On August 31, 2018 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a biotechnology company pioneering the development of a new class of ready-to-use cellular therapies, reported second quarter 2018 financial results (Press release, Rubius Therapeutics, AUG 31, 2018, View Source [SID1234529444]).

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!

As of June 30, 2018, Rubius had cash, cash equivalents and marketable securities of $181.7 million. This amount excludes $257.9 million of proceeds after deducting underwriting discounts and commissions from Rubius’ initial public offering (IPO), which closed in July 2018. Based upon its current operating plan, Rubius expects its existing capital resources will be sufficient to fund operations into 2021.

"We have made significant progress over the course of this year – the highlight of which was the closing of our IPO," said Pablo J. Cagnoni, M.D., chief executive officer of Rubius Therapeutics, Inc. "The IPO proceeds give Rubius the resources to execute against our vision of creating life-changing cellular medicines to treat patients with rare diseases, cancer and autoimmune diseases. We are on track to file our first Investigational New Drug application for RTX-134 during the first quarter of 2019, for the treatment of patients with phenylketonuria, a rare disease affecting approximately 16,500 people in the U.S."(1)

Recent Business Highlights

Initial Public Offering

· On July 23, 2018, Rubius closed its IPO, raising $257.9 million of proceeds after deducting underwriting discounts and commissions. The proceeds from this offering, together with existing cash, cash equivalents and marketable securities are expected to be used:

·to purchase, renovate, customize and operate its manufacturing facility;

· to advance RTX-134 through a Phase 1/2a clinical proof-of-concept trial;

· to advance and expand its RED PLATFORMä and its research and development pipeline, including early discovery efforts and IND-enabling studies, and to initiate additional proof-of-concept trials in rare diseases, cancer and autoimmune diseases; and for working capital and other general corporate purposes.

Manufacturing Facility

· On July 31, 2018, Rubius purchased an existing 135,000-square foot manufacturing facility in Smithfield, Rhode Island.

· The company plans to invest approximately $95.0 million through 2020, which includes the $8.0 million purchase price.

Strategic Hires and Internal Capabilities

·Continued to build a leading scientific team and attract experienced leadership to deliver against Rubius’ vision, including the hiring of Pablo J. Cagnoni, M.D., as chief executive officer.

· Strengthened internal capabilities in discovery, platform and therapeutic development and manufacturing.

Second Quarter Financial Results

Cash, cash equivalents and marketable securities totaled $181.7 million as of June 30, 2018, compared to $104.3 million as of December 31, 2017. The increase in cash reflects the net proceeds from Rubius’ Series C preferred stock financing during the first quarter of 2018, offset by its cash used in operations during the period. Research and development expenses were $12.0 million during the second quarter of 2018, compared with $4.8 million for the second quarter of 2017. The increase was attributable to costs incurred in preparation of Rubius’ planned Phase 1/2a clinical trial of RTX-134 in phenylketonuria, as well as an increase in headcount in its research and development function and an increase in external manufacturing and research costs to improve and expand its manufacturing capabilities, prepare for clinical scale production and expand its in vivo testing to support clinical candidate selection. General and administrative expenses were $16.3 million during the second quarter of 2018, compared to $4.2 million for the second quarter of 2017. The increase was primarily due to an increase in stock-based compensation and increases in personnel costs and professional fees as Rubius prepared to operate as a public company.

On August 31, 2018 Exact Sciences Corp. (Nasdaq: EXAS) reported that company management will be presenting at the following investor conferences during September and invited investors to participate by webcast (Press release, Exact Sciences, AUG 31, 2018, View Source [SID1234529313]).

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!

Baird 2018 Global Healthcare Conference, New York
Fireside chat on Wednesday, Sept. 5, at 12:15 p.m. ET
Morgan Stanley 16th Annual Global Healthcare Conference, New York
Fireside chat on Wednesday, Sept. 12, at 4:15 p.m. ET

The webcast can be accessed in the investor relations section of Exact Sciences’ website at www.exactsciences.com.

On August 31, 2018 Kiadis Pharma N.V. ("Kiadis Pharma" or the "Company") (Euronext Amsterdam and Brussels: KDS), a clinical-stage biopharmaceutical company, reported its unaudited interim Financial Results for the six months ended June 30, 2018, which have been prepared in accordance with IAS 34 as adopted by the European Union (Press release, Kiadis, AUG 31, 2018, View Source [SID1234529210]).

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!

Arthur Lahr, CEO of Kiadis Pharma, commented: "We have made tremendous progress in the last six months: ATIR101 is now very close to potential CHMP opinion in 2018, we are on track with our Phase 3 trial, and obtained further confirmatory data from our Phase 2 trials. To allow us to ramp up our Phase 3 trial and prepare for commercialization in the EU we also raised substantial equity and debt facilities that extended our cash runway into the third quarter of 2019, and, upon positive CHMP opinion, potentially into the first quarter of 2020. We have also significantly strengthened our organization in medical, operations, commercial and finance functions. Kiadis is in great shape and well positioned to deliver on the promise of ATIR101."

Operating highlights – ATIR101 (including post reporting period)

European marketing authorization application for ATIR101:
Responses to the Day 120 List of Questions submitted in March 2018;
Day 180 List of Issues received in May 2018 and responses submitted in August 2018;
On track to obtain CHMP opinion from the European Medicines Agency in the fourth quarter of 2018.
Phase 3 trial CR-AIR-009, comparing ATIR101 against the post-transplant cyclophosphamide (PTCy) or ‘Baltimore’ protocol:
Progress in line with internal plans: 14 clinical sites are currently open for recruitment, 16 patients have been enrolled;
Protocol amendment submitted to regulatory authorities: number of patients increased to 250 to further increase power [80% power to detect 16% Graft-versus-host-disease-free and Relapse-Free Survival (GRFS) difference]; interim analysis to occur after 2/3 of GRFS events to increase chance of positive read out, now expected in the second half of 2020; conditioning regimens harmonized between the two treatment arms to reduce heterogeneity.
Phase 2 trial CR-AIR-008 (‘008’): The last patient received a single dose of ATIR101 in January 2018.
Pooled analysis: Further analysis of 1-year Phase 2 pooled data [Intention-To-Treat (ITT), 37 patients] from studies CR-AIR-007 and single dose CR-AIR-008 shows GRFS 53% [95% confidence interval (CI), 39%-72%]; Overall Survival (OS) 58% (95% CI, 44%-77%), in line with Phase 2 CR-AIR-007 trial. For the PTCy/Baltimore protocol, single site data from Johns Hopkins (McCurdy et al. 2017) and Atlanta (Solh et al, 2016) show a disease-risk index (DRI) normalized 1-year GRFS value of 40% and 30%, respectively.
Operating highlights – Organization (including post reporting period)

Robbert van Heekeren resigned as Chief Financial Officer and as member of the Management Board.
Scott A. Holmes appointed as new Chief Financial Officer.
Organization strengthened across all functions, comprises 73 employees, up from 51 a year ago. Key new appointments include head of Medical US (former Iovance/ Dendreon), head of Medical EU (former Genzyme/ AstraZeneca), head of market access EU (former Genzyme/ Novo Nordisk), head of pharmacovigilance (former Astellas), head of facilities (former Merck/ Douwe Egberts).
Otto Schwarz, former Chief Operating Officer of Actelion and Mr. Subhanu Saxena, former Chief Executive Officer of Cipla and former member of the senior executive team of Novartis, were appointed as Supervisory Board members of the Company at the Annual General Meeting of shareholders in June 2018. Mr. Stuart Chapman resigned from the Supervisory Board following the shareholders’ meeting.
Financial highlights (including post reporting period)

In the first six months of 2018, the Company did not generate any revenues. Total operating expenses increased by EUR2.9 million from EUR8.2 million in the first six months of 2017 to EUR11.1 million in the same period of 2018. This increase was primarily caused by a further expansion of the workforce in all areas of the organization, the move to a larger building which includes a commercial manufacturing facility, laboratories and office space, and consultancy expenses for business development and market access.
In the first six months of 2018, net financial result came in at EUR3.0 million compared to EUR0.4 million for the same period of 2017. Higher finance costs were mainly the result of higher interest expenses on loans and borrowings, and a net foreign exchange loss in the first six months of 2018 compared to a net foreign exchange gain in 2017.
The net loss for the six months ended June 30, 2018 came at a level of EUR14.1 million compared to a loss of EUR8.5 million for the six months ended June 30, 2017. Operating expenses and net result for the first six months of 2018 were in line with management expectations.
The Company ended the first six months of 2018 with EUR41.7 million in cash and cash equivalents. In March 2018, the Company issued 2.6 million shares and raised EUR23.4 million in gross proceeds.
On July 31, 2018, the Company received a new debt facility from Kreos Capital V (UK) Ltd providing the Company with up to EUR20 million of additional financing.

On August 30, 2018 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported that it is scheduled to
present at the following upcoming events (Press release, Arrowhead Pharmaceuticals, AUG 30, 2018, View Source [SID1234529190]):

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!

Annual B. Riley FBR Healthcare Conference– New York, September 4, 2018
Bruce Given, M.D., Arrowhead’s chief operating officer, and Vincent Anzalone, CFA, vice president of investo relations, will participate in three panel
discussions

18th World Gastroenterologists Summit – Auckland, New Zealand, September 7-8, 2018
September 7, 09:45 a.m. NZST – Dr. Given will deliver a keynote presentation titled, "Hepatitis B in focus: New biology, new targets and real hope for finite therapy"

European Respiratory Society International Congress 2018 – Paris, September 15-19, 2018
September 16, 12:15 p.m. CEST –Erik Bush, Ph.D., Arrowhead’s director of extra-hepatic targeting, will deliver an oral presentation titled, " Targeting

ENaC with an epithelial RNAi trigger delivery platform for the treatment of cystic fibrosis"

Antivirals: Targeting HBV and Beyond – Boston, September 25, 2018
September 25, 12:00 p.m. EDT –James Hamilton, M.D., Arrowhead’s vice president of clinical development, will deliver an oral presentation titled, "Using siRNA to target the HBV transcriptome"

A copy of presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website after the presentations conclude.

On August 30, 2018 Zai Lab Limited ("Zai Lab" or the "Company") (NASDAQ:ZLAB), a Shanghai-based innovative biopharmaceutical company, reported financial results for the six months ended June 30, 2018, and provided a corporate update (Press release, Zai Laboratory, AUG 30, 2018, View Source [SID1234529211]).

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 first half of 2018 has been a period of tremendous progress for Zai Lab, marked by the continued advancement of our clinical programs, expansion of our therapeutic pipeline, preparation for our commercial launch and strengthening of our management team," said Dr. Samantha Du, Zai Lab’s Chief Executive Officer. "For ZL-2306 (niraparib), we continue to enroll patients in three separate pivotal trials in China targeting ovarian and small cell lung cancer, and are exploring additional indications by planning to initiate several more trials in the remainder of the year. The goal of our comprehensive clinical development program is to establish ZL-2306 (niraparib) as the leading PARP inhibitor in China. In addition, we continue to advance our multiple late-stage clinical programs in both oncology and infectious diseases. The past six months also marked the exciting transition of Zai Lab to a commercial stage company. In anticipation of the launch of ZL-2306 (niraparib) in Hong Kong later this year and in China, we started building our sales and marketing team, and importantly, appointed industry veteran William Liang as Chief Commercial Officer. In addition, we appointed Yong-Jiang Hei, M.D., Ph.D., as our Chief Medical Officer (CMO) for oncology to lead and advance our oncology pipeline. As we approach our upcoming clinical and commercial milestones throughout the remainder of 2018, we believe that Zai Lab is well-positioned to continue our positive momentum."

Recent Clinical Highlights

ZL-2306 (niraparib)

In August 2018, Zai Lab announced the early completion of an open-label study to evaluate the pharmacokinetic (PK) profile of ZL-2306 (niraparib) made in China in Chinese ovarian cancer patients. The study demonstrated a comparable PK profile of Chinese patients who were administered ZL-2306 to the PK profile of patients evaluated in Tesaro’s clinical trials using product manufactured outside of China. These results support the regulatory review of ZL-2306 (niraparib) in China.

In August 2018, Zai Lab enrolled the first patient in its Phase III registration trial of ZL-2306 (niraparib) as a first-line maintenance therapy in small cell lung cancer (SCLC) in China. This will be the first clinical trial of ZL-2306 (niraparib) in this type of cancer.

In June 2018, Zai Lab dosed the first patient in its Phase III China registration trial of ZL-2306 (niraparib) for first-line maintenance therapy of patients with platinum-responsive ovarian cancer.

FPA144 (bemarituzumab)

In May 2018, Zai Lab received clinical trial application (CTA) approval from China’s National Drug Administration (CNDA) to enroll Chinese patients in the Phase I/III FIGHT (FGFR2b Inhibition in Gastric and Gastroesophageal Junction Cancer Treatment) global registrational trial, evaluating FPA144 (bemarituzumab) in combination with a modified FOLFOX chemotherapy regimen. Zai Lab received CTA approval three months ahead of schedule.

ZL-2401 (omadacycline)

In April 2018, Zai Lab’s bridging approach leveraging the Phase III ZL-2401 (omadacycline) studies conducted abroad was accepted by China’s Center for Drug Evaluation (CDE). An agreement was reached with CDE for the Company’s proposal to conduct a truncated clinical program for approval in China. Zai Lab obtained CTA approval from CNDA within three and a half months of submitting its application and is ready to implement the requested China studies in the fourth quarter of 2018.

ZL-3101 (Fugan)

In August 2018, results of a Phase II study evaluating ZL-3101 (Fugan), a natural product in patients with mild to moderate atopic dermatitis, received four months ahead of schedule, showed ZL-3101 was safe and well-tolerated, but did not show signs of efficacy over placebo. The Company elected to discontinue further development of ZL-3101 (Fugan) based on these findings and shift related resources to other programs.

Recent Business & Corporate Developments

In August 2018, Zai Lab appointed Yong-Jiang Hei, M.D., Ph.D., as its CMO, Oncology. Dr. Hei is a seasoned industry expert with over 20 years of experience in clinical development in oncology and will support the strategic development and expansion of the Company’s growing oncology clinical pipeline. Most notably, he served at Amgen, Inc. for approximately 10 years in multiple roles of increasing responsibility in oncology global development. Dr. Qi Liu will transition to an advisory role.

In August 2018, Zai Lab appointed Kai-Xian Chen, Ph.D., to its Board of Directors. Professor Chen is a globally recognized scientist and widely regarded as a pioneer in the field of interdisciplinary healthcare research. In connection with Professor Chen’s appointment, Marietta Wu, Ph.D. retired from the Board of Directors.

In June 2018, Zai Lab appointed William Liang as Chief Commercial Officer. William brings more than two decades of experience in the pharmaceutical industry, with expertise in commercial launch, strategy and operations. Prior to joining Zai Lab, William served as Vice President at AstraZeneca heading up the Oncology Business Unit in China.

In June 2018, Zai Lab completed construction of a biologics pilot facility using GE Healthcare’s Flex Factory platform technology. The facility is capable of supporting biologics drug products for clinical and non-clinical development of Zai Lab’s drug candidates.

In May 2018, Zai Lab entered into an exclusive worldwide agreement with Crescendo Biologics for ZL-1102, a topical, innovative antibody for potential application in inflammatory indications, including psoriasis.

In April 2018, Zai Lab entered into an exclusive license agreement in Asia-Pacific, and a global development agreement, with Entasis Therapeutics (Entasis) for ETX2514, a novel broad-spectrum intravenous inhibitor of ß-lactamases, for the treatment of a variety of serious multidrug-resistant (MDR) infections. In combination with sulbactam, ETX2514 is particularly active against MDR Acinetobacter baumannii infections.

Zai Lab continues to expand its platform and human resources. As of June 30, 2018, Zai Lab employed 182 full-time employees, including 37 employees with M.D. or Ph.D. degrees. Currently, approximately 85% of the Company’s employees are engaged in R&D activities. Employee mix is projected to change as the Company continues to build out its commercial team.

Zai Lab has, from time to time, evaluated partnership opportunities and may, in the future, make acquisitions of, or investments in, companies that Zai Lab believes have products or capabilities that are a strategic or commercial fit with Zai Lab’s current drug candidates and business or otherwise offer opportunities for the Company.

Upcoming Milestones

ZL-2306 (niraparib)

On September 23, 2018, the design of the Phase III study of ZL-2306 (niraparib) as maintenance therapy in first line platinum-responsive small cell lung cancer (SCLC) patients will be presented at the International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer (WCLC).

On September 21, 2018, results from a Phase I PK and safety study of ZL-2306 (niraparib) in Chinese patients with epithelial ovarian cancer (OC) will be presented at the 21st Annual Meeting of Chinese Society of Clinical Oncology (CSCO).

Zai Lab plans to begin commercializing ZL-2306 (niraparib) as a second-line maintenance therapy in patients with recurrent platinum-sensitive ovarian cancer in Hong Kong during the fourth quarter of 2018, and in Macau thereafter.

ZL-2401 (omadacycline)

Zai Lab expects to initiate a bioequivalence and PK bridging study in China to demonstrate comparability in the PK profile of Chinese patients administered with ZL-2401 (omadacycline) to non-Chinese patients, in preparation for its New Drug Application (NDA) submission in China. This program is expected to start in the fourth quarter of 2018.

Paratek Pharmaceuticals has a Prescription Drug User Fee Act (PDUFA) action date set in October 2018 for its NDA for once-daily oral and intravenous formulations of omadacycline for the treatment of community-acquired bacterial pneumonia (CABP) and acute skin and skin structure infections (ABSSSI). On August 8, 2018, Antimicrobials Drug Advisory Committee of the U.S. FDA voted in favor of the approval of IV and oral omadacycline.

FPA 144 (bemarituzumab)

In collaboration with Five Prime, Zai Lab plans to initiate patient dosing in the China portion of the randomized, controlled Phase III portion of the Phase I/III FIGHT global registration trial in the fourth quarter of 2018.

ETX2514

In collaboration with Entasis, Zai Lab plans to initiate patient dosing in the Asia-Pacific portion of the Phase III global registration trial of ETX2514 for MDR Acinetobacter pneumonia and bloodstream infections in 2019.

ZL-2301 (brivanib)

Data from a Phase II trial of ZL-2301 (brivanib) as a second-line treatment for advanced hepatocellular cancer patients in China are expected in the second half of 2018.

On September 22, 2018, preliminary study results from the Phase II trial of ZL-2301 (brivanib) in advanced HCC patients with systemic treatment failure or intolerance will be presented at the 21st Annual Meeting of Chinese Society of Clinical Oncology (CSCO).

ZL-2302

In the fourth quarter of 2018, Zai Lab plans to initiate Phase I clinical trials in China of ZL-2302 for the treatment of patients with non-small cell lung cancer (NSCLC) who have ALK mutations and developed crizotinib resistance and/or brain metastasis.

Financial Results for the Six Months Ended June 30, 2018

As of June 30, 2018, cash and cash equivalents and short-term investments totaled $177.7 million.

Research and development expenses were $34.6 million for the six months ended June 30, 2018 compared to $20.9 million for the same period in 2017. The increase was primarily due to higher clinical and preclinical costs from the advancement of the Company’s expanded pipeline, and expansion of research efforts to support internal programs.

General and administrative expenses were $6.4 million for the six months ended June 30, 2018 compared to $4.0 million for the same period in 2017. The increase was primarily due to the increase in payroll and payroll-related expenses as a result of the increased headcount from expanded operations, and increased costs associated with operating as a public company.

For the six months ended June 30, 2018, Zai Lab reported a net loss of $41.5 million, or basic and diluted net loss per share attributable to common stockholders of $0.83, compared to a net loss of $24.4 million, or basic and diluted net loss per share attributable to common stockholders of $2.30, for the same period in 2017.