PHIO PHARMACEUTICALS TO PRESENT AT THE H.C. WAINWRIGHT 21ST ANNUAL HEALTHCARE CONFERENCE

On September 3, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, announced today that it will present at the H.C. Wainwright 21st Annual Healthcare Conference in New York City (Press release, Phio Pharmaceuticals, SEP 3, 2019, View Source [SID1234539207]).

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Dr. Gerrit Dispersyn, Dr. Med. Sc., President and CEO, will present an overview of the Company’s RNAi technology and an update on the Company’s development activities. The presentation will be held September 10, 2019, at 11:40 AM in the Kennedy II room (4th Floor) at the Lotte New York Palace Hotel. Dr. Dispersyn will also be available for investor and executive meetings throughout the conference.

The presentation will be webcast live and archived under the "Investors – Events and Presentations" section of the Company’s website, www.phiopharma.com.

BioLineRx Successfully Completes Dose-Escalation Part of Phase 1/2a Clinical Study for AGI-134, a Novel Immunotherapy for Treatment of Solid Tumors

On September 3, 2019 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported that it has successfully completed the dose-escalation part of the Phase 1/2a clinical study for AGI-134, a novel compound that evokes a direct anti-tumor response, as well as a vaccine effect, via a unique, multi-arm mechanism that targets patient-specific tumor neoantigens (Press release, BioLineRx, SEP 3, 2019, View Source [SID1234539206]). AGI-134 was found to be safe and well tolerated, with no serious drug-related adverse events or dose-limiting toxicities reported. The maximal tolerated dose was not reached and the recommended dose for part 2 of the study was determined.

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The ongoing Phase 1/2a study is a multicenter, open-label study expected to take place at approximately 15 sites in the US, UK and Israel. The objectives of the study are to evaluate the safety and tolerability of AGI-134 at the recommended dose in multiple solid tumor types, to evaluate a wide array of biomarkers, and to validate AGI-134’s mechanism of action. Furthermore, efficacy will be assessed by clinical and pharmacodynamic parameters. The dose-expansion part 2 of the study is expected to commence shortly.

Prof. Mark Middleton of the University of Oxford, the study’s principal investigator, stated, "We are pleased with these initial safety results of the first-in-human clinical trial assessing AGI-134 for the treatment of solid tumors. AGI-134 represents a new mechanistic class of cancer immunotherapies, with a unique and highly differentiated mode of action, harnessing pre-existing immune machinery to trigger a systemic anti-tumor response and create a pro-inflammatory tumor microenvironment. We expect the mechanistic assessments performed during the study to further elucidate and confirm AGI-134’s activity. These assessments are ongoing and will be extended during part 2 of the study."

"We are excited with the positive results of the first part of the Phase 1/2a of our second lead oncology asset," said Philip Serlin, Chief Executive Officer of BioLineRx. "Numerous pre-clinical studies to date have demonstrated that treatment with AGI-134 leads to regression of established primary tumors, prevents growth of untreated distal secondary tumors, and triggers a vaccine effect that may prevent the development of future metastases. Following the FDA’s recent IND approval for AGI-134, we plan to add sites in the US to the study, which is currently being conducted in the UK and Israel, by the first half of 2020. We are looking forward to initiating part 2 of the study shortly, with initial results expected by year-end 2020."

"In addition, we are also waiting with great anticipation for the upcoming top-line data for BL-8040’s COMBAT/KEYNOTE-202 trial in pancreatic cancer, which is running according to schedule and is expected to read out by the end of the year," added Mr. Serlin.

About AGI-134

AGI-134 is a synthetic alpha-Gal glycolipid in development for solid tumors that is highly differentiated from other cancer immunotherapies. AGI-134 is designed to label cancer cells with alpha-Gal via intra-tumoral administration, thereby targeting the body’s pre-existing, highly abundant anti-alpha-Gal (anti-Gal) antibodies and redirecting them to treated tumors. Binding of anti-Gal antibodies to the treated tumors results in activation of the complement cascade, which destroys the tumor cells and creates a pro-inflammatory tumor microenvironment that also induces a systemic, specific anti-tumor (vaccine) response to the patient’s own tumor neo-antigens.

AGI-134 has been evaluated in numerous pre-clinical studies. In a mouse melanoma model, treatment with AGI-134 led to regression of established primary tumors and suppression of secondary tumor (metastases) development. Synergy has also been demonstrated in additional pre-clinical studies when combined with an anti-PD-1 immune checkpoint inhibitor, offering the potential to broaden the utility of such immunotherapies, and improve the rate and duration of responses in multiple cancer types. AGI-134 was obtained by BioLineRx through the acquisition of Agalimmune Ltd.

Verastem Oncology Announces COPIKTRA™ (Duvelisib) Presentations at the Society of Hematologic Oncology 2019 Annual Meeting

On September 3, 2019 Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, reported that four posters highlighting clinical data for COPIKTRA (duvelisib) will be presented at the Society of Hematologic Oncology 2019 Annual Meeting taking place September 11-14, 2019, in Houston (Press release, Verastem, SEP 3, 2019, View Source [SID1234539205]). Three posters describe various analyses from the Phase 3 DUO study evaluating the efficacy and safety of COPIKTRA in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). The fourth poster describes efficacy and safety data from the Phase 2 DYNAMO evaluating COPIKTRA patients with refractory marginal zone lymphoma (MZL).

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"The data presented at SOHO this year highlight the outcomes from the Phase 3 DUO study in the labeled indication, as well as supplemental lymphocytosis data, and continue to support its ongoing use in patients with relapsed or refractory CLL/SLL," said Hagop Youssoufian, MSc, M.D., Head of Medical Strategy at Verastem Oncology. "These findings not only support the ongoing use of duvelisib in its labeled indication for patients with relapsed or refractory CLL/SLL, but they also suggest that therapy with duvelisib may offer promise warranting further investigation in patients with refractory MZL, for whom limited treatment options exist."

Dose modification data from the DUO trial in patients with relapsed or refractory CLL/SLL will also be presented.

"As we continually evaluate how to optimize the benefit-risk profile of cancer therapy, we have found that dose modifications may be used to manage adverse events while allowing patients to continue to benefit from treatment," commented Ian Flinn, MD, PhD, Director, Lymphoma Research at Sarah Cannon Research Institute and lead investigator of the DUO study. "The DUO trial looked at dosing modification data and the impact on patient response to duvelisib or progression-free-survival. We look forward to sharing these results as we continue to study more effective treatment approaches for patients with relapsed or refractory CLL/SLL."

COPIKTRA, a targeted oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, received approval as monotherapy from the U.S. Food and Drug Administration (FDA) in September 2018 for the treatment of patients with relapsed or refractory CLL/SLL after at least two prior therapies. COPIKTRA also received accelerated approval for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Continued approval in FL may be contingent upon verification and description of clinical benefit in confirmatory trials.

Details for the SOHO 2019 poster presentations are as follows:

Title: An improved benefit-risk profile of duvelisib in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who received ≥ 2 prior therapies
Lead author:Matthew Davids, Dana-Farber Cancer Institute
Poster No.: CLL-097
Date and time:Wednesday, September 11, 2019; 5:15 PM – 8:00 PM CDT
Location: Ballroom of Americas DEF

Title: Effect of dose modifications on response to duvelisib in patients with relapsed/refractory (R/R) CLL/SLL in the DUO trial
Lead author:Ian Flinn, Sarah Cannon Research Institute
Poster No.: CLL-029
Date and time:Wednesday, September 11, 2019; 5:15 PM – 8:00 PM CDT
Location: Ballroom of Americas DEF

Title: Patterns of duvelisib-induced lymphocytosis in patients with relapsed/refractory chronic lymphocytic leukemia/small lymphocytic lymphoma, including those with high-risk factors treated in the DUO trial
Lead author:Jacqueline Barrientos, Zucker School of Medicine at Hofstra/Northwell
Poster No.: CLL-096
Date and time:Wednesday, September 11, 2019; 5:15 PM – 8:00 PM CDT
Location: Ballroom of Americas DEF

Title: Characterization of duvelisib in patients with refractory marginal zone lymphoma: data from the phase 2 DYNAMO trial
Lead author:Eric Jacobsen, Dana-Farber Cancer Institute
Poster No.: IBCL-064
Date and time:Wednesday, September 11, 2019; 5:15 PM – 8:00 PM CDT
Location: Ballroom of Americas DEF

PDF copies of these poster presentations will be available here after the meeting.

Important Safety Information

WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS

Fatal and/or serious infections occurred in 31% of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% of COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.
WARNINGS AND PRECAUTIONS

Infections: Serious, including fatal (4%), infections occurred in 31% of patients receiving COPIKTRA (N=442). The most common serious infections were pneumonia, sepsis, and lower respiratory infections. Median time to onset of any grade infection was 3 months, with 75% of cases occurring within 6 months. Treat infections prior to initiation of COPIKTRA. Advise patients to report new or worsening signs and symptoms of infection. Cases of Pneumocystis jirovecii pneumonia (PJP) (1%) and cytomegalovirus (CMV) reactivation/infection (1%) occurred in patients taking COPIKTRA. Provide prophylaxis for PJP during treatment and following completion of treatment until the absolute CD4+ T cell count is greater than 200 cells/µL. Consider prophylactic antivirals during COPIKTRA treatment to prevent CMV infection including CMV reactivation.

Diarrhea or Colitis: Serious, including fatal (<1%), diarrhea or colitis occurred in 18% of patients receiving COPIKTRA (N=442). Median time to onset of any grade diarrhea or colitis was 4 months, with 75% of cases occurring by 8 months. The median event duration was 0.5 months. Advise patients to report any new or worsening diarrhea.

Cutaneous Reactions: Serious, including fatal (<1%), cutaneous reactions occurred in 5% of patients receiving COPIKTRA (N=442). Fatal cases included drug reaction with eosinophilia and systemic symptoms (DRESS) and toxic epidermal necrolysis (TEN). Median time to onset of any grade cutaneous reaction was 3 months with a median event duration of 1 month. Presenting features for the serious events were primarily described as pruritic, erythematous, or maculo-papular. Less common presenting features include exanthem, desquamation, erythroderma, skin exfoliation, keratinocyte necrosis, and papular rash. Advise patients to report new or worsening cutaneous reactions.

Pneumonitis: Serious, including fatal (<1%), pneumonitis without an apparent infectious cause occurred in 5% of patients receiving COPIKTRA (N=442). Median time to onset of any grade pneumonitis was 4 months with 75% of cases occurring within 9 months. The median event duration was 1 month with 75% of cases resolving by 2 months.

Hepatotoxicity: Grade 3 and 4 ALT and/or AST elevation developed in 8% and 2%, respectively, of patients receiving COPIKTRA (N=442). Two percent of patients had both an ALT or AST > 3 X ULN and total bilirubin > 2 X ULN. Median time to onset of any grade transaminase elevation was 2 months with a median event duration of 1 month. Monitor hepatic function during treatment with COPIKTRA.

Neutropenia: Grade 3 or 4 neutropenia occurred in 42% of patients receiving COPIKTRA (N=442), with Grade 4 neutropenia occurring in 24% of all patients. Median time to onset of grade ≥3 neutropenia was 2 months. Monitor neutrophil counts at least every 2 weeks for the first 2 months of COPIKTRA therapy, and at least weekly in patients with neutrophil counts < 1.0 Gi/L (Grade 3-4).

Embryo-Fetal Toxicity: COPIKTRA can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus and conduct pregnancy testing before initiating COPIKTRA treatment. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment and for at least 1 month after the last dose.

ADVERSE REACTIONS

B-cell Malignancies Summary

Fatal adverse reactions within 30 days of the last dose occurred in 8% (36/442) of patients treated with COPIKTRA 25 mg BID. Serious adverse reactions were reported in 289 patients (65%). The most frequent serious adverse reactions that occurred were infection (31%), diarrhea or colitis (18%), pneumonia (17%), rash (5%), and pneumonitis (5%). The most common adverse reactions (reported in ≥20% of patients) were diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain and anemia.

CLL/SLL

Fatal adverse reactions within 30 days of the last dose occurred in 12% (19/158) of patients treated with COPIKTRA and in 4% (7/155) of patients treated with ofatumumab. Serious adverse reactions were reported in 73% (115/158) of patients treated with COPIKTRA and most often involved infection (38%; 60/158) and diarrhea or colitis (23%; 36/158). The most common adverse reactions with COPIKTRA (≥20% of patients) were diarrhea or colitis, neutropenia, pyrexia, upper respiratory tract infection, pneumonia, rash, fatigue, nausea, anemia and cough.

For specific information on the management of the adverse reactions above, please review Dose Modifications for Adverse Reactions within the full Prescribing Information.

DRUG INTERACTIONS

CYP3A Inducers: Coadministration with a strong CYP3A inducer may reduce COPIKTRA efficacy. Avoid coadministration with strong CYP3A4 inducers.

CYP3A Inhibitors: Coadministration with a strong CYP3A inhibitor may increase the risk of COPIKTRA toxicities. Reduce COPIKTRA dose to 15 mg BID when coadministered with a strong CYP3A4 inhibitor.

CYP3A Substrates: Coadministration of COPIKTRA with sensitive CYP3A4 substrates may increase the risk of toxicities of these drugs. Consider reducing the dose of the sensitive CYP3A4 substrate and monitor for signs of toxicities of the coadministered sensitive CYP3A substrate.

To report Adverse Reactions, contact FDA at 1-800-FDA-1088 (1-800-332-1088) or www.fda.gov/medwatch and Verastem Oncology at 1-877-7RXVSTM (1-877-779-8786).

Please see accompanying full Prescribing Information, including Boxed Warning.

About Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are cancers that affect lymphocytes and are essentially the same disease, with the only difference being the location where the cancer primarily occurs. When most of the cancer cells are located in the bloodstream and the bone marrow, the disease is referred to as CLL, although the lymph nodes and spleen are often involved. When the cancer cells are located mostly in the lymph nodes, the disease is called SLL. The symptoms of CLL/SLL include a tender, swollen abdomen and feeling full even after eating only a small amount. Other symptoms can include fatigue, shortness of breath, anemia, bruising easily, night sweats, weight loss, and frequent infections. However, many patients with CLL/SLL will live for years without symptoms. There are approximately 200,000 patients in the US affected by CLL/SLL with nearly 20,000 new diagnoses this year alone. While there are therapies currently available, real-world data reveals that a significant number of patients either relapse following treatment, become refractory to current agents, or are unable to tolerate treatment, representing a significant medical need. The potential of additional oral agents, particularly as a monotherapy that can be used in the general community physician’s armamentarium, may hold significant value in the treatment of patients with CLL/SLL.

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed by Verastem Oncology for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track status, and is being investigated in combination with other agents through investigator-sponsored studies.4 For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

argenx to Present at Morgan Stanley 17th Annual Global Healthcare Conference

On September 3, 2019 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported that Tim Van Hauwermeiren, Chief Executive Officer, will present at the Morgan Stanley 17th Annual Global Healthcare Conference on Monday, September 9, 2019 at 10:30 am ET in New York (Press release, argenx, SEP 3, 2019, View Source [SID1234539204]).

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The live webcast may be accessed on the homepage of the argenx website at www.argenx.com. Shortly after the presentation, a replay of the webcast will be available for 90 days on the argenx website.

Santhera Announces Financial Results for the First Half-Year 2019

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]).

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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