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Ayala Pharmaceuticals Reports Second Quarter 2020 Financial Results and Provides Business Update

On August 13, 2020 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA) (the Company or Ayala), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, reported financial results for the second quarter ended June 30, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Ayala Pharmaceuticals, AUG 13, 2020, View Source [SID1234563990]).

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"Our successful IPO in the second quarter represents an important step for Ayala as we work to make meaningful strides across our exciting programs. With a well-capitalized foundation on which to further develop our novel pipeline of therapies for genetically defined cancers, we are poised to execute across multiple milestones in the remainder of 2020 and through the first half of 2021," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "We are looking forward to presenting new interim data from our Phase 2 ACCURACY study of AL101 for the treatment of ACC in September 2020, while also continuing to dose patients in the 6mg dose cohort of the study. Now more than ever, the need for innovation and drug development is evident and we are pleased by the additional clinical progress we have made to-date. This quarter our IND was accepted for our Phase 2 TENACITY study of AL101 monotherapy in patients with recurrent or metastatic triple negative breast cancer who have undergone up to three prior lines of therapy and we are on track to begin dosing patients this year."

Key Business and Clinical Highlights

Phase 2 TENACITY Study of AL101 For Treatment of Triple Negative Breast Cancer: In April 2020, the U.S. Food and Drug Administration (FDA) accepted the investigational new drug application (IND) for the Phase 2 TENACITY study of AL101 for the treatment of Triple Negative Breast Cancer (TNBC). The FDA approved the dosing to commence at 6mg in a monotherapy study to evaluate TNBC patients bearing Notch activating mutations who have undergone 3 or fewer prior lines of therapy. Ayala has opened its first U.S. clinical site for the study.
Upcoming Milestones

New Interim Data from Phase 2 ACCURACY Study of AL101 for the Treatment of Recurrent/Metastatic Adenoid Cystic Carcinoma to be Presented at European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Virtual Congress 2020: Ayala will provide an oral presentation of new interim data from its ongoing Phase 2 ACCURACY study of AL101 for the treatment of recurrent/metastatic adenoid cystic carcinoma (R/M ACC) harboring Notch activating mutations at the upcoming ESMO (Free ESMO Whitepaper) Virtual Congress 2020 on September 18, 2020, at 03:55 CEST.

On Track to Begin Patient Dosing in Phase 2 TENACITY Study of AL101 for the Treatment of TNBC: Ayala expects to initiate patient dosing before year-end 2020 in the Phase 2 TENACITY study of AL101 for the treatment of TNBC harboring Notch activating mutations.

On Track to Initiate Two Phase 2 Clinical Trials in 2021:

Phase 2 Study of AL102 for the Treatment of Desmoid Tumors: Ayala expects to initiate a Phase 2 study of AL102, a potent, selective, oral gamma secretase inhibitor, in patients with desmoid tumors, a category of rare, disfiguring and often debilitating soft tissue tumors, in the first half of 2021.

Phase 2 Study of AL101 for the Treatment of Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia: Based on findings from Ayala’s Phase 1 study of AL101 and supporting data from its preclinical studies, Ayala expects to initiate a Phase 2 study of AL101 for the treatment of relapsed or refractory T-cell acute lymphoblastic leukemia (R/R T-ALL), an aggressive and rare form of T-cell specific leukemia, in the first half of 2021.
Second Quarter 2020 Financial Results

Cash Position: Cash and cash equivalents were $57.4 million as of June 30, 2020, as compared to $16.7 million as of December 31, 2019. Ayala expects its existing cash balance to fund operations through multiple potential key clinical and development milestones into the second half of 2022.

Collaboration Revenue: Collaboration revenue was $1.0 million for the second quarter of 2020 compared to $0.9 million for the same period in 2019. The increase in revenue was attributed to the advancement of Ayala’s collaboration with Novartis for the development of AL102 in combination with Novartis’ anti-B-cell maturation antigen agent.

R&D Expenses: Research and development expenses were $5.1 million for the second quarter of 2020, compared to $3.4 million for the same period in 2019. The increase was primarily driven by additional costs in connection with the advancement of AL101 trials and other preclinical development.

G&A Expenses: General and administrative expenses were $1.5 million for the second quarter of 2020, compared to $1.0 million for the same period in 2019. The increase was primarily related to higher professional services and personnel costs to support the growth of the Company.

Net Income/Loss: Net loss was $6.7 million, or $0.74 loss per share, for the second quarter of 2020, compared to $3.8 million, or $0.76 loss per share, for the same period in 2019, mainly attributable to the advancement of the clinical trials and other preclinical development.

Delcath Systems, Inc. Announces Second Quarter 2020 Results

On August 13, 2020 Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology company focused on the treatment of rare primary and metastatic cancers of the liver, announces financial results for the quarter ended June 30, 2020, and will host an earnings call on August 13, 2020 at 4:30 p.m.

Recent Corporate Highlights:

Completed a $22 million public offering, led by healthcare-focused investors, to allow completion of the Company’s Phase 3 registration trial of Melphalan/HDS in liver-dominant metastatic ocular melanoma (mOM) and refiling of a New Drug Application (NDA) with FDA.

Uplisted to the NASDAQ Capital Market.

Announced management and board transitions.

Initiated pre-commercialization work for Melphalan/HDS in mOM.

Initial physician and payer surveys have highlighted the high-unmet medical need in mOM as well as the expectation of ultra-orphan oncology pricing dynamic for Melphalan/HDS.
Expected Milestones:

Late 2020/early 2021 – COVID-19 has affected clinical trials globally, including our Phase 3 FOCUS registration trial for Melphalan/HDS in liver-dominant ocular melanoma. Importantly, however, throughout these months, the trial protocol remained intact and ongoing trial patients continued to receive treatments. While access to clinical sites for data entry and monitoring was severely restricted during the quarter, the majority of the study’s European and US sites began to ease these restrictions subsequent to quarter end. In addition, we implemented a number of steps to increase data monitoring efforts in light of the impact of the pandemic. Based on the current trajectory of site access, management is focused on delivering top-line results by year-end 2020/early 2021.

Mid-2021– New Drug Application (NDA) submission of Melphalan/HDS in liver-dominant mOM. During the quarter management took steps to ensure progress on key elements of our NDA submission. Those included, among other things, required non-clinical studies and Chemistry, Manufacturing and Controls (CMC) work to ensure that any potential COVID-19 clinical data delays would not affect our timelines to NDA submission.

Initiation of additional clinical studies for Melphalan/HDS in liver-dominant orphan cancers of high unmet-medical need. During the quarter, in-line with the overall restructuring efforts, management initiated a comprehensive review of the multitude of potential pipeline opportunities available for the Company to pursue, as potential label-expansion, beyond mOM. The analysis comprises available clinical evidence, based on the European commercial experience, where Melphalan/HDS is approved as a device-only configuration under the brand name CHEMOSAT, as well as the potential US commercial opportunity. Based on the conclusions of this analysis Delcath expects to initiate at least one additional clinical development program of Melphalan/HDS in coming quarters.
John Purpura, interim CEO of Delcath commented, "Q2 was a transformational quarter for Delcath. The recent $22 million public offering along with our NASDAQ uplisting have been the culmination of a strategic restructuring achieved over the last year. With $51.5 million raised in the 12-month period ended June 30, 2020, led by fundamental healthcare focused investors, Delcath has been restructured, recapitalized and refocused. Our current cash resources, along with expected cash milestones from our European commercialization partner, medac GmBH, provide us with a sufficient runway through multiple value inflection points. These include completion of our Phase 3 FOCUS trial in metastatic ocular melanoma and the refiling of a New Drug Application (NDA) with FDA by mid-2021."

Mr. Purpura added, "Working towards the possibility of having Melphalan/HDS available as a treatment for mOM patients, who have limited therapeutic options, is Delcath’s top priority. With Melphalan/HDS set for potential FDA approval in the second half of 2021, as the only labelled mOM-specific therapy in the US, Delcath has begun pre-commercialization activities which it intends to accelerate in coming quarters. Initial work has highlighted oncologists’ perceptions of the high-unmet medical need of mOM patients, the potential front-line positioning of Melphalan/HDS in this setting and the expectation of attractive ultra-orphan pricing dynamics for our therapy."

Mr. Purpura concluded, "Interventional Oncology has become, in recent years, an integrated, fast-growing segment of cancer care. We believe that Melphalan/HDS is uniquely positioned as a potentially well differentiated, high-value, interventional oncology treatment paradigm targeting orphan and ultra-orphan indications of high unmet medical need. Beyond mOM, Delcath is currently looking to initiate additional studies in one or more liver-dominant metastatic indications for which Melphalan/HDS could be applicable. We expect the next 12 months to be transformational for Delcath and are looking forward to providing updates on our progress throughout."

Second Quarter 2020 Financial Results:

Income Statement Highlights. Product revenue for the three months ended June 30, 2020 was approximately $262 thousand, compared to $221 thousand for the prior year period from our sales of CHEMOSAT procedures in Europe. Selling, general and administrative expenses were approximately $2.3 million compared to $2.7 million in the prior year quarter. Research and development expenses for the second quarter were $2.2 million compared to $1.7 million in the prior year quarter. Total operating expenses for the second quarter were $4.5 million compared with $4.4 million in the prior year quarter.

We recorded a net loss for the three months ended June 30, 2020, of $4.3 million, compared to a net loss of $6.0 million for the same period in 2019.

Balance Sheet Highlights. At June 30, 2020, we had cash, cash equivalents and restricted cash totaling $16.2 million, as compared to cash, cash equivalents and restricted cash totaling $10.2 million at December 31, 2019 and $1.4 million at June 30, 2019. During the three months ended June 30, 2020 and June 30, 2019, we used $7.9 million and $3.2 million, respectively, of cash in our operating activities. In Q2 we made a number of one-time cash payments not indicative of the usual cash usage trend totaling approximately $3.3 million, including compensation payable subsequent to resignations of executives and a director, and past-due payables.

We believe our cash resources and anticipated milestone payments, are adequate to fund our operating activities into mid-year 2021.

Conference Call Information

To participate in this event, dial approximately 5 to 10 minutes before the beginning of the call. Please ask for the Delcath Second Quarter Conference Call when reaching an operator.

Mission Bio Raises $70M in Series C Funding Led by Novo Holdings to Accelerate Development of Cancer Therapies with Single-cell Multi-omics

On August 13, 2020 Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, reported $70 million in its Series C financing led by Novo Growth, the growth equity arm of Novo Holdings (Press release, Mission Bio, AUG 13, 2020, View Source [SID1234563630]). Soleus Capital also joins the round, along with earlier investors Mayfield, Cota, and Agilent, bringing the company’s total funding to more than $120 million. To support Mission Bio’s rapid growth, the funds will scale its single-cell multi-omics technology, the Tapestri Platform, to expand the company’s reach in more effective clinical trials for novel cancer treatments, as well as characterization for cell and gene therapy.

As part of this announcement, Robert Ghenchev, Senior Partner and Head of Novo Growth, has joined Mission Bio’s board of directors. "In Novo Growth, our strategy is to support companies developing cutting edge science that has significant potential to positively impact patients and society. Single-cell analysis has proven highly valuable in cancer research, and we are confident the technology will be used increasingly to unlock key insights into blood cancers, as well as solid tumor indications. We further see a clear opportunity in drug development, where the technology can bring great impact to patients’ lives," said Ghenchev. "We are impressed with Mission Bio’s innate approach to data integration at the single-cell level and are excited to contribute more than capital by activating our broad life science network to support their mission to greatly impact the success and economics of precision therapy development."

Over the past year, Mission Bio has advanced its efforts to support drug development through clinical trials and the translation of research into novel biomarkers with its growing install base in NCI cancer centers and key biopharma companies. The Tapestri Platform is the first-ever single-cell multi-omics platform capable of detecting DNA and protein changes simultaneously from the same cell — a capability necessary for the development of more impactful precision therapies. The award-winning technology has been adopted by Agios, LabCorp, Onconova Therapeutics, and other companies to optimize clinical trials and improve the likelihood of success. Leading cancer centers, including Stanford, MD Anderson Cancer Center, the University of California at San Francisco, and Memorial Sloan Kettering Cancer Center, have leveraged the Tapestri Platform to support their work in therapeutic resistance and treatment response monitoring.

"We are on a mission to help our customers eliminate cancer. With access to multiple layers of the cellular profile, the Tapestri Platform can help identify novel biomarkers in diseased cells, monitor therapy resistance and response, and accelerate novel therapies through clinical trials," said Charlie Silver, CEO, and Co-founder of Mission Bio. "We are delighted to partner with Novo Holdings to advance our impact in drug development and cell and gene therapy."

Micronoma Launches with $3 Million Seed Funding

On August 13, 2020 Micronoma, an early cancer-detection biotech that seeks to develop and commercialize a minimally invasive, microbiome-based method, reported that it has closed a $3 million seed financing round led by microbiome-focused investor, SymBiosis, LLC (Press release, Micronoma, AUG 13, 2020, View Source [SID1234563621]).

The proceeds from funding will be used to further advance cancer detection technology with the development of pioneering microbiome research.

Micronoma was incorporated in June 2019 by its three co-founders: Sandrine Miller-Montgomery, CEO of Micronoma, previously Executive Director of the Center for Microbiome Innovation; Dr. Rob Knight, Director of the Center for Microbiome Innovation at the University of California San Diego (UC San Diego) and Greg Poore, an M.D.-Ph.D. candidate at UC San Diego School of Medicine and co-inventor. Micronoma has exclusively licensed the original IP on microbial-based cancer diagnostics and therapeutics, created by Poore and Knight, from UC San Diego.

Micronoma’s technology has shown that distinct cancer types can be diagnosed sensitively and specifically solely using microbial nucleic acids in human blood and tissues. This approach was first demonstrated using a cancer sequencing database of >10,000 patients from 32 cancer types, then validated on >150 real-world patient samples from Moore’s Cancer Center at UC San Diego Health, across three cancer types and comparing results to those from healthy, non-cancer controls. The results of the study were published in Nature in March 2020 (doi: 10.1038/s41586-020-2095-1).

While Micronoma’s method is capable of querying more than one cancer type in a single small blood sample, the company will first focus on the early detection of lung cancer. Despite its high prevalence and mortality rate, lung cancer has remained challenging to detect at an early stage, including recent liquid biopsy methods focusing on cell-free tumor DNA (ctDNA) or methylation-based methods.

"Micronoma is the only company of its kind that uses the individual’s microbiome data to detect lung cancer in its early stages, so this first seed funding is a major milestone for our company," said Sandrine Miller-Montgomery, CEO of Micronoma. "Between the technical expertise and business acumen of our founding teams and the strong initial financial support of our investors, we are now ready to expeditiously develop and commercialize our first assay, with an overall aim to decrease the number of preventable deaths from disease."

The funding will also enable Micronoma to start development work and the creation of their CLIA (Clinical Laboratory Improvement Amendments) lab. The goal is to provide this new diagnostic to clinicians within the next two years.

"Advances in understanding the microbiome in recent years have led us to better understand its importance in the environment and human health in general. We are now applying this knowledge to cancer diagnostics in order to make a dent in the four-million lives lost a year to preventable cancer death," said Knight. "By offering a novel set of biomarkers from the microbiome found in a simple blood draw, we have a potential new option for early detection. With early detection comes the prospect for early treatment with a better outcome for the patient and all involved."

Sosei Heptares Operational Highlights and Consolidated Results for the First Half 2020

On August 13, 2020 Sosei Group Corporation ("the Company") (TSE: 4565) reported its consolidated results for the first half ended 30 June 202 (Press release, Sosei, AUG 13, 2020, View Source [SID1234563620])0. The full report can be accessed by clicking here.

Operational Highlights for 1H 2020

New global agreement signed with AbbVie – the discovery collaboration and option to license collaboration will initially focus on discovery of novel small molecules targeting inflammatory and autoimmune diseases. The Company will conduct and fund R&D activities through Investigational New Drug (IND)-enabling studies. For the first target, the Company is eligible to receive up to US$32 million in upfront and near-term milestone payments, as well as potential option, development and commercial milestones of up to US$377 million, plus royalties on global sales. AbbVie has the option to expand the collaboration up to a total of four targets.
Measures put in place to successfully operate in response to the COVID-19 pandemic – policies and practises have been rapidly implemented to ensure safety of employees and other stakeholders and to reduce the spread of coronavirus, while also prioritizing revenue-generating work for the major collaboration partners.
New COVID-19 R&D program launched – multidisciplinary team created to apply the Company’s world-leading structure-based drug design capabilities to the global research efforts to discover drugs targeting the SARS-CoV-2 coronavirus and to treat COVID-19, also considering future variants of SARS-CoV-2.
Enerzair Breezhaler (QVM149) approved in Japan as a maintenance treatment of uncontrolled asthma in adult patients – Enerzair is once-daily, first-in-class inhaled LABA/LAMA/ICS combination for asthma patients, being developed by Novartis, and in which Sosei Heptares has an economic interest. The achievement of this milestone resulted in a US$1.25 million payment to Sosei Heptares from Novartis.
High-impact publication in Nature Reviews Drug Discovery – highlighting potential of structure-based approaches to generate peptide drugs targeting G protein-coupled receptors (GPCRs).
Excellent scientific progress made with orexin agonist program in conjunction with spin-off companies Orexia and Inexia – progress building unique orexin modulator drug discovery and design engine triggered next tranche of funding from Medixci. Further orexin receptor structural data and insights generated to help optimize drug discovery targeting neurological diseases.
Post-period Highlights

Approximately US$194 million raised from International Offering of shares and convertible bonds – the Company intends to use the majority of funds to pursue strategic growth initiatives including:
a potentially transformative acquisition to secure long-term revenue growth;
investments in novel technologies that complement and future-proof its drug discovery platform;
expansion of its drug candidate discovery and early development into new target classes; and
in-licensing late-stage clinical assets to develop for the Japanese market.
The balance of the funds will be used to support organic growth initiatives, such as investments in current research activities and general corporate purposes.

Enerzair Breezhaler approved in Europe for asthma – the approval also includes an optional digital companion with sensor and app designed to improve compliance and to better support therapeutic decisions. Sosei Heptares received a US$5 million milestone payment from Novartis, as a result of this approval.
Financial Highlights for the Six-month Period ended 30 June 2020

Revenue totalled JPY 2,516 million (US$23.2 million*) (a decrease of JPY 2,540 million (US$22.7million) vs. the prior corresponding period), and was primarily due to the absence of major milestone payments from existing collaborations. This is because the timing of new business development deals and progress related to existing programs can vary considerably from quarter to quarter. The prior corresponding period included a one-off US$15 million major milestone payment from AstraZeneca.
The Company expects to achieve new upfront and milestone payments later in the fiscal year.

Cash R&D expenses were carefully managed and totalled JPY 1,500 million (US$13.9 million) (a decrease of JPY 362 million (US$3.0 million) vs. the prior corresponding period). This was primarily related to a reduction in project activity caused by the operational impact of the COVID-19 pandemic, as well as the successful recovery of excess costs incorrectly charged by one supplier. In the period under review, 95% of R&D spend related to the UK operations.
Cash G&A expenses totalled JPY 925 million (US$8.5 million) (a decrease of JPY 314 million (US$2.8 million) vs. the prior corresponding period), and was primarily related to a reduction in the UK National Insurance liability linked to share-based payments as a result of the decline in the Company’s share price over the period.
Cash loss** totalled JPY 181 million (US$1.7 million) vs. a cash earnings of JPY 1,596 million (US$14.5 million) in the prior corresponding period. The main reason for the decrease was due to the decrease in revenue as stated above.
Net loss totalled JPY 2,117 million (US$19.6 million) vs. a net profit of JPY 395 million (US$3.6 million) in the prior corresponding period. The main reason for the net loss was due to the decrease in revenue as stated above.
Stable cash balance was maintained despite the net loss, with cash at hand of JPY 15,362 million (US$142.6 million) as at 30 June 2020..
After the period ended June 30, 2020, the Company successfully completed an International Offering of new shares and euro-yen denominated convertible bonds due 2025 that raised a total of JPY 20.9 billion (approximately US$194 million). The Group intends to use the majority of the funds for a potentially transformative acquisition to pursue strategic growth.
*Convenience conversion to US$ at the following rates: 2020: 1US$ =108.253 JPY; 2019: 1US$ =110.064 JPY

**Non-IFRS measure

Shinichi Tamura, Chairman, President and CEO of Sosei Heptares, commented: "I am pleased to report that Sosei Heptares has made good progress in the first half of 2020, our 30th anniversary year and a period that has presented significant challenges for businesses globally. Our response to these challenges has been rapid and impressive, allowing us to ensure employee safety and maintain a high level of business continuity. We have added another major pharma partnership with AbbVie and have secured the long-term growth capital we require to execute our ambitious corporate objectives. These include both organic and inorganic strategic options including a potentially transformative acquisition, as well as investments that enhance our own world-leading discovery and early development capabilities. We are excited by the future and motivated more than ever to build a profitable, global biopharmaceutical company delivering novel drug candidates to treat serious disease as well as value for shareholders."