity and preliminary efficacy of ITI-1001 in patients with Newly-Diagnosed Glioblastoma (GBM) Having Unmethylated MGMT Promoter Mutations. The Phase 1 trial is anticipated to start in 2021.

About UNITE

ITI’s investigational UNITE platform, or UNiversal Intracellular Targeted Expression, works by fusing pathogenic antigens with the Lysosomal Associated Membrane Protein, an endogenous protein in humans, for immune processing. In this way, ITI’s vaccines (DNA or RNA) have the potential to utilize the body’s natural biochemistry to develop a broad immune response including antibody production, cytokine release and critical immunological memory. This approach could put UNITE technology at the crossroads of immunotherapies in a number of illnesses, including cancer, allergy and infectious diseases. UNITE is currently being employed in Phase II clinical trials as a cancer immunotherapy. ITI is also collaborating with academic centers and biotechnology companies to study the use of UNITE in cancer types of high mortality, including cases where there are limited treatment options like glioblastoma and acute myeloid leukemia. ITI believes that these early clinical studies may provide a proof of concept for UNITE therapy in cancer, and if successful, set the stage for future studies, including combinations in these tumor types and others. Preclinical data is currently being developed to explore whether LAMP nucleic acid constructs may amplify and activate the immune response in highly immunogenic tumor types and be used to create immune responses to tumor types that otherwise do not provoke an immune response.

On March 3, 2020 Hutchison China MediTech Limited ("Chi-Med") (Nasdaq/AIM: HCM), a commercial-stage biopharmaceutical company with eight oncology drug candidates in development around the world and a deep commercial presence in China, reported its audited financial results for the year ended December 31, 2019 and provides updates on key clinical and commercial developments (Press release, Hutchison China MediTech, MAR 3, 2020, View Source [SID1234555118]).

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"2019 was a year in which we laid the foundations for a new era for Chi-Med," said Simon To, Chairman of Chi-Med. "Our first launched drug, Elunate, is set to broaden patient access this year due to its recent addition to the NRDL1 in China. We are scaling up our oncology commercial team in preparation for the potential launch of surufatinib, our first un-partnered oncology drug candidate, late this year in non-pancreatic NET2; and another two NDA3 submissions are imminent, one with savolitinib in lung cancer and a second with surufatinib in pancreatic NET, with launches anticipated for 2021."

"Based on extensive clinical data, we also expect to initiate multiple global registration studies this year with fruquintinib, surufatinib and potentially savolitinib. China registration studies with certain of our hematological malignancy assets are also in planning."

"We believe that the potential launches of multiple new oncology products will address a broad range of unmet medical needs and benefit a large number of patients, propelling Chi-Med rapidly forward."

RECENT OPERATING HIGHLIGHTS

Set out below are some of Chi-Med’s operating highlights for 2019 and so far this year. For more details, please refer to "Operations Review" below.

SAVOLITINIB — GLOBAL

· AstraZeneca4 collaboration — Prime position in EGFR TKI5 resistant NSCLC6:

· EGFRm7 NSCLC patients with acquired resistance to Tagrisso driven by MET8 amplification: Published full results of TATTON study in The Lancet Oncology in 2020 for the savolitinib/Tagrisso combination reporting 30% ORR9 and 5.4 months’ median PFS10 in 69 patients;

The SAVANNAH Phase II study, with registration potential, underway in North and South America, Europe and Asia, is on-target for interim analysis in mid-2020 and enrollment completion by end-2020;

·EGFRm NSCLC patients with acquired resistance to Iressa or Tarceva driven by MET amplification: Published full results of TATTON study in Lancet Oncology for the savolitinib/Tagrisso combination reporting 64% ORR and 9.0 months’ median PFS in 93 patients; and

Completed enrollment in 70 patients Phase II registration study — MET Exon 14 deletion NSCLC: Interim China Phase II data presented at CSCO11. As a result of our regulatory interaction with the NMPA12, we now expect to submit savolitinib NDA in early 2020.

· Papillary renal cell carcinoma ("PRCC") — Renewed global development strategy:

· Actively evaluating restart in MET-driven PRCC: In late 2018, enrollment was terminated in SAVOIR, a global Phase III registration study of savolitinib monotherapy compared with sunitinib monotherapy in MET-positive PRCC. Data from the approximately 60 patients randomized in SAVOIR prior to termination has matured during 2019 and will be presented at an upcoming scientific conference in mid-2020. Based on these data, AstraZeneca and Chi-Med are actively evaluating the opportunity to restart clinical work in PRCC for monotherapy savolitinib; and

· Preliminary signal for savolitinib/Imfinzi (PD-L113) combination in all PRCC: Presented data for the PRCC cohort of the CALYPSO Phase II study at ASCO (Free ASCO Whitepaper) GU14 showing the combination was tolerable and associated with durable efficacy. Median OS15 was 12.3 months and twelve-month OS rate was 52%. Based on these data, AstraZeneca and Chi-Med continue to explore development of the savolitinib and Imfinzi combination.

·Promising savolitinib efficacy in MET-amplified gastric cancer: The VIKTORY Phase II umbrella trial results were published in Cancer Discovery16. VIKTORY sequenced 715 metastatic gastric cancer patients, with MET-amplification observed in 3.5% of patients. In MET-amplified gastric cancer patients, savolitinib monotherapy met pre-specified 6-week PFS rate and reported an ORR of 50%.

SURUFATINIB — CHINA

·First targeted therapy to address NETs of all origins: Recently reported two positive Phase III studies, SANET-ep (mid-2019) in non-pancreatic NET and SANET-p (early 2020) in pancreatic NET. Both studies were terminated early following positive interim analyses that confirmed they had already met their median PFS primary endpoint:

· China Non-pancreatic NET: Presented full results of SANET-ep Phase III study at ESMO (Free ESMO Whitepaper)17 reporting a median PFS for surufatinib of 9.2 months as compared to 3.8 months for placebo (HR 0.334, p<0.0001). An NDA for the treatment of non-pancreatic NETs was submitted to the NMPA in China in November 2019 and Priority Review status was granted in December 2019; and

· China Pancreatic NET: Following positive interim analysis and early termination of the SANET-p Phase III study, NDA preparations are now underway.

·Initiated China Phase II/III study in biliary tract cancer ("BTC"): Based on preliminary Phase Ib/IIa data, we initiated a Phase IIb/III registration study in BTC in China in March 2019; and

·Progressed PD-118 combination development: Completed a Phase I dose-finding study in China of surufatinib plus Tuoyi, an approved PD-1 monoclonal antibody from Junshi19, then initiated an exploratory Phase II study of the combination in early 2020 in multiple solid tumor indications. Phase I development of surufatinib plus Tyvyt, an approved PD-1 monoclonal antibody from Innovent20, is also in planning.

FRUQUINTINIB — CHINA

·Progress on Elunate (fruquintinib capsules) in third-line colorectal cancer ("CRC") in China:

· 17.6 million in sales during 2019: In-market sales of Elunate to third-parties, as provided by Lilly21, in the first full year since its late 2018 launch; and

·Inclusion in the National Reimbursement Drug List ("NRDL"): Elunate was included in the China NRDL in November 2019, with reimbursement effective January 1, 2020. NRDL inclusion now makes Elunate a highly attractive approved therapy in third-line CRC in China in terms of price, efficacy and safety profile. Elunate sales22 in January-February 2020, were $6.6 million.

· Phase III interim analysis in second-line gastric cancer: In April 2019, an interim analysis for futility of the FRUTIGA study in China was performed. The IDMC23 recommended to continue the study without changes; and

·Progressed PD-1 combination development: Approaching completion of Phase I dose-finding study in China of Elunate plus Tyvyt (Innovent). Phase I development of Elunate plus genolimzumab, a PD-1 monoclonal antibody under development by Genor24, is also now underway.

OTHER DEVELOPMENT CANDIDATES — CHINA

· Non-Hodgkin’s lymphoma ("NHL"): Advanced Phase Ib dose expansion of both of our NHL assets, HMPL-523 (selective Syk25 inhibitor) and HMPL-689 (selective PI3Kδ26 inhibitor) in China. We expect these Phase I/Ib studies to inform our China registration study decisions in 2020;

· HMPL-453 — selective FGFR27 1/2/3 inhibitor: We completed Phase I development, with a Phase II study in advanced malignant mesothelioma in China set to initiate; an

IND28 clearance in China for HMPL-306: Our ninth in-house discovered asset, an IDH29 1/2 dual inhibitor, received China IND clearance in late 2019 with Phase I set to initiate.

INTERNATIONAL OPERATIONS

· Global development footprint: Through our International organization, based in New Jersey, we have rapidly expanded our clinical and regulatory capabilities in the U.S., Europe and now Japan;

· Fruquintinib: Completed EOP230 meetings with U.S. Food and Drug Administration ("FDA") in February 2020, regarding our global Phase III, the FRESCO2 study, in colorectal cancer. Europe and Japan EOP2 meetings are planned shortly;

·Surufatinib: Data from a U.S. Phase I/Ib study were presented at ESMO (Free ESMO Whitepaper). In late 2019, the U.S. FDA granted Orphan Drug designation for the treatment of pancreatic NET. Regulatory consultations in U.S., Europe and Japan are underway, to clarify registration pathway for surufatinib in NETs; and

·HMPL-523 and HMPL-689: Expanded development into the U.S. and Europe during 2019. Twenty Phase I sites are now enrolling and have completed multiple dose cohorts.

ORGANIZATION

·Chi-Med Group compensation and share-based incentive policy: The Group has comprehensively reviewed its compensation and share-based incentives policies, performed benchmarking research on peer group U.S. and China biotech companies and established a new competitive policy to ensure we are able to attract and retain top talent; and

· Establishment of China oncology commercial organization: Currently over 140 commercial staff, aiming to recruit a total of 300-350 staff to support potential surufatinib launch in late 2020.

UPDATE ON IMPACT OF COVID-19

·Improvising amid Covid-19 challenges: The outbreak is posing some challenges to our operations resulting from restrictions on movement in China. Reduced patient hospital visits for clinical assessment affected the conduct of certain clinical studies and commercial team activities. To-date, none of our manufacturing operations in China have been materially affected. Our teams have adapted quickly and effectively thus far across our businesses, and we will continue to closely monitor what is an evolving situation. At this stage we are unable to assess the long-term effect of the outbreak, if any.

KEY EVENTS PLANNED FOR 2020

Early 2020:

· Savolitinib — Phase Ib/II data (CALYPSO) — PRCC cohort overall survival results for the Imfinzi / savolitinib combination presented at ASCO (Free ASCO Whitepaper) GU (February 2020);

· HMPL-453 — Phase II study start — FGFR 1/2/3 inhibitor in advanced malignant mesothelioma;

·Savolitinib — NDA submission in MET exon 14 deletion NSCLC in China — first NDA submission globally for savolitinib;

· Surufatinib — NDA submission in pancreatic NET in China — following the recent positive SANET-p Phase III interim analysis;

·PD-1 combos — Initiation of multiple Phase II studies in China — for surufatinib/fruquintinib in combination with Tuoyi/Tyvyt; and

· PD-1 combos — Phase I dose-finding data for surufatinib plus Tuoyi combination — presentation of preliminary data at major scientific conference.

Mid-2020:

HMPL-306 — First in Human dose of IDH 1/2 inhibitor — initiate Phase I study in China;

·Savolitinib — Data from terminated Phase III study (SAVOIR) — presentation at major scientific conference of data comparing savolitinib to sunitinib in MET-driven PRCC patients; Mature data from about 60 patients;

Savolitinib — Interim analysis on SAVANNAH — interim analysis on first ~50 patients on SAVANNAH Phase II study of the savolitinib/Tagrisso combination;

Surufatinib — Completion of global regulatory consultations — clarity on U.S., Europe and Japan registration pathway for surufatinib in NETs. Initiation of required clinical studies in U.S. and Europe;

Savolitinib — MET exon 14 deletion NSCLC data — presentation of full data from the savolitinib Phase II registration intent study at major scientific conference;

Surufatinib — Phase III data (SANET-p) — presentation of full data from the SANET-p study in pancreatic-NET patients at a major scientific conference;

Fruquintinib — Second Phase III interim analysis (FRUTIGA) — interim analysis for futility in second-line gastric cancer Phase III in China of fruquintinib / Taxol (paclitaxel) combination;

Fruquintinib — Global Phase III study (FRESCO2) — initiation of registration study of fruquintinib in >3rd line colorectal cancer in U.S., Europe and Japan; and

HMPL-523 — Global Phase Ib expansion — in indolent NHL in U.S. and Europe.

Late 2020:

Surufatinib — Phase II/III interim analysis — for futility in second-line BTC in China;

· Surufatinib — Potential NDA approval and launch for non-pancreatic NET in China — first un-partnered oncology drug launch for Chi-Med in China. Commercial team of 300-350 medical sales personnel in place for launch;

· HMPL-689 — Potential registration study start — in indolent NHL in China;

Fruquintinib — Enrollment completion of FRUTIGA — to complete enrollment of China Phase III registration study in second-line gastric cancer;

Savolitinib — Enrollment completion of SAVANNAH — AstraZeneca to complete enrollment of global Phase II study, with registration potential, of savolitinib/Tagrisso combination; and

HMPL-689 — Global Phase Ib expansion — in indolent NHL in U.S. and Europe.

FINANCIAL HIGHLIGHTS

The items below are selected financial data for the year ended December 31, 2019. All dollars are expressed in US dollar currency unless otherwise stated. For more details, please refer to "Financial Review", "Operations Review" and "Audited Consolidated Financial Statements" below.

OVERALL GROUP:

Group revenue of $204.9 million (2018: $214.1m).

Net loss attributable to Chi-Med of $106.0 million (2018: net loss of $74.8m).

Adjusted Group net cash flows excluding financing activities was -$82.3 million (2018: -$49.1m). Cash from our Commercial Platform, as well as cash received from our multi-national partners, continued to offset a material portion of our R&D31 expenses.

Recent Nasdaq follow-on strengthens cash position. We held cash, cash equivalents and short-term investments of $217.2 million as of December 31, 2019 (December 31, 2018: $301.0m). In January 2020, we conducted a Nasdaq follow-on offering, raising an additional $110.1 million in net proceeds, to further strengthen our cash position; and

Additional unutilized bank facilities of $119.3 million (December 31, 2018: $119.3m) and borrowings of $26.8 million (December 31, 2018: $26.7m).

INNOVATION PLATFORM:

Consolidated revenue was $16.0 million (2018: $37.6m) mainly from service fee payments from AstraZeneca and Lilly. 2018 revenues included a one-time $13.5 million milestone payment from Lilly following fruquintinib approval; and

Net loss from our Innovation Platform attributable to Chi-Med of $133.2 million (2018: net loss of $104.4m) resulting from expansion in the development of our eight clinical drug candidates, five of which are now in global development, and establishment of sizable international clinical and regulatory operations.

COMMERCIAL PLATFORM:

Total consolidated sales up 7% (11% at CER32) to $188.9 million (2018: $176.5m) mainly due to continued progress on our Prescription Drugs subsidiary Hutchison Sinopharm33 as well as manufacturing sales and royalties from Elunate during its first full year on the market;

Total consolidated net income from our Commercial Platform attributable to Chi-Med up 9% (13% at CER) to $47.4 million (2018: $43.4m) underpinned by the growing profits of our legacy operations in China as well as Elunate.

FINANCIAL GUIDANCE

In 2019 we performed in-line with our most recent guidance. We provide Financial Guidance for 2020 below.

In 2020, on the broader Innovation Platform, we plan to continue to increase our investment in R&D particularly on clinical development of our main assets in the U.S., Europe and Japan as well as in China (as discussed in the "Product pipeline progress" section below). On the Commercial Platform, we expect to continue to generate cash flow directly through our subsidiaries and via dividends from our joint ventures. We assume at this stage that the financial impact of the recent Covid-19 outbreak will not be material to the Group. Since we cannot predict how the situation will evolve, we will monitor and adjust if new material information emerges.

2020 Guidance

Adjusted (non-GAAP) Innovation Platform segment operating loss

$(180) — (210) million

Adjusted (non-GAAP) Group Net Cash Flows excluding financing activities

$(140) — (160) million

Use of Non-GAAP Financial Measures and Reconciliation — References in this announcement to adjusted Innovation Platform segment operating loss, adjusted Group net cash flows excluding financing activities and financial measures reported at CER are based on non-GAAP financial measures. Please see the "Use of Non-GAAP Financial Measures and Reconciliation" below for further information relevant to the interpretation of these financial measures and reconciliations of these financial measures to the most comparable GAAP measures, respectively.

Conference Call and Audio Webcast Presentation Scheduled Today at 1:00 p.m. GMT / 8:00 a.m. EST / 9:00 p.m. HKT — Investors may participate in the call as follows: +44 20 3936 2999 (U.K.) / 1 845 213 3398 (U.S.) / +852 5808 4954 (Hong Kong), or access a live audio webcast of the call via Chi-Med’s website at www.chi-med.com/investors/event-information/.

Additional dial-in numbers are also available at Chi-Med’s website. Please use participant access code "413486."

FINANCIAL STATEMENTS

Chi-Med will today file with the U.S. Securities and Exchange Commission its Annual Report on Form 20-F.

ANNUAL GENERAL MEETING

The Annual General Meeting of Chi-Med will be held at 4th Floor, Hutchison House, 5 Hester Road, Battersea, London SW11 4AN on Monday, April 27, 2020 at 11:00 a.m.

On March 3, 2020 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company developing potentially life-changing treatments for cancer and other serious diseases, reported it will be presenting at the third annual LD Micro Virtual Conference on Wednesday, March 4 at 1:40 p.m. EST (Press release, Genprex, MAR 3, 2020, View Source [SID1234555117]). Genprex’s Chairman and Chief Executive Officer, Rodney Varner, will lead the company’s presentation and will be answering questions from investors.

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To access the live presentation, please use the following link: http://bit.ly/2PFjomt.

"We are delighted to be hosting our third virtual event in order to showcase some of the truly unique names in micro-cap," stated Chris Lahiji, President of LD Micro. "There are many people and companies who are unable to attend our live events due to any number of reasons, so we are happy to offer an additional way for companies to present to investors without taking a lot of time out of their day-to-day operations. While virtual events will never replace the experience of sitting in the same room as other humans, it is a great format for updating the investor community and getting increased exposure."

The conference will be held via webcast and will feature more than 40 companies in the small/micro-cap space.

On March 3, 2020 Exicure, Inc. (NASDAQ:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported full year financial results for year ended December 31, 2019 and provided an update on corporate progress (Press release, Exicure, MAR 3, 2020, View Source [SID1234555116]).

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"2019 was a pivotal year for Exicure as we joined strong scientific and clinical progress with the capital resources and distinguished investors necessary to expand our pipeline and build our organization," said Dr. David Giljohann, Chief Executive Officer of Exicure. "Looking ahead, we intend to invest in growing a pipeline of drug candidates targeting neurological indications, as well as advance our Merkel cell and cutaneous squamous cell carcinoma programs into Phase 2 trials. We believe our ongoing clinical progress in immuno-oncology and dermatology has influenced our ability to expand our SNA platform in additional therapeutic areas through collaborative partnerships. Our collaboration with Allergan in hair loss disorders is just one of what we hope will be a number of such partnerships expanding our SNA platform into new therapeutic areas," concluded Dr. Giljohann.

Corporate Progress
Key achievements for Exicure during 2019 include:

Established Friedreich’s ataxia as Exicure’s first neurological indication, being developed in collaboration with the Friedreich’s Ataxia Research Alliance

Announced preliminary data in the ongoing Phase 1b/2 trial of lead immuno-oncology candidate AST-008 in patients with solid tumors

Entered a collaboration agreement with Allergan for two discovery programs in hair loss disorders with $25 million upfront and up to $725 million in potential milestones

Expanded scientific advisory board to include neurology experts Dr. Susan Perlman and Dr. Hank Paulson

Expanded Board of Directors with the addition of Jeffrey L. Cleland of Orpheris, Bali Muralidhar of Abingworth LLP, Bosun Hau of Tybourne Capital Management, and Tim Walbert of Horizon Therapeutics

Raised approximately $90.8 million in gross proceeds from the sale of common stock in two public offerings and up-listed to the NASDAQ Global Market

Pipeline Updates
Neurology

In December of 2019, Exicure announced the development of XCUR-FXN, an SNA-based therapeutic candidate, for the treatment of Friedreich’s ataxia (FA). FA is driven by triplet repeats in the frataxin gene which compromises the patient’s ability to generate adequate levels of frataxin protein. Exicure believes its SNA technology has the potential to address this genetic challenge and that its therapeutic strategy may lead to increases in the frataxin protein. Exicure will be developing XCUR-FXN with guidance from, and in collaboration with, the Friedreich’s Ataxia Research Alliance. Preclinical research is ongoing and IND-enabling studies for XCUR-FXN are expected in late 2020.

In 2020, Exicure expects to continue pre-clinical research on the application of its SNA technology in neurological conditions, building on its early proof-of-concept work with nusinersen and its new therapeutic candidate, XCUR-FXN. Exicure is currently exploring additional neurological conditions, including spinocerebellar ataxia, Batten disease, amyotrophic lateral sclerosis (ALS), and Huntington’s disease.

Immuno-oncology; AST-008

In December of 2019, Exicure announced preliminary results from the Phase 1b study of AST-008 in patients with solid tumors. AST-008 is an investigational SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications, and is being evaluated in combination with pembrolizumab in patients with solid tumors. At that time the study had enrolled fourteen patients including five melanoma patients, four Merkel cell carcinoma (MCC) patients, two cutaneous squamous cell carcinoma patients, two head and neck squamous cell carcinoma patients, and one mucosal melanoma patient. Prior to enrolling, most patients had progressive disease on anti-PD-1 or anti-PD-L1 antibodies.

Available data, as of December 11, 2019, showed that AST-008 administration, alone or in combination with pembrolizumab, produced cytokine and chemokine expression and immune cell activation in patient blood indicative of desired immune activation. Of the 4 MCC patients, one patient, who had previously progressed on anti-PD-1 antibody therapy, had confirmed stable disease with decreased target lesion diameters for a period in excess of twelve weeks, while a second MCC patient experienced a target lesion complete response and a confirmed overall partial response longer than 24 weeks. No treatment-related serious adverse events or dose-limiting toxicities have been observed. The most common reported adverse event was injection site reactions.

Exicure is now completing the Phase 1b study and preparing to begin a Phase 2 study in both Merkel cell carcinoma and in cutaneous squamous cell carcinoma. Exicure currently has seven trial sites open and seeks to expand to about fifteen sites.

Collaborations
Exicure entered into a collaboration with Allergan Pharmaceuticals International Limited in late 2019 and is now actively engaged in preclinical research and discovery in two clinical programs related to the treatment of hair loss disorders. Under the terms of the collaboration, Exicure received a $25 million upfront payment and is eligible to receive up to $725 million in potential milestones. In early 2019,

Exicure also entered into a collaboration agreement with Dermelix Biotherapeutics under which Dermelix will develop a targeted therapy for the treatment of Netherton Syndrome (NS).

2019 Financial Results and Financial Guidance
Cash Position: As of December 31, 2019, Exicure had cash and cash equivalents of $48.5 million and short-term investments of $62.3 million for a total of $110.8 million compared to $26.3 million of cash and cash equivalents and no short-term investments as of December 31, 2018. In 2019, Exicure raised approximately $90.8 million in gross proceeds from the sale of common stock and received a $25 million upfront payment in connection with the Collaboration Agreement with Allergan.

Research and Development Expense: Research and development expense was $19.3 million for the year ended December 31, 2019 compared to $14.1 million for the year ended December 31, 2018. The increase in research and development expense of $5.2 million was primarily due to higher platform and discovery-related expenses of $4.7 million, higher employee-related expense of $0.9 million and higher facilities, depreciation, and other expenses of $0.2 million, partially offset by a net decrease of $0.6 million in costs related to our clinical development programs. The increase in platform and discovery-related expenses is mostly due to a license fee of $3.8 million paid to Northwestern University in connection with the $25.0 million upfront payment received from Allergan.

General and Administrative Expense: General and administrative expense was $8.6 million for the year ended December 31, 2019 and $7.8 million for the year ended December 31, 2018, an increase of $0.8 million. This increase was primarily due to higher compensation and related expenses, recruiting fees in connection with adding two new board members, higher D&O insurance premiums, Nasdaq listing costs and lease costs associated with our Cambridge, MA office. These increased costs were partially offset by reductions in legal and other transaction costs.
Net Loss: Net loss was $26.3 million for the year ended December 31, 2019, compared to net loss of $22.4 million for the year ended December 31, 2018, an increase in loss of $3.9 million. Net loss reflects the changes in expenses discussed above and is offset by an increase in revenue of $1.2 million for the year ended December 31, 2019 compared to revenue for the year ended 2018. Revenue in 2019 was primarily related to the Dermelix Collaboration.
Cash Runway Guidance: Exicure believes that, based on its current operating plans and as of the date of this press release, its existing cash and cash equivalents as of December 31, 2019 is sufficient to meet its anticipated cash requirements into early 2022.

About Friedreich’s Ataxia (FA)
FA is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults, and involves the loss of strength and coordination usually leading to wheelchair use, diminished vision, hearing and speech, scoliosis, increased risk of diabetes, and a life-threatening heart condition. There are no FDA-approved treatments. An estimated 5,000 patients in the US and 15,000 patients worldwide are affected by FA.

About FARA
The Friedreich’s Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich’s ataxia. www.CureFA.org.