On August 29, 2019 Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LTB4) systems are implicated, reported financial results for the second quarter ended June 30, 2019 and recent clinical progress (Press release, Akari Therapeutics, AUG 29, 2019, View Source [SID1234539160]).

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"We are pleased with the progress we have made advancing our BP, HSCT-TMA and AKC programs and are encouraged by the initial data we have received to date in these programs," said Clive Richardson, Chief Executive Officer of Akari Therapeutics. "Both AKC and BP have further planned clinical readouts this year, providing a potential opportunity for advancing both programs into pivotal trials in 2020 and further supporting the novel therapeutic role of combined C5 and LTB4 treatment. In addition, we are planning to start a pivotal clinical trial for HSCT-TMA in the fourth quarter of this year."

Second Quarter 2019 and Recent Business Highlights

Pediatric HSCT-TMA
The Company continues to progress towards a pivotal trial for HSCT-TMA with nomacopan, which is expected to start in the fourth quarter of 2019. This condition has an estimated 80% mortality rate in children with this severe disease, with currently no approved treatments. In a March 2019 meeting, a framework for the trial design was agreed with the U.S. Food and Drug Administration (FDA). In August 2019, the FDA granted Fast Track designation for nomacopan for the treatment of HSCT-TMA in pediatric patients.
Phase II clinical trial in patients with BP
Initial results from the first three patients with mild-to-moderate BP in the ongoing Phase II trial with nomacopan demonstrated a rapid reduction in BP Disease Area Index (BPDAI) score and blistering of 52% and 87%, respectively, by day 42. There were no drug related serious adverse events. The Company anticipates new safety and efficacy data in mild-to-moderate patients from this study to be given as an oral presentation at the the 28th European Academy of Dermatology and Venereology (EADV) Congress, October 10, 2019.
In early August, the Company announced new data demonstrating synergistic benefits of nomacopan’s dual C5 and LTB4 inhibitory activity in pemphigoid disease, generated by Dr. Christian Sadik’s group at University of Lubeck, Germany, and published in the August 2019 edition of JCI Insight [link].
Phase I/II clinical trial in patients with AKC
Successfully completed Part A of TRACKER, a Phase I/II clinical trial evaluating the safety and efficacy of topical nomacopan in patients with moderate-to-severe AKC. Results showed a rapid response and an overall improvement of 55% in the composite clinical score, which was composed of an improvement in symptoms of 62% and signs of 52% by Day 56. Three patients were treated with twice daily nomacopan eye drops in addition to standard of care for up to 56 days, with one patient completing 14 days and then withdrawing for reasons unrelated to the study. All patients had been on maximal topical cyclosporine, the standard of care, for at least three months prior to entry. The nomacopan eye drops were found to be comfortable and well tolerated with no serious adverse events. Enrollment in the Part B placebo-controlled efficacy arm in 16 patients continues to progress, with data read out planned for the fourth quarter of 2019.
Clive Richardson has been appointed permanent Chief Executive Officer of Akari after having served as interim Chief Executive Officer since May, 2018
Upcoming Events and Milestones

HSCT-TMA pivotal clinical trial expected to start in the fourth quarter of 2019.
Mild-to-moderate BP trial data to be presented at EADV Congress, October 10, 2019.
Completion of Part B of AKC Phase I/II trial by the fourth quarter of 2019.
Second Quarter 2019 Financial Results

Research and development (R&D) expenses in the second quarter of 2019 were $3.6 million, as compared to R&D expenses of $5.1 million in the same quarter the prior year. This decrease was primarily due to lower manufacturing expenses as the Company had previously manufactured clinical trial material for supply through 2019, which was slightly offset by higher clinical trial costs and personnel expenses. R&D expenses for the six months ended June 30, 2019 were $1.3 million reflecting the receipt of a Q1 R&D tax credit of $4.9 million.
General and administrative (G&A) expenses in the second quarter of 2019 were $2.4 million, as compared to $2.9 million in the same quarter last year. This decrease was primarily due to lower expenses associated with professional services, personnel and rent, partially offset by higher stock-based non-cash compensation expenses.
Total other income for the second quarter of 2019 was $1.9 million, as compared to total other expense of $43,000 in the same period the prior year. This change was primarily due to $2.0 million of higher income related to the change in the fair value of the stock option liabilities in 2019 compared to 2018, and to higher foreign exchange gains of approximately $39,000 in 2019 as compared to 2018.
Net loss for the second quarter of 2019 was $4.1 million, compared to a net loss of $8.0 million for the same period in 2018. The decrease in net loss in the second quarter of 2019 was due primarily to the change in the fair value of the stock option liabilities and foreign exchange gains previously cited, accompanied by lower operating expenses in the second quarter of 2019.
As of June 30, 2019, the Company had cash of $2.7 million, as compared to cash of $5.4 million as of December 31, 2018. On July 3, 2019, the Company sold to certain institutional investors, accredited investors and an existing shareholder, RPC Pharma Ltd., an affiliated entity of Dr. Ray Prudo, Akari’s Chairman, an aggregate 2,368,392 registered American Depository Shares (ADSs) of Akari at a purchase price of $1.90 per ADS, resulting in gross proceeds of approximately $4.5 million. Additionally, for each ADS purchased by investors, the investors received an unregistered warrant to purchase one-half ADS. The warrants have an exercise price of $3.00 per ADS, were exercisable upon their issuance and will expire five years from the issuance date.
As of June 30, 2019, the Company has sold to Aspire Capital Fund, LLC (Aspire Capital) a total of $2.0 million of ordinary shares. Subsequent to June 30, 2019, the Company sold to Aspire Capital a further $3.5 million of ordinary shares and approximately $14.5 million remains available for draw down under the purchase agreement entered into with Aspire Capital.

On August 29, 2019 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported the expanded use of the VENTANA PD-L1 (SP142) Assay3,4 in triple-negative breast cancer (TNBC) for patients living in CE (Conformité Européenne) markets where the Roche cancer immunotherapy medicine Tecentriq is approved (Press release, Hoffmann-La Roche, AUG 29, 2019, View Source [SID1234539108]). It is the first companion diagnostic to aid in identifying triple-negative breast cancer patients eligible for treatment with Tecentriq (atezolizumab)5 plus chemotherapy (nab-paclitaxel).6 Assessment of PD-L1 biomarker status on tumour-infiltrating immune cells with the assay is essential for identifying those patients most likely to benefit from the treatment.

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Today’s announcement follows the U.S. Food and Drug Administration approval of the assay in March 2019 as the first companion diagnostic to identify triple-negative breast cancer patients eligible for the Tecentriq combination. A diagnosis of triple-negative breast cancer means that the three most common proteins associated with breast cancer growth – estrogen receptor, progesterone receptor and HER2/neu – are not expressed on the tumour.

"Until recently, the only treatment option for metastatic triple-negative breast cancer patients was chemotherapy," said Thomas Schinecker, Head of Roche Diagnostics. "With our expanding menu of companion diagnostics and targeted cancer immunotherapies, Roche is proud to continue to deliver on our mission to make personalised healthcare a global reality, ensuring the right treatment for the right patient at the right time."

The VENTANA PD-L1 (SP142) Assay was developed to enhance visual contrast of tumour-infiltrating immune cell staining. In triple-negative breast cancer, PD-L1 is primarily expressed on tumour-infiltrating immune cells rather than on tumour cells themselves.7

Launched in 2016, the VENTANA PD-L1 (SP142) Assay is the primary diagnostic assay within the Tecentriq clinical development program and was used to enroll and stratify patients in Tecentriq clinical trials. It was the enrollment assay used in the IMpassion130 trial, the first positive phase III immunotherapy regimen study in triple-negative breast cancer. The assay was the first to evaluate patient PD-L1 biomarker status using immune cell staining and scoring within the tumour microenvironment.8

About the VENTANA PD-L1 (SP142) Assay
The VENTANA PD-L1 (SP142) Assay is available on the fully automated BenchMark IHC/ISH series instruments and uses the OptiView DAB IHC Detection Kit with OptiView Amplification Kit. The VENTANA PD-L1 (SP142) Assay performs specific staining of tumour cells and immune cells. The assay was previously approved by the FDA and CE marked for use as a companion diagnostic in urothelial carcinoma (UC)5 and as a predictive assay in non-small cell lung cancer (NSCLC) with Tecentriq. See the Tecentriq product label for more information on PD-L1 expression levels in therapeutic guidance for various cancer indications.

About the IMpassion130 study
The IMpassion130 study is a phase III, multicenter, randomized, double-blind study evaluating the efficacy, safety and pharmacokinetics of Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel in people with unresectable locally advanced or metastatic triple-negative breast cancer who have not received prior systemic therapy for metastatic breast cancer (mBC). For details of the study go to www.roche.com.

About Tecentriq
Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1, which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T cells. Tecentriq is a cancer immunotherapy that has the potential to be used as a foundational combination partner with other immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers. The development of Tecentriq and its clinical programme is based on our greater understanding of how the immune system interacts with tumours and how harnessing a person’s immune system combats cancer more effectively.

Tecentriq is approved in the US, EU and countries around the world, either alone or in combination with targeted therapies and/or chemotherapies in various forms of non-small cell and small cell lung cancer, certain types of metastatic urothelial cancer, and in PD-L1-positive triple-negative breast cancer.

On August 29, 2019 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 MediciNova will attend the Morgan Stanley Global Healthcare Conference which will be held at the Grand Hyatt New York in New York City from September 9 – 11, 2019 (Press release, MediciNova, AUG 29, 2019, View Source [SID1234539146]). Yuichi Iwaki, MD, PhD, President and Chief Executive Officer, will be available for one-on-one meetings at this conference and investors may request a one-on-one meeting through Morgan Stanley.

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On August 29, 2019 Vivoryon Therapeutics AG (Euronext Amsterdam: VVY), reported its financial results for the first six months of 2019 ending June 30 (Press release, Vivoryon Therapeutics, AUG 29, 2019, View Source [SID1234539140]). The full interim report is available on the company website (View Source).

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KEY HIGHLIGHTS
Probiodrug AG and the Alzheimer’s Disease Cooperative Study (ADCS) received USD 15 million grant from NIH for US-based Phase 2b trial
Probiodrug raised EUR 8.2 million from investors in successful private placement of new shares
Probiodrug AG became Vivoryon Therapeutics AG
Shareholder’s meeting resolved for a capital increase to be implemented before the end of the year 2019
Vivoryon entered into a collaboration with University of Kiel to select candidates from QPCTL inhibitor portfolio

POST PERIOD HIGHLIGHTS
Business development efforts in the first half year 2019 led to the announcement of a strategic collaboration with MorphoSys in July for the development of QPCTL technology in immuno-oncology. Vivoryon entered into an exclusive Option Agreement for an up to EUR 15 million equity commitment.

Comment from Dr. Ulrich Dauer, Chief Executive Officer of Vivoryon Therapeutics:

The last six months stand as a transformative time period for Vivoryon during which the team strategically advanced and repositioned the Company through the achievement of several noteworthy corporate goals including the licensing agreement with MorphoSys for our QPCTL inhibitors in oncology, in addition to the validation of our Alzheimer’s Disease program from the NIH. Having collaborated with the management team to redefine our objectives and the most optimal steps to achieve them, we remain well-positioned to reach the next set of corporate and clinical milestones.

Looking back at the first half of 2019, we strengthened three core elements of the Company: our corporate and financial position, the recognition of our potential in oncology and the validation of our approach in Alzheimer’s Disease.

Starting with the completion of a successful private placement, we enhanced our financial standing and added a consortium of new strategic investors to our shareholder base. On top of this, we rebranded and redefined our position as a company. Our new name, "Vivoryon Therapeutics AG," highlights our strong commitment to developing a treatment for patients suffering from Alzheimer’s Disease and emphasizes the fresh perspectives and momentum we aim to leverage to efficiently advance the Company.

From a pipeline perspective, we have made quantifiable progress in monetizing the opportunity within our approach in immuno-oncology. This was emphasized by our licensing agreement with MorphoSys, which delivered on our promise to combine our unique portfolio of proprietary small molecule QPCTL inhibitors with a leading antibody technology. In addition to this, we also initiated a research collaboration with the University of Kiel to further uncover the therapeutic potential of our QPCTL inhibitors in cellular cancer models.

In regard to our Alzheimer’s program, we received the approval of a USD 15 million NIH grant intended for a US Phase 2b trial, further validating our differentiated therapeutic approach to this detrimental neurodegenerative disease. Our lead candidate, PQ912, stands out from other treatments in the field, as it inhibits the production of the pyroglutamated form of abeta, a neuro- and synaptotoxic driver of Alzheimer’s initiation and progression. Our aim is to advance the Phase 2b clinical trial in Europe during the first quarter of 2020.

As we enter into the second half of the year, we will continue to finalize preparations for the Alzheimer’s clinical trial, explore the potential of our unique proprietary position in cancer and identify additional opportunities within our discovery and development pipeline of small molecule therapeutics. With our near-term objective focused on securing the required financial runway to meet these goals, we look forward to connecting with potential investors and partners in the global biotechnology industry and sharing our revitalized vision of leveraging our versatile scientific approach to advance drug candidates in complex and large medical need indications.

Details of the Financial Results (according to IFRS)

Net loss

The operating loss for first half of 2019 was reduced by 26% to EUR 3,077k (H1 2018: EUR 4,133k). This was driven by lower research and development expenses of EUR 1,862k (H1 2018: EUR 2,572k) and lower general and administrative of EUR 1,223k (H1 2018: EUR 1,578k).

Consequently, net loss was reduced to EUR 3,091k (H1 2018: EUR 4,120k).

All results are in line with management expectations.

Cash

Vivoryon Therapeutics held EUR 7,999k in cash and cash equivalents as of June 30, 2019 (Dec 31, 2018: EUR 3,783k).

The cash flow from financing activities in amount of EUR 7,644k resulted from the successful private placement of new shares that raised EUR 8,187k in gross proceeds, which were offset by the associated transaction costs of EUR 523k.

VIVORYON THERAPEUTICS’ CURRENT VISION
With 20+ years of unmatched understanding in identifying protein-modifying enzymes that play a critical role in disease initiation and progression, Vivoryon Therapeutics scientific expertise has facilitated the creation of a discovery and development platform for small molecule therapeutics.

Having demonstrated success by developing a novel therapeutic in type 2 diabetes, the Company is advancing their lead candidate, PQ912, in Alzheimer’s Disease and their entire portfolio of QPCT and QPCTL inhibitors in oncology.

NEW THERAPEUTIC APPROACH
PQ912 in Alzheimer’s Disease

PQ912 is a first-in-class inhibitor of the QC enzyme that addresses a very distinct disease pathway and provides a different mode of action in contrast to other available Alzheimer’s Disease programs in development. Positive results from a Phase 2a clinical trial published in June 2017 demonstrated a significant improvement in the synaptic function of PQ912-treated Alzheimer’s patients versus a control group in addition to measurable improvements in the memory performance of patients treated with PQ912, despite the short treatment time of only twelve weeks.

The upcoming Phase 2b European, multicenter study, "SAPHIR," will be conducted over 56 weeks on average and will test varying doses of PQ912. Trial initiation is anticipated in the first quarter of 2020 with the topline results expected in the second half of 2022.

The SAPHIR clinical trial in Europe is led by Professor Philip Scheltens, Director of the Alzheimer Center VU University Medical Center Amsterdam, NL. A second complementary trial, so called US trial, is in the planning phase and will be run by Professor Howard Feldman, Director of Alzheimer’s Disease Cooperative Study (ADCS), in San Diego, USA. The National Institutes of Health (NIH) is funding, in part, the US Phase 2b core program with an NIH Research Project grant expected to total of USD 15 million over four years.

QPCTL Inhibitors in Oncology and Beyond

Recently published results as well as internal research have shown that the Glutaminyl-peptide cyclotransferase-like (QPCTL) is an interesting therapeutic target to silence the "do not eat me" signal provided by the interaction of CD47 (expressed on cancer cells), with the protein SIRP-alpha (expressed on macrophages and other myeloid cells). Tumor immunotherapy that targets this interaction is a current focus of innovation in cancer drug development. Combining a therapeutic tumor-targeted antibody of choice with the inhibition of the CD47/SIRP-alpha interaction is expected to lead to significant therapeutic improvements. By possessing the broadest portfolio of small molecule QPCTL inhibitors and the clinically most advanced compounds in that field, Vivoryon is uniquely positioned to address this attractive target in immuno-oncology. QPCTL inhibitors are expected to have considerable therapeutic advantages compared to antibody approaches that are currently explored in clinical studies to silence the CD47/SIRP-alpha interactions.

Based on Vivoryon Therapeutics’ data, PQ912 could be advanced into clinical Phase I studies in cancer. In addition, the Company entered into an exclusive Option Agreement with MorphoSys on small molecule inhibitors of QPCTL, silencing the CD47-SIRP alpha signaling in immuno-oncology. Vivoryon Therapeutics owns a broader set of highly promising QPCTL inhibitor compounds in advanced preclinical stages of development.

Within the collaboration of the University of Kiel, Vivoryon Therapeutics will also fund focused research with the clear goal of further validating its QPCTL inhibitors in cellular cancer models. The Company’s highly active compounds will be tested individually and in combination with therapeutic antibodies.

CONFERENCE CALL
Vivoryon Therapeutics will host a conference call and webcast open to the public today, August 29, 2019 at 3:00 pm CEST / 09:00 am EST; the presentation will also be available on the company website. The conference will be held in English. A question & Answer session will follow the presentation of the Half year results.

To participate in the conference call, please call one of the following numbers 10 minutes prior to commencement.

A live webcast and slides will be made available at: www.vivoryon.com/investors-news/financial-information/

Approximately a day after the call, a slide-synchronized audio replay of the conference will be available on: www.vivoryon.com/investors-news/financial-information/

FINANCIAL STATEMENTS

January to June 2019
Vivoryon Therapeutics has finalized its financial statements for the first six months of 2019 according to German GAAP ("HGB") and IFRS. The auditor, KPMG, has reviewed the IFRS statements. The reports are available on the company website (View Source).

Financial calendar 2019
November 28, 2019 Interim Management Statement Q3 2019

For more information, please contact:

Vivoryon Therapeutics AG

Dr. Ulrich Dauer, CEO
Email: [email protected]

MC Services AG
Anne Hennecke, Susanne Kutter
Tel: +49 (0) 211 529 252 27
Email: [email protected]

Trophic Communication
Gretchen Schweitzer, Joanne Tudorica
Tel.: +49 172 861 8540 / +49 176 2103 7191
Email: [email protected] / [email protected]

On August 29, 2019 Bausch Health Companies Inc. (NYSE/TSX: BHC) reported that the Company will participate in three investor conferences (Press release, Valeant, AUG 29, 2019, View Source [SID1234539139]).

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Paul S. Herendeen, executive vice president and chief financial officer, Sam Eldessouky, senior vice president and corporate controller, and Arthur J. Shannon, senior vice president and head of Investor Relations and Communications, are scheduled to participate at the Morgan Stanley Global Healthcare Conference in New York on Sept. 10, 2019 at 10:35 a.m. ET.

Paul S. Herendeen, executive vice president and chief financial officer, William Woodfield, vice president and treasurer, and Arthur J. Shannon, senior vice president and head of Investor Relations and Communications, are scheduled to participate at the Deutsche Bank Annual Leveraged Finance Conference in Scottsdale, Ariz. on Sept. 24, 2019 at 10:40 a.m. MT (1:40 p.m. ET).

Joseph C. Papa, chairman and chief executive officer, and Arthur J. Shannon, senior vice president and head of Investor Relations and Communications, are scheduled to participate at the Cantor Fitzgerald Global Healthcare Conference in New York on Oct. 2, 2019 at 10:05 a.m. ET.

A live webcast and audio archive of the events will be available on the Investor Relations page of the Bausch Health Companies Inc. web site at: View Source