On September 3, 2019 Santhera Pharmaceuticals (SIX: SANN) reported first half-year results as of June 30, 2019, and provides an update on its pipeline and strategic focus (Press release, Santhera Pharmaceuticals, SEP 3, 2019, View Source [SID1234539203]).

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!

Thomas Meier, PhD, Chief Executive Officer of Santhera, said: "Following a series of transactions which strategically reshaped the direction of the Company, we are well positioned to advance our long-term growth strategy of focusing on medicines to treat neuromuscular and pulmonary diseases. For 2019, we are on track to achieve our strategic objectives, having filed for conditional marketing authorization of Puldysa (idebenone) in Europe for the preservation of respiratory function in Duchenne muscular dystrophy (DMD) patients. We expect an opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) mid-2020. Our goal is to help all DMD patients, irrespective of causative mutations, disease stage or age. We were therefore very pleased about the recent publication by ReveraGen of positive Phase IIa-extension study results with vamorolone in the journal Neurology. These data together with previous publications provide proof-of-concept that vamorolone can improve gross muscle function outcomes in young patients with DMD and indicate a better tolerability profile than standard corticosteroids."

"During the period, we announced an exclusive agreement with Chiesi Group to out-license Raxone for the treatment of Leber’s hereditary optic neuropathy (LHON) for a total consideration of up to EUR 93 million with an upfront payment of EUR 44 million. In monetizing this commercial product, we have strengthened our financial position to focus on upcoming regulatory milestones and commercialization activities for our neuromuscular and pulmonary product candidates, which are core to our long-term growth strategy."

Financial highlights:

1H-2019 sales on track with CHF 18.3 million, an increase of 14% compared to 1H-2018
Operating expenses of CHF 38.2 million (1H-2018: CHF 39.9 million)
Operating result of CHF –22.4 million (1H-2018: CHF –26.3 million) leading to a net result of CHF –26.9 million (1H-2018: CHF –27.4 million)
Cash and cash equivalents of CHF 43.7 million (August 31, 2019)
Full-year sales guidance of CHF 25-27 million
First half-year overview:

Regulatory momentum for Puldysa in DMD
In June, the European Medicines Agency (EMA) validated Santhera’s application for conditional marketing authorization (CMA) for Puldysa (idebenone) in the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) who are not using glucocorticoids. The review process by the EMA’s CHMP has begun and the Company expects an opinion by the CHMP around mid-2020. In addition, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) renewed its positive scientific opinion for idebenone for patients with DMD in respiratory function decline who are not taking glucocorticoids, under the Early Access to Medicines Scheme (EAMS).

Long-term data with Puldysa
In February, Santhera announced the results of its SYROS study, which investigated long-term efficacy with idebenone in slowing respiratory function loss in patients with DMD. The study demonstrated that long-term treatment with idebenone consistently contributed to the preservation of respiratory function for up to 6 years in a real-world setting. This long-term data further supports the potential for idebenone to positively modify the course of respiratory function decline and delay the time to clinically relevant milestones.

Strong Raxone performance underscores Santhera’s commercial expertise
Net sales of Raxone in Europe amounted to CHF 18.3 million (1H-2018: CHF 16.0 million) which corresponds to a 14% increase year-on-year, in line with the previous full-year product sales guidance. In May, the Company announced that it had entered into an exclusive license agreement with Chiesi Group for Raxone for the treatment of LHON, for a total consideration of up to EUR 93 million. With the closing at the end of July, Santhera has now transferred all rights to Chiesi Group for the development, commercialization and distribution of Raxone for the treatment of LHON and any other potential ophthalmological indications for all territories worldwide except the US and Canada. After the closing and for an interim period, Santhera will provide support services to Chiesi Group to enable a seamless handover of the business and will continue to commercialize Raxone for LHON in France.

Positive study data with vamorolone published– pivotal study enrolling
Last month, positive data from 6-month Phase IIa-extension study (VBP15-003) with vamorolone in DMD were published by ReveraGen in Neurology [1]. The data demonstrated dose-related improvement of gross muscle function in patients with DMD treated with vamorolone. Vamorolone was reported to be safe and well tolerated up to the highest dose tested (6.0 mg/kg/day). Biomarker data indicated reduced occurrence of side effects typical for traditional corticosteroid drugs. Based on these data, vamorolone has potential to replace standard corticosteroids currently used in patients with DMD.
ReveraGen is presently enrolling the Phase IIb VISION-DMD study [2] (VBP15-004; clinicaltrials.gov: NCT03439670), designed as a pivotal efficacy and safety trial. The study is expected to be fully enrolled by the end of 2019. Accordingly, the 6-month randomized placebo-controlled treatment period would end by mid-2020, followed by data analysis. NDA submission could be towards year end 2020.

Collaboration to advance gene therapy research for rare neuromuscular disease
Santhera initiated a collaboration with the Biozentrum of the University of Basel to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A). The preclinical research collaboration builds on previous work with omigapil, which was recently studied in a Phase I clinical trial and could act complementary. The program is supported by public funding for innovation in Switzerland through a grant from Innosuisse – the Suisse Innovation Agency.

Continued investment in clinical development
Santhera is running several late-stage clinical trials, among them SIDEROS, the largest ever conducted study in patients with DMD. The SIDEROS study is 84% recruited and positive outcome of the study will form the basis for NDA submission for patients with DMD irrespective of glucocorticoid status, currently expected in 2H-2021. In addition, the preparation of the MAA for Puldysa, remaining post-approval clinical work for Raxone in LHON and increased clinical development work with POL6014 entailed slightly higher development expenses of CHF 19.3 million (+2% year-on-year). Overall, operating expenses showed a small decline (–4%) driven by lower expenses for commercial activities (–10%).

Liquidity base allows for the continuation of the strategy as planned
In April, Santhera raised CHF 7.1 million by the placement of 500,000 shares. As of the end of June 2019, Santhera had cash and cash equivalents of CHF 12.7 million (December 31, 2018: CHF 22.0 million). Together with the net proceeds from the initial payment from Chiesi Group following closing of the licensing transaction, liquid funds amounted to CHF 43.7 million (August 31, 2019), allowing the Company to proceed with its clinical trial program and regulatory filings as planned.

Outlook and Guidance
Based on the performance in the first half-year of 2019, the Company expects to achieve annual net sales with Raxone in the currently approved indication LHON of CHF 25-27 million in 2019, taking into account that Chiesi Group has taken over commercial sales for Raxone from August in all European countries except France. The operational priorities for 2019 are the preparation of European market entry with Puldysa in DMD in 2020, completing enrollment into the DMD SIDEROS trial to support the planned US-submission of Puldysa in DMD, and advancing the other clinical stage candidates in the pipeline, particularly vamorolone and POL6014.

References:
[1] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 2019. View Source
[2] View Source

Half-year Report
The Santhera Half-year Report 2019 is available for download on the Company’s website at www.santhera.com/investors-and-media/investor-toolbox/financial-reports.

Conference Call
Santhera will host a conference call on September 3, 2019 at 13:00 CEST / 12:00 BST / 07:00 EDT. Thomas Meier, PhD, CEO of Santhera, will discuss the half-year 2019 financial results and will provide an update on corporate developments. Participants are invited to call one of the following numbers 10-15 minutes before the conference call starts (no dial-in code is required):
Europe: +41 58 310 50 00
UK: +44 207 107 06 13
USA: +1 631 570 56 13

On September 3, 2019 Zai Lab Limited ("Zai Lab" or "the Company") (NASDAQ: ZLAB), a China and U.S.-based innovative commercial-stage biopharmaceutical company, reported financial results for the six months ended June 30, 2019 and provided a corporate update (Press release, Zai Laboratory, SEP 3, 2019, View Source [SID1234539202]).

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!

"During the first half of 2019 we continued to make significant progress toward our objective of becoming a fully integrated global biopharmaceutical company. Our portfolio currently includes ten clinical assets, nine of which are in late stage, targeting over 20 indications," said Dr. Samantha Du, Founder and Chief Executive Officer of Zai Lab. "We launched our two lead oncology products, ZEJULA and Optune in Hong Kong and ZEJULA in Macau. We submitted applications for marketing approval in China for both of these therapeutics and have built a full commercial platform. Other late-stage product candidates, such as margetuximab, ripretinib, bemarituzumab, omadacycline and sulbactam-durlobactam, continue to advance in clinical development and hit significant milestones through efforts by Zai Lab and our strategic partners. As always, we constantly search for novel and potentially best-in-class clinical candidates that would both complement our pipeline across key indications and address unmet medical needs in Greater China. So far this year, we entered into two collaborative agreements, one with Deciphera on an advanced clinical-stage gastrointestinal cancer drug candidate ripretinib and the other with Incyte on a potential globally competitive anti-PD-1 antibody, INCMGA0012, which may be combined with several of our other oncology candidates. With a growing pipeline and execution progress, Zai Lab has established a global reputation as a China biotech pioneer. Zai Lab is already an integrated biopharma with nearly 600 employees across six offices in Greater China and the U.S. With proven global and local clinical development, regulatory and business development expertise, we also look forward to demonstrating our commercial and discovery capabilities in the near future. The proceeds from our recent financing positions us to execute on all of our top initiatives as we continue to build and expand our business. We expect the upcoming months to potentially be transformative for Zai Lab as we prepare for the marketing approvals for ZEJULA and Optune in China."

Recent Pipeline and Product Highlights

Oncology

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

In August 2019, Zai Lab announced publication of "Phase I Pharmacokinetic Study of Niraparib in Chinese Patients with Epithelial Ovarian Cancer" in The Oncologist, demonstrating that the pharmacokinetic (PK) profile of niraparib in Chinese patients were comparable to that of patients evaluated in GSK’s global PK study.

In July 2019, our partner GSK (formerly Tesaro) announced positive top-line results of the pivotal Phase 3 PRIMA study of ZEJULA as first line maintenance in patients with ovarian cancer. The study met its primary endpoint of a statistically significant improvement in progression free survival (PFS) for women regardless of biomarker status.

In June 2019, GSK announced the U.S. Food and Drug Administration’s (FDA) acceptance of the supplemental New Drug Application (NDA) for ZEJULA in late-stage ovarian cancer based on the results of the QUADRA study. The application was granted priority review with a PDUFA action date of October 24, 2019.

In June 2019, Zai Lab announced receipt of marketing authorization to commercialize ZEJULA in Macau for women with relapsed ovarian cancer.

In January 2019, Zai Lab announced that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has granted priority review status to the NDA for niraparib for the maintenance treatment of adult patients with recurrent epithelial, ovarian, fallopian tube or primary peritoneal ovarian cancer who are in a complete or partial response to platinum-based chemotherapy. NMPA acceptance of the NDA was announced in December 2018, more than one year ahead of expectations.

In January 2019, Zai Lab announced that it completed patient enrollment in its pivotal clinical trial of niraparib, which is in development for second-line maintenance therapy in patients with recurrent platinum-sensitive ovarian cancer.

Optune, a Tumor Treating Fields delivery system that uses electric fields tuned to specific frequencies to disrupt cancer cell division, inhibiting tumor growth and causing affected cancer cells to die.

In August 2019, the NMPA granted Innovative Medical Device Designation for Optune, which will allow the Company to take advantage of an expedited approval process for Optune.

In May 2019, our partner Novocure received the U.S. FDA approval for the NovoTTF-100L System in combination with chemotherapy for the first-line treatment of unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM). NovoTTF-100L, the first treatment for MPM approved by the U.S. FDA in more than 15 years, is a non-invasive, antimitotic cancer treatment that delivers Tumor Treating Fields to the region of the tumor.

In March 2019, Novocure initiated a Phase 3 clinical trial of Tumor Treating Fields in patients with recurrent ovarian cancer. Novocure’s Tumor Treating Fields is currently in late stage clinical development for four solid tumor indications including non-small cell lung cancer, brain metastases, pancreatic and ovarian cancers.

In February 2019, Optune was launched in Hong Kong for patients with GBM.

Margetuximab, an investigational, Fc-optimized monoclonal antibody (mAb) that targets human epidermal growth factor receptor 2 (HER2).

In February and June 2019, our partner MacroGenics announced positive top-line results from its SOPHIA Phase 3 study of margetuximab in patients with HER2-positive metastatic breast cancer and presented the primary analysis at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June 2019. The trial met the first sequential primary endpoint of prolongation of PFS in patients treated with the combination of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy.

In January 2019, MacroGenics presented updated clinical data from a combination study of margetuximab plus pembrolizumab in gastric cancer at the 2019 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium. Based on these positive data, MacroGenics and Zai Lab plan to initiate a global registrational clinical trial of margetuximab in combination with checkpoint inhibitor molecules, including INCMGA0012 (anti-PD-1 mAb) and MGD013 (bispecific PD-1 x LAG-3 molecule) in first line patients with HER2-positive gastric or gastroesophageal junction cancer in 2019.

Ripretinib, 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 August 2019, our partner Deciphera announced positive top-line data from the pivotal Phase 3 INVICTUS clinical study of ripretinib in patients with fourth-line and fourth-line plus GIST. The study met its primary endpoint of a significantly improved median PFS of 6.3 months compared to 1.0 month in the placebo arm, and the risk of disease progression or death was reduced by 85% (HR of 0.15, p<0.0001) compared to placebo.

In June 2019, Zai Lab entered into an exclusive license agreement with Deciphera for the right to develop and commercialize ripretinib in Greater China.

INCMGA0012, an investigational mAb that inhibits PD-1, currently being evaluated as a monotherapy in registrational trials for patients with MSI-high endometrial cancer, Merkel cell carcinoma and anal cancer.

In July 2019, Zai Lab announced a collaboration and license agreement with Incyte for the development and commercialization of INCMGA0012 in Greater China. The collaboration enables Zai Lab to rapidly explore the potential of a competitive anti PD-1 as both monotherapy and combination therapy, unlocking the full potential of the Company’s pipeline.

Bemarituzumab, a first-in-class isoform-selective antibody with enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) in development as a targeted immunotherapy for tumors that overexpress FGFR2b.

Enrollment in the pivotal FGFR2b Inhibition in Gastric and Gastroesophageal Junction Cancer Treatment (FIGHT) trial continues ahead of projections due to FGFR2b biomarker prevalence that has remained steady at more than 30%.

Our partner Five Prime Therapeutics plans to pause enrollment in the fourth quarter of 2019 and conduct an early futility analysis for the FIGHT trial by the first half of 2020.

Infectious Disease

NUZYRA (Omadacycline), 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 July 2019, enrollment of the CDE-requested bridging trial in China was completed ahead of schedule. Zai Lab also completed the bioequivalence, PK and microbiology studies on time.

Sulbactam-durlobactam, a novel, broad-spectrum and potent inhibitor of Class A, C, and D β-lactamases. This is a novel IV antibiotic for the treatment of infections caused by carbapenem-resistant Acinetobacter.

In May 2019, Zai Lab received Clinical Trial Application (CTA) acceptance from the NMPA to initiate a Phase 3 clinical trial for the treatment of carbapenem-resistant Acinetobacter baumannii infections in China. The Acinetobacter Treatment Trial Against Colistin (ATTACK) study is a global Phase 3 clinical trial that will enroll approximately 300 patients from 18 countries. Zai Lab will be responsible for patient enrollment in China, and potentially provide early access for patients in Asia-pacific countries. Entasis Therapeutics will be responsible for patient enrollment in the U.S. and Europe.

Recent Corporate Developments

In July 2019, Dr. Alex A. Adjei, M.D., Ph.D., a world-renowned clinical scientist from the Mayo Clinic, with expertise in drug development, cancer pharmacology and early phase clinical trials, joined Zai Lab’s Scientific Advisory Board (SAB). Dr. Adjei will advise Zai Lab as it continues to expand the clinical trials of the multiple innovative molecules in its oncology pipeline.

In June 2019, Zai Lab appointed Valeria Fantin, Ph.D., as Chief Scientific Officer to further enhance internal discovery capabilities and continue to grow its U.S. presence. Dr. Fantin will lead the Company’s internal drug discovery effort on a global basis and is directly reporting to Dr. Samantha Du.

In May 2019, Zai Lab raised $216.2 million, net of underwriting fees, in a public offering of American Depositary Shares, each representing one ordinary share of the Company.

In February 2019, Immuno-Oncology Pioneer, Lieping Chen, M.D., Ph.D., from the Yale School of Medicine, joined Zai Lab’s SAB. Dr. Chen will advise Zai Lab as it continues to progress its internally-developed oncology pipeline.

Zai Lab continues to expand its U.S. presence to enhance internal drug discovery efforts. As of July 31, 2019, our U.S. R&D center in the San Francisco Bay Area has approximately 10 employees and is growing.

Zai Lab continues to expand its platform, particularly its R&D and commercial teams. As of July 31, 2019, Zai Lab employed 577 full-time employees, with 247 and 246 employees engaged in R&D and commercial activities, respectively.

Anticipated Upcoming Milestones

ZEJULA

GSK to present PRIMA data at an upcoming medical conference

Potential U.S. FDA approval for ovarian cancer late line treatment in the fourth quarter of 2019

Potential China NDA approval for ovarian cancer second-line maintenance therapy

Potential China commercial launch

Completion of enrollment of China Phase 3 clinical trial for ovarian cancer first-line maintenance therapy (PRIME)

China second-line ovarian cancer Phase 3 clinical trial (NORA) to reach target events by the fourth quarter of 2019 with readout in the first half of 2020

Zai Lab to present data from PRIME and NORA studies at Asian Society of Gynecologic Oncology (ASGO) 2019

Continued enrollment of the China small cell lung cancer (SCLC) Phase 3 clinical trial

Initiate trials in other key indications in China.

Optune

Potential China GBM MAA approval with trial waiver

Potential China commercial launch

Initiate exploratory trial in China for gastric cancer with first patient in by the end of 2019

Prepare trials in other key indications in China

Margetuximab

Initiate bridging trial of heavily pretreated HER2-positive metastatic breast cancer patients in China

MacroGenics to present the results from a pre-specified interim overall survival (OS) analysis and submit a Biologics License Application (BLA) to the U.S. FDA in the fourth quarter of 2019

Potential China CTA approval for the planned global gastric cancer and join MacroGenics to initiate the global registrational trial in combination with checkpoint inhibitor molecules, including INCMGA 0012 and MGD013, for patients with gastric or gastroesophageal junction cancer in 2019

Ripretinib

Deciphera to present INVICTUS data at an upcoming medical conference

Deciphera to submit NDA to the U.S. FDA for fourth-line and fourth-line plus GIST by the first quarter of 2020

Discussions with the NMPA for an accelerated approval for fourth-line and fourth-line plus GIST, leveraging the INVICTUS data

Potential China CTA approval for INTRIGUE trial for second-line GIST patients and contribute Chinese patients to the global registrational study

Brivanib

Potential CTA approval to initiate a combination study of brivanib and a checkpoint inhibitor in the treatment of second-line hepatocellular cancer

NUZYRA

Complete the Phase 3 clinical trial in China and submit China NDA for two major indications: CABP and ABSSSI

Sulbactam-durlobactam

Initiate ATTACK global Phase 3 registrational study in China with Entasis

Discovery

Announce one to two global investigational new drug (IND) applications by 2020

First Half 2019 Financial Results

For the six months ended June 30, 2019, net revenues were $3.4 million, reflecting the commercial launch of our two lead oncology products in Hong Kong, with $1.9 million and $1.5 million from ZEJULA and Optune, respectively.

Since the Hong Kong launch of ZEJULA in October 2018, it has rapidly gained market share in the region despite being launched more than two years behind LYNPARZA. Based on IQVIA* (formerly IMS) data, ZEJULA is now the market leading PARP inhibitor with market share in Hong Kong of 66% in the second quarter of 2019.

Officially launched in February 2019, Optune’s revenue has ramped ahead of expectations, with strong momentum generated for the second half of the year.

R&D expenses were $58.9 million for the six months ended June 30, 2019 compared to $34.6 million for the same period in 2018. The increase in R&D expenses was primarily attributable to an increase in licensing fees, ongoing and newly initiated late-stage clinical trials, headcount and payroll and payroll-related expenses, and expansion of research efforts to support internal programs.

Selling, general and administrative expenses were $29.5 million for the six months ended June 30, 2019 compared to $6.4 million for the same period in 2018. The increase was primarily attributable to an increase in payroll and payroll-related expenses as Zai Lab continued to expand its commercial operations in China.

For the six months ended June 30, 2019, Zai Lab reported a net loss of $83.3 million, or a net loss per share attributable to common stockholders of $1.37, compared to a net loss of $41.5 million, or net loss per share attributable to common stockholders of $0.83, for the same period in 2018.

As of June 30, 2019, cash and cash equivalents and short-term investments totaled $393.2 million. This includes net proceeds of $216.2 million obtained in a public offering of American Depositary Shares in May 2019.

* IQVIA Hong Kong Pharmaceutical Audit QTR 2Q2019 data

Conference Call and Webcast Information

Zai Lab will host a live conference call and webcast today, September 3, 2019 at 8:30 a.m. EDT to review its financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of Zai Lab’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. Alternatively, please call (866) 519-4004 (U.S.); +65 67135090 (International); +852 30186771 (Hong Kong); 4006208038 (China) to listen to the live conference call. The conference ID number for the live call is 6785308. A replay of the webcast will be available for on Zai Lab’s website for two weeks following the live conference call. The conference ID for the replay is 6785308.

On September 3, 2019 Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported that an overview of the company’s business strategy and development-stage programs will be given at two upcoming conferences being held in New York on September 10th and September 11th (Press release, Spectrum Pharmaceuticals, SEP 3, 2019, View Source [SID1234539201]).

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!

Rodman & Renshaw 21st Annual Global Investment Conference, Sponsored by H.C. Wainwright
When: Tuesday, September 10, 2019 at 9:10 AM ET
Morgan Stanley 17th Annual Global Healthcare Conference
When: Wednesday, September 11, 2019 at 1:35 PM ET
The presentations will be webcast live and may be accessed by visiting Spectrum’s website at View Source

On September 3, 2019 Pfizer Inc. reported invites investors and the general public to listen to a webcast of a discussion with Frank D’Amelio, Chief Financial Officer and Executive Vice President, Global Supply and Business Operations, at the Morgan Stanley 17th Annual Global Healthcare Conference on Monday, September 9, 2019 at 3:40 p.m. Eastern Daylight Time (Press release, Pfizer, SEP 3, 2019, View Source [SID1234539200]).

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!

To listen to the webcast, visit our web site at www.pfizer.com/investors. Information on accessing and pre-registering for the webcast will be available at www.pfizer.com/investors beginning today.

Visitors will be able to listen to an archived copy of the webcast at www.pfizer.com/investors.

On September 3, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, reported positive topline results from an investigator-sponsored Phase 2 trial of its lead drug candidate, tipifarnib, in patients with relapsed or refractory urothelial carcinomas that carry HRAS mutations (Press release, Kura Oncology, SEP 3, 2019, View Source [SID1234539198]).

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 ongoing, single-agent, single-arm trial is designed to enroll at least 18 patients, with a primary endpoint of progression-free survival (PFS) rate at 6 months. Secondary endpoints include objective response rate, duration of response and safety. The trial is being conducted at the Samsung Medical Center in Korea.

To date, more than 200 patients with relapsed or refractory urothelial carcinoma have been screened for the presence of tumor HRAS mutations. A total of 15 patients were identified to carry tumors with HRAS mutations. Two patients withdrew from the trial prior to their first response assessment. Of the 13 evaluable patients, five experienced confirmed objective responses, according to RECIST 1.1 criteria, for an overall response rate of 38%. Notably, four patients have experienced PFS of greater than 6 months. According to the trial protocol, the primary endpoint is met when at least four patients achieve PFS at 6 months.

"Although the treatment paradigm for advanced urothelial carcinoma has evolved with the introduction of checkpoint inhibitors, there remains a need for more precise and effective treatment options for these patients," said Se Hoon Park, M.D., Ph.D., Samsung Medical Center, principal investigator for the trial. "These biomarker-driven data in patients with relapsed or refractory urothelial carcinoma are promising and further underscore the potential for tipifarnib in HRAS mutant solid tumors."

All patients joined the trial upon progression from at least one prior systemic chemotherapy cycle, with a median of one prior therapy. Tipifarnib has been generally well-tolerated in the trial; adverse events observed are consistent with the known safety profile of tipifarnib. Further analyses of the trial are ongoing, and detailed data are expected to be presented at a future medical meeting.

"We are very encouraged to see that this investigator-sponsored trial in HRAS mutant urothelial carcinoma met its primary endpoint prior to the completion of enrollment," said Antonio Gualberto, M.D., Ph.D., Head of Development and Chief Medical Officer of Kura Oncology. "This study represents the fourth clinical proof-of-concept for tipifarnib and the second proof-of-concept in a HRAS mutant solid tumor indication. We believe that more than five percent of urothelial carcinoma patients carry the HRAS mutation, which represents a meaningful additional market opportunity for tipifarnib. Based on these results, we are currently evaluating next steps for tipifarnib in this indication and look forward to the presentation of the full data set at an upcoming medical meeting."

About Urothelial Carcinoma

Urothelial carcinoma, also known as transitional cell carcinoma, develops from urothelial cells that line the inside of the bladder. Urothelial carcinoma accounts for 90 percent of all bladder cancers, and can also arise in the renal pelvis and ureters. The American Cancer Society estimates approximately 80,470 new cases of bladder cancer in the United States for 2019. Despite the approval of checkpoint inhibitors in recent years, the treatment of patients with advanced urothelial carcinoma in the second-line setting remains a significant unmet need.

About Tipifarnib

Kura Oncology’s lead drug candidate, tipifarnib, is a potent, selective and orally bioavailable inhibitor of farnesyl transferase in-licensed from Janssen in December 2014. Previously, tipifarnib was studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets; however, no molecular mechanism of action had been determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next-generation sequencing as well as emerging information about cancer genetics and tumor biology, Kura is seeking to identify those patients most likely to benefit from tipifarnib. In November 2018, following an end of Phase 2 meeting with the U.S. Food and Drug Administration, Kura initiated its first registration-directed trial of tipifarnib in patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC). In addition to HRAS mutant HNSCC and HRAS mutant urothelial carcinoma, the Company has achieved clinical proof-of-concept with tipifarnib in angioimmunoblastic T-cell lymphoma and CXCL12-expressing peripheral T-cell lymphoma.