On January 31, 2020 G1 Therapeutics Presented the corporate presentation (Presentation, G1 Therapeutics, JAN 13, 2020, View Source [SID1234553063]).

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On January 13, 2020 Aduro Biotech presented the corporate presentation (Presentation, Aduro Biotech, JAN 13, 2020, View Source [SID1234553062]).

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On January 13, 2020 Emergent BioSolutions Inc. (NYSE: EBS) reported selected preliminary unaudited 2019 financial results and its financial forecast for 2020 (Press release, Emergent BioSolutions, JAN 13, 2020, View Source [SID1234553059]).

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Robert G. Kramer Sr., president and chief executive officer of Emergent BioSolutions Inc. stated, "Our preliminary results for 2019 reflect another strong year of execution on the part of the entire Emergent team. With total revenue of $1.1 billion, anticipated gross margin of 56% to 58%, which is a 200 to 400 basis point improvement over prior year, and margins on adjusted net income and adjusted EBITDA of 14% (1) and 26% (2), respectively, our financial performance reflects the consistency and durability of our business model and its focus on public health threat solutions. Moreover, we achieved our key operational goals for the year, which included securing over $3 billion of renewed contract value, expanding markets for our existing products and services portfolio, and continuing investments to advance our product pipeline and enhance our manufacturing infrastructure."

Continuing, Kramer stated, "As we enter 2020, we are planning for continued expansion of our business and a financial forecast that at the midpoint anticipates 11% year-over-year growth in total revenue along with strong absolute and relative gains in adjusted net income and adjusted EBITDA. We intend to achieve these outcomes while simultaneously expanding our portfolio of advanced stage product candidates that address serious global public health threats. As a result, we are confident in the strength of our business model as we pursue the sustained growth and value creation to be realized in the 2020-2024 growth strategy we announced at the Analyst & Investor Day we held in November, which contemplates annual revenue in excess of $2 billion by 2024."
PRELIMINARY 2019 FINANCIAL RESULTS (Unaudited)
The company is providing the following preliminary, unaudited financial results for full year 2019.

Total Revenue
For the full year 2019, the company anticipates total revenue of $1,100 to $1,105 million, the midpoint of which represents a $320 million or 41% increase from 2018, the second year in a row of approximately 40% year-over-year growth in total revenue. This annual increase is due primarily to the contribution of sales of NARCAN (naloxone HCl) Nasal Spray and ACAM2000 (Smallpox (Vaccinia) Vaccine, Live) in 2019 as well as higher contracts and grants revenue, offset by lower anthrax vaccine revenue (now a combination of BioThrax (Anthrax Vaccine Adsorbed) and AV7909 (Anthrax Vaccine Adsorbed, Adjuvanted) (4)).

Net Income (GAAP and Adjusted)
For the full year 2019, the company anticipates net income of $53 to $63 million, which reflects the impact of two significant items, contingent consideration related to the Adapt acquisition and a non-cash impairment. The company also anticipates adjusted net income of $150 to $160 million, the midpoint of which represents a $33 million or 27% increase from 2018, reflecting the impact of both increased product sales as well as higher average margins on the products contributing to product sales in 2019. (See "Reconciliation of Net Income to Adjusted Net Income, EBITDA and Adjusted EBITDA" for a definition of terms and reconciliation tables.)

Note:
The preliminary 2019 financial results are subject to revision and will be finalized upon completion of the company’s external audit, which is anticipated by late February 2020. The company’s final audited financial results could differ materially from these selected preliminary results.
2020 FINANCIAL FORECAST

For the full year of 2020, the company’s financial forecast includes the impact of the following items:

continued growth in sales of NARCAN Nasal Spray to a range of $285 — $315 million;

combined deliveries of AV7909 (4) and BioThrax to the Strategic National Stockpile (SNS) in a range of $270 — $300 million;

deliveries of ACAM2000 in a range of $180 — $200 million under procurement contracts with the U.S. government and other foreign governments;

deliveries of raxibacumab to the SNS under the anticipated follow-on procurement contract with the ASPR;

domestic and international sales of the other medical countermeasures that comprise Other Product sales;

continued expansion of CMO services revenue;

continued improvement of gross margin in a range of 200 — 400 basis points, driven by improved product mix; and

continued investment in discretionary development projects funded by the company, most notably the anticipated Phase 3 studies for both the Chikungunya and FLU-IGIV product candidates, among other R&D projects.

The outlook for 2020 does not include estimates for potential new corporate development or other M&A transactions.

PRESENTATION WEBCAST
The company will provide an update on the current business and discuss preliminary 2019 financial results, the forecast and corporate goals for 2020, and long-term goals for the five-year period 2020-2024 during its presentation at the 38th Annual J.P. Morgan Healthcare Conference on January 14, 2020 at 10:30 AM Pacific time.

A live webcast of the presentation can be accessed through Emergent’s website. Visit www.emergentbiosolutions.com and select the "Investors" section. An on-demand replay of the webcast can also be accessed in the investors section after the presentation has concluded.

FOOTNOTES

Adjusted Net Income margin is defined as Adjusted Net Income divided by total revenues. For the 2019 Preliminary Results, we reference this metric using the midpoints of the relevant factors. Specifically: $155/$1,103 = 14%.

Adjusted EBITDA margin is defined as Adjusted EBITDA divided by total revenues. For the 2019 Preliminary Results, we reference this metric using the midpoints of the relevant factors. Specifically: $285/$1,103 = 26%.

See "Reconciliation of Net Income to Adjusted Net Income, EBITDA and Adjusted EBITDA" for a definition of terms and a reconciliation tables.

AV7909 is a product candidate not yet approved by the FDA or any other health regulatory agency but procured by the U.S. government under special circumstances.

RECONCILIATION OF NET INCOME TO ADJUSTED NET INCOME, EBITDA AND ADJUSTED EBITDA
This press release contains five financial measures (Adjusted Net Income, Adjusted Net Income margin, EBITDA (Earnings Before Interest, Taxes, Depreciation and Amortization), Adjusted EBITDA and Adjusted EBITDA margin) that are considered "non-GAAP" financial measures under applicable Securities and Exchange Commission rules and regulations. These non-GAAP financial measures should be considered supplemental to and not a substitute for financial information prepared in accordance with generally accepted accounting principles. The Company’s definition of these non-GAAP measures may differ from similarly titled measures used by others. Adjusted Net Income adjusts for specified items that can be highly variable or difficult to predict, or reflect the non-cash impact of charges resulting from purchase accounting. All adjustments are tax effected utilizing the federal statutory tax rate for the US, except for changes in the fair value of contingent consideration as the vast majority is non-deductible for tax purposes. Adjusted Net Income margin is defined as Adjusted Net Income divided by total revenues. EBITDA reflects net income excluding the impact of depreciation, amortization, interest expense and provision for income taxes. Adjusted EBITDA also excludes specified items that can be highly variable and the non-cash impact of certain purchase accounting adjustments. Adjusted EBITDA margin is defined as Adjusted EBITDA divided by total revenues. The Company views these non-GAAP financial measures as a means to facilitate management’s financial and operational decision-making, including evaluation of the Company’s historical operating results and comparison to competitors’ operating results. These non-GAAP financial measures reflect an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results and the reconciliations to the corresponding GAAP financial measure may provide a more complete understanding of factors and trends affecting the Company’s business.

The determination of the amounts that are excluded from these non-GAAP financial measures are a matter of management judgment and depend upon, among other factors, the nature of the underlying expense or income amounts. Because non-GAAP financial measures exclude the effect of items that will increase or decrease the Company’s reported results of operations, management strongly encourages investors to review the Company’s consolidated financial statements and publicly filed reports in their entirety.

On January 13, 2020 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that it has initiated a Phase II study in China of surufatinib in combination with Tuoyi (toripalimab) in patients with advanced solid tumors (Press release, Hutchison China MediTech, JAN 13, 2020, https://www.chi-med.com/chi-med-initiates-a-phase-ii-trial-of-surufatinib-in-combination-with-tuoyi-in-patients-with-advanced-solid-tumors/ [SID1234553057]). This follows the recent completion of the Phase I dose finding study and successful establishment of the Phase II combination dosing regimen for surufatinib and Tuoyi.

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This China Phase II clinical study is part of a global collaboration with Shanghai Junshi Biosciences Co. Ltd. ("Junshi"), to evaluate surufatinib, Chi-Med’s oral angio-immuno kinase inhibitor, in combination with Tuoyi, Junshi’s anti-programmed cell death protein 1 ("PD-1") monoclonal antibody which was approved by China’s National Medical Products Administration ("NMPA") in December 2018. The study is designed to test the potential synergistic anti-tumor effects of the combination’s ability to simultaneously target multiple cell types and signaling pathways in the tumor microenvironment.

The Phase II study plans to explore multiple solid tumor patient populations. The primary outcome measures are objective response rate (ORR) and safety. The secondary outcomes include duration of response (DoR), progression-free survival ("PFS"), disease control rate (DCR) and overall survival ("OS"). The lead principal investigator of the study is Professor Lin Shen, Vice President of Peking University Hospital and Cancer Institute. Additional details may be found at clinicaltrials.gov, using identifier NCT04169672.

Christian Hogg, Chief Executive Officer of Chi-Med, said, "We are excited to move into Phase II development on the surufatinib toripalimab combination and look forward to identifying patient groups that could benefit from this innovative treatment regimen."

In December 2019, surufatinib was granted Priority Review status by the Center for Drug Evaluation (CDE) of China’s NMPA for its New Drug Application ("NDA") for the treatment of patients with advanced non-pancreatic neuroendocrine tumors ("NET").

In November 2019, the NDA for surufatinib for the treatment of non-pancreatic NET was accepted for review by the NMPA, and the U.S. Food and Drug Administration granted Orphan Drug designation to surufatinib for the treatment of pancreatic NET.

About Surufatinib
Surufatinib (previously known as HMPL-012 or sulfatinib) is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor ("VEGFR") and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies. Surufatinib is in several late-stage and proof-of-concept clinical trials in China and proof-of-concept clinical trials in the U.S.

According to Frost & Sullivan, the market for anti-angiogenesis VEGF/VEGFR inhibitors in China has grown from US$500 million in 2015 to over US$1.5 billion in 2019 and is expected to reach US$5 billion by 2026.

Chi-Med currently retains all rights to surufatinib worldwide.

Non-Pancreatic NET in China: In 2015, we initiated the SANET-ep study, a Phase III study of surufatinib in advanced neuroendocrine tumors – extra-pancreatic patients in China for whom there is no effective therapy. In June 2019, a 198-patient interim analysis was conducted, leading the independent data monitoring committee to determine that the study met the pre-defined primary endpoint of progression-free survival ("PFS") and should be stopped early. The positive results were highlighted in an oral presentation at the 2019 European Society for Medical Oncology Congress in September 2019. In November 2019, the NDA was accepted for review by the NMPA (clinicaltrials.gov identifier: NCT02588170) and subsequently granted Priority Review status.

Pancreatic NET in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pancreatic NET in China. The primary endpoint is PFS. We expect an interim analysis in the first half of 2020 and enrollment to complete in 2020 (clinicaltrials.gov identifier: NCT02589821).

NET in the U.S. and Europe: We are planning a U.S. registration study in NET patients based on the encouraging data from the Phase II and Phase III studies of surufatinib in NET in China (clinicaltrials.gov identifier: NCT02267967), and the ongoing Phase II study in the U.S. (clinicaltrials.gov identifier: NCT02549937).

Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier NCT03873532).

Immunotherapy combinations: In November 2018 and September 2019, we entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies. This included global collaborations to evaluate the combination of surufatinib with Tuoyi, approved in China by Junshi, and with Tyvyt, approved in China by Innovent Biologics, Inc.

On January 13, 2020 AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck & Co., Inc. inside the US and Canada) reported that a supplemental New Drug Application for Lynparza (olaparib) in combination with bevacizumab has been accepted and granted Priority Review in the US for the maintenance treatment of patients with advanced ovarian cancer who are in complete or partial response to 1st-line platinum-based chemotherapy with bevacizumab (Press release, AstraZeneca, JAN 13, 2020, View Source [SID1234553056]).

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A Prescription Drug User Fee Act (PDUFA) date is set for the second quarter of 2020.

The Priority Review by the US Food and Drug Administration (FDA) was based on results from the pivotal Phase III PAOLA-1 trial, which were published in The New England Journal of Medicine. The trial compared Lynparza when added to standard-of-care (SoC) bevacizumab vs. bevacizumab alone in patients with advanced ovarian cancer in the 1st-line maintenance setting, regardless of their biomarker status or outcome from previous surgery.

The investigator-assessed results showed Lynparza added to bevacizumab reduced the risk of disease progression or death by 41% based on a hazard ratio of 0.59 (p<0.0001) and improved progression-free survival (PFS) to a median of 22.1 months vs. 16.6 months for patients treated with bevacizumab alone.

At two years after trial initiation, 46% of patients treated with Lynparza added to bevacizumab showed no disease progression vs. 28% of patients treated with bevacizumab alone. The safety and tolerability profiles of Lynparza and bevacizumab were consistent with previous trials for each medicine and showed no detriment to quality of life.

Lynparza is the only PARP inhibitor with two positive randomised Phase III trials in the 1st-line maintenance setting for advanced ovarian cancer. It is the only PARP inhibitor approved in the US as a 1st-line maintenance treatment for patients with BRCA-mutated advanced ovarian cancer, based on the SOLO-1 trial. If approved, this would be the fourth indication for ovarian cancer patients in the US for Lynparza.

Ovarian cancer

Ovarian cancer is the eighth most common cause of death from cancer in women worldwide. In 2018, there were nearly 300,000 new cases diagnosed and around 185,000 deaths.1 Most women are diagnosed with advanced (Stage III or IV) ovarian cancer and have a five-year survival rate of approximately 30%.2 For newly diagnosed advanced ovarian cancer, the primary aim of treatment is to delay progression of the disease for as long as possible and maintain the patient’s quality of life with the intent of achieving complete remission or cure.3,4,5,6

PAOLA-1

PAOLA-1 is a double-blind Phase III trial testing the efficacy and safety of Lynparza added to standard-of-care bevacizumab vs. bevacizumab alone, as a 1st-line maintenance treatment for newly diagnosed advanced FIGO Stage III-IV high-grade serous or endometroid ovarian, fallopian tube, or peritoneal cancer patients who had a complete or partial response to 1st-line treatment with platinum-based chemotherapy and bevacizumab.

Results showed Lynparza added to bevacizumab demonstrated a statistically significant and clinically meaningful improvement in PFS, reducing the risk of disease progression or death by 41% and improving PFS to a median of 22.1 months versus 16.6 months for those treated with bevacizumab alone (HR 0.59 [95% CI, 0.49-0.72], p<0.0001). The sensitivity analysis of blinded independent central review (BICR) of PFS was consistent, showing a similar improvement with a median of 26.1 months for Lynparza added to bevacizumab vs. 18.3 months for bevacizumab alone (HR 0.63 [95% CI, 0.51-0.77], p<0.0001).

Overall Grade 3 or above adverse events (AEs) were 57% for Lynparza added to bevacizumab and 51% for bevacizumab alone. The most common AEs ≥20% were nausea (53%), fatigue (53%), hypertension (46%), anaemia (41%), lymphopenia (24%), vomiting (22%) and arthralgia (22%). Grade 3 or above AEs were hypertension (19%), anaemia (17%), lymphopenia (7%), neutropenia (6%), fatigue (5%), nausea (2%), diarrhoea (2%), leukopenia (2%) vomiting (1%) and abdominal pain (1%). AEs led to dose interruption in 54% of patients on Lynparza added to bevacizumab while 20% of patients discontinued treatment.

PAOLA-1 is an ENGOT (European Network of Gynaecological Oncological Trial groups) trial, sponsored by ARCAGY Research (Association de Recherche sur les CAncers dont GYnécologiques) on behalf of GINECO (Groupe d’Investigateurs National des Etudes des Cancers Ovariens et du sein). ARCAGY-GINECO is an academic group specialising in clinical and translational research in patients’ cancers and a member of the GCIG (Gynecologic Cancer InterGroup).

Lynparza

Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in homologous recombination repair, such as mutations in BRCA1 and/or BRCA2. Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. Lynparza is being tested in a range of PARP-dependent tumour types with defects and dependencies in the DDR pathway.

Lynparza is currently approved in 65 countries, including those in the EU, for the maintenance treatment of platinum-sensitive relapsed ovarian cancer, regardless of BRCA status. It is approved in the US, the EU, Japan, China and several other countries as 1st-line maintenance treatment of BRCA-mutated advanced ovarian cancer following response to platinum-based chemotherapy. It is also approved in 44 countries, including the US and Japan, for germline BRCA-mutated, HER2-negative, metastatic breast cancer, previously treated with chemotherapy; in the EU, this includes locally advanced breast cancer. It is approved in the US as a 1st-line maintenance treatment for germline BRCA-mutated metastatic pancreatic cancer. Regulatory reviews are underway in other jurisdictions for ovarian, breast and pancreatic cancers.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, is approved for the treatment of advanced ovarian cancer, metastatic breast cancer and metastatic pancreatic cancer and has been used to treat over 30,000 patients worldwide. Lynparza has the broadest and most advanced clinical-trial development programme of any PARP inhibitor, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

The AstraZeneca and MSD strategic oncology collaboration

In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca’s main capabilities, the Company is actively pursuing innovative partnerships and investment that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.