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On December 12, 2018 Santhera Pharmaceuticals (SIX: SANN) reported its financial results for the nine months ended September 30, 2018, and confirms its guidance and positive outlook (Press release, Santhera Pharmaceuticals, DEC 12, 2018, View Source [SID1234532027]).

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Santhera is on track to meet its strategic and financial goals for 2018. The Company further delivered on its active in-licensing strategy for high quality, late-stage rare disease assets by acquiring an exclusive sub-license option to vamorolone, the first-in-class dissociative steroid currently in development for the treatment of Duchenne muscular dystrophy (DMD). On the commercial side, the Company has further grown sales of the revenue-generating product Raxone for the treatment of Leber’s hereditary optic neuropathy (LHON) and expects to meet its 2018 full-year sales guidance in the range of CHF 30-32 million.

Santhera continued its business expansion and grew sales to CHF 23.6 million in the first nine months of 2018 which corresponds to a 45% growth rate (compared to the same period in the previous year). Development expenses increased by 49% compared to the same prior-year period, primarily driven by clinical development activities for POL6014 for the treatment of cystic fibrosis (CF) and regulatory work in preparation of filings for idebenone in the indication DMD. Efficient use of resources resulted in lower marketing and sales expenses (-6%) and a slower rise in general and administrative expenses (+18%). Taken together, this contributed to an increase of operating expenses by 20% compared to the same period of last year. In summary, Santhera closed the nine-month period under review with a net result of CHF -39.9 million (Jan.-Sept. 2017: CHF -33.3 million).

Financial highlights:

Sales of CHF 23.6 million (Jan.-Sept. 2018), reflecting an increase by 45% compared to same period last year (Jan.-Sept. 2017: CHF 16.3 million)
Operating expenses of CHF 57.2 million (Jan.-Sept. 2017: CHF 47.7 million)
Operating result of CHF -37.2 million (Jan.-Sept. 2017: CHF -34.2 million), leading to a net result of CHF -39.9 million (Jan.-Sept. 2017: CHF -33.3 million)
Cash, cash equivalents and short-term financial assets of CHF 25.4 million (as of September 30, 2018)
2018 full-year sales guidance of CHF 30-32 million confirmed
Corporate highlights:

Agreement with Idorsia Ltd under which Santhera is to acquire the option to an exclusive sub-license of the first-in-class dissociative steroid vamorolone in all indications and all countries worldwide except Japan and South Korea (November 20, 2018)
Idorsia became largest shareholder in Santhera
Extraordinary General Meeting (EGM) approval of the capital increase in connection with the vamorolone sub-license agreement (December 11, 2018)
Operational highlights (July 2018 to present day):

Analysis of new data linking study findings with idebenone in DMD to clinically relevant patient benefits for inclusion in regulatory submissions in Europe and the U.S.
Positive opinion on orphan drug designation received from European regulators for POL6014 for the treatment of CF
Start of a Phase Ib/IIa multiple ascending dose (MAD) trial with POL6014 in patients with CF
Revenue Guidance:
Santhera will continue to grow its international business, advance its pipeline programs and actively proceed with business development initiatives to expand its late stage product portfolio. Based on its sales performance to date this year, the Company confirms its previous guidance and expects to reach a full-year sales for Raxone in the range of CHF 30-32 million in 2018.

Note
The financial statements as of and for the nine months ended September 30, 2018 have been prepared and are being published exceptionally on the occasion of and in connection with the capital increase proposed to and approved by the EGM held on December 11, 2018. Santhera publishes results in line with the disclosure requirements of the SIX Swiss Exchange. Santhera does not plan on publishing quarterly reports in the future.

On December 12, 2018 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number: 4875), reported that Yuichi Iwaki, MD, PhD, President and Chief Executive Officer, and Geoffrey O’Brien, JD/MBA, Vice President and Executive Officer, will present a corporate overview at the J.P. Morgan Healthcare Conference on Wednesday, January 9, 2019 at 2:00 pm at the Westin St. Francis Hotel in San Francisco, California (Press release, MediciNova, DEC 12, 2018, View Source;p=irol-newsArticle&ID=2380360 [SID1234532029]). MediciNova’s management team, including Dr. Yuichi Iwaki, Geoffrey O’Brien, and Kazuko Matsuda, MD, PhD, MPH, Chief Medical Officer, will be available for one-on-one meetings at this conference and investors may request a one-on-one meeting through J.P. Morgan.

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On December 12, 2018 Zai Lab Limited (NASDAQ: ZLAB), a Shanghai-based innovative commercial stage biopharmaceutical company, reported that the China National Medical Products Administration (NMPA) has accepted its New Drug Application (NDA) for ZEJULA (niraparib, or ZL-2306) as a Category 1 drug 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 (Press release, Zai Laboratory, DEC 12, 2018, View Source [SID1234532051]). ZEJULA is a potent and highly selective PARP1/2 inhibitor that does not require BRCA mutation or other biomarker testing prior to administration.

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"The NMPA’s acceptance of our NDA submission for ZEJULA represents a major milestone for Zai Lab as this is our first ever NDA submission in Mainland China," said Dr. Samantha Du, Founder and Chief Executive Officer of Zai Lab. "ZEJULA will offer an important, new treatment option to more than 50,000 Chinese patients who suffer from ovarian cancer every year and we are grateful that NMPA recognizes this critical medical need and the promise of ZEJULA. Zai is a leader in the field of innovative oncology treatments in China and we have a deep and highly-differentiated pipeline, including three U.S. FDA-approved products and four other assets in late stage clinical development. As a result, we expect additional regulatory submissions in the coming years as we continue to advance our pipeline."

Dr. Yong-Jiang Hei, Chief Medical Officer for Oncology of Zai Lab said, "We believe ZEJULA is a best-in-class PARP inhibitor due to its compelling efficacy, once-daily dosing and superior pharmacokinetic properties including its ability to cross the blood brain barrier. The NDA submission based on the Category 1 designation of ZEJULA is a result of China-based clinical trials and manufacturing conducted by Zai Lab. We plan to expand our development efforts in collaboration with our partner Tesaro across several additional indications including, but not limited to, first-line maintenance treatment of ovarian cancer, lung cancer and gastric cancer."

William Liang, Chief Commercial Officer noted, "The Zai Lab commercial team is very excited about the attractive profile of ZEJULA and plans to leverage ZEJULA’s recent Hong Kong approval and commercial launch to prepare for the launch in China. If approved, we believe ZEJULA will provide a differentiated treatment option to benefit more ovarian cancer patients. We also intend to closely collaborate with local authorities and NGOs to develop patient assistant programs to increase access to more women who could benefit from this treatment. Zai Lab is committed to making a meaningful impact on the way cancer is treated in China and will continue to develop and bring new innovative oncology treatment options to patients in need."

About Ovarian Cancer

Ovarian cancer is one of the most common gynecologic cancers in China with approximately 51,000 newly diagnosed cases and 23,000 deaths in China in 2014. The 5-year overall survival rate of ovarian cancer patients is 46% across all stages, but only 29% in patients are diagnosed with distant metastatic disease. While platinum-based chemotherapy is effective at inducing an initial response in ovarian cancer, the disease will recur in the majority of women. Effective treatment options for patients with platinum-sensitive recurrent ovarian cancer remain limited. New agents that prolong the duration of response following platinum-based treatment and delay the inevitable relapse of ovarian cancer will benefit patients with ovarian cancer in China.

About ZEJULA

ZEJULA (niraparib, ZL-2306) is a highly potent and selective oral, once-daily small molecule poly (ADP-ribose) PARP 1/2inhibitor. It was approved in March 2017 by the FDA in the United States and in November 2017 by the EMA in the European Union under the trade name ZEJULA as a maintenance treatment for women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. Based on the approval status in the United States and European Union by our partner, Tesaro, Zai Lab has obtained the approval for marketing ZEJULA in Hong Kong in October 2018.

On December 12, 2018 OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing novel cancer immunotherapies, reported an update regarding tumor responses in its KEYNOTE-695 global, multicenter Phase 2b, open-label trial of intratumoral delivery of TAVO (tavokinogene telseplasmid / IL-12) with intravenous KEYTRUDA (pembrolizumab) in patients with unresectable, advanced melanoma (Press release, OncoSec Medical, DEC 12, 2018, View Source [SID1234532030]). The Company previously reported at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 33rd Annual Meeting (SITC) (Free SITC Whitepaper) that, of the first nine patients to complete 12 weeks of treatment and reach initial tumor evaluation (by RECIST v1.1), two patients had a partial response. The Company now reports that both responses have been confirmed by blinded independent review at the first assessment timepoint. Further, in both cases, response duration has now reached six months. Importantly, one of these previously assessed partial responses has now become a complete response per investigator assessment at the six-month tumor evaluation timepoint. Enrollment is ongoing and the Company expects to complete the study next year.

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"Complete responses in this patient population are rare. One of the first partial responses to be observed in this study now being assessed by the investigator at six months as a complete response is exciting. Durable responses reaching the six-month mark demonstrate that the benefits of TAVO are clinically meaningful," said Daniel J. O’Connor, President and CEO of OncoSec. "The evidence of the potential of TAVO in conjunction with PD-1 inhibition to improve outcomes in this patient population is mounting and we look forward to providing updates as necessary regarding the progress of this trial."

KEYNOTE-695 enrollment criteria with respect to anti-PD-1 checkpoint failure is highly restrictive. In order to be considered an anti-PD-1 checkpoint failure, all patients must have Stage III/IV metastatic melanoma progressive disease after at least four prior cycles of either KEYTRUDA or OPDIVO. Literature suggests that retreatment with a PD-1 therapy following failure of a PD-1 therapy offers approximately a 5% response rate1. Disease progression is determined according to RECIST v1.1, measured by radiologic assessment, with confirmation of progression by second assessment.

"It’s becoming clear that TAVO can reverse PD-1 resistance in refractory metastatic melanoma patients. This is important because the majority of melanoma patients do not respond to PD-1 treatment," said Adil Daud, MD, HS Clinical Professor, Department of Medicine (Hematology/Oncology), UCSF; Director, Melanoma Clinical Research, UCSF Helen Diller Family Comprehensive Cancer Center and Lead Principle Investigator of KEYNOTE-695. "Patients in KEYNOTE-695 have unequivocally failed all approved anti-PD-1 therapies. The tumor responses seen in this trial, now independently verified by a blinded review at the first assessment, deepening and continuing for months, gives us confidence in the potential of this treatment combination for patients who are non-responsive to anti-PD-1 therapy."

Eligible patients in KEYNOTE-695 had refractory, locally advanced or metastatic disease defined as unresectable Stage III/IV metastatic melanoma that had definitively progressed on a full-course of anti-PD-1 treatment with KEYTRUDA (pembrolizumab) or OPDIVO (nivolumab). As previously reported, TAVO was well-tolerated, with primarily Grade 1 adverse advents associated with injection site or procedural pain. One TAVO related Grade 3 SAE of cellulitis was reported and resolved. KEYNOTE-695 is expected to be completed in 2019. Based on the outcome of the study, the Company plans to file for accelerated approval by end of 2019 or early 2020.

TAVO has received both Orphan Drug and Fast-Track Designation by the U.S. Food & Drug Administration.

1 Long GV, Weber JS, Larkin J et al. Nivolumab for patients with advanced melanoma treated beyond progression: analysis of 2 Phase 3 clinical trials. JAMA Oncol. 3(11), 1511–1519 (2017).