On May 7, 2020 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported financial results for its fiscal 2020 second quarter (Press release, Anavex Life Sciences, MAY 7, 2020, View Source [SID1234561603]).

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"We are pleased to report that we are approaching the conclusion of the double-blind Phase 2 Parkinson’s disease dementia trial with ANAVEX2-73 (blarcamesine) and we expect to report top line results by mid-2020," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "Both the Alzheimer’s disease Phase 2b/3 trial and clinical Rett syndrome programs are also proceeding as planned. Our focus on Precision Medicine studies for neurological disorders remains persistent."

Program Updates:

Enrollment for the two randomized, double-blind, placebo-controlled Phase 2 ANAVEX2-73 (blarcamesine) U.S. Phase 2 Rett syndrome trial[1] and the AVATAR Rett syndrome trial[2] each have surpassed the 50% mark and continue to enroll.
Despite the outbreak of COVID-19, all clinical trial programs continue to proceed. While Rett syndrome protocols have always by default allowed at-home visits, Anavex implemented contingency plans as recommended by local regulatory authorities for the Phase 2b/3 ANAVEX2-73 (blarcamesine) Alzheimer’s disease (AD) study[3] and the Phase 2 study in Parkinson’s Disease Dementia (PDD) study[4] to ensure remote or virtual assessments for all active patients and all respective extension studies. Because ANAVEX2-73 (blarcamesine) is an oral formulation, study participants are able to receive shipments of their study medication in a controlled and compliant fashion, and direct-to-patient delivery is occurring in multiple countries.
In January 2020, Anavex announced achieving the enrollment target for the ANAVEX2-73 (blarcamesine) Phase 2 study in Parkinson’s disease dementia (PDD). Topline results from this randomized, double-blind, placebo-controlled study are expected by mid-2020.
In February 2020, Anavex announced a publication in the peer-reviewed Journal of Neuroimmunology titled "Sigma-1 Receptor Agonists as Potential Protective Therapies in Multiple Sclerosis" featuring preclinical data of ANAVEX2-73 (blarcamesine) relevant to multiple sclerosis.
In April 2020, Anavex announced a publication in the peer-reviewed journal Alzheimer’s & Dementia: Translational Research & Clinical Interventions,entitled, "A precision medicine framework using Artificial Intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer’s disease therapy: Analysis of the Blarcamesine (ANAVEX2-73) Phase 2a clinical study" highlights the relevance of phenotypic and genotypic precision medicine analyses of Whole Exome Sequencing (WES) and gene expression (RNAseq) data in drug development and in particular the potential to identify patients’ genetic variants and gene expression changes that may predict increased chances of success of Alzheimer’s disease treatments.
Financial Highlights:

Cash and cash equivalents of $26.6 million at March 31, 2020, compared to $22.2 million at fiscal year ended September 30, 2019.
Research and development expenses of $6.1 million for both the current quarter as well as the comparable quarter in 2019.
General and administrative expenses of $1.7 million for the quarter as compared to $2.1 million for the comparable quarter in 2019.
Net loss of $7.2 million, or $0.12 per share for the quarter, compared to net loss of $7.2 million, or $0.15 per share in the comparable quarter of 2019.
The financial information for the fiscal quarter ended March 31, 2020 should be read in conjunction with the Company’s consolidated interim financial statements, which will appear on EDGAR, www.sec.gov and will be available on the Anavex website at www.anavex.com.

Conference Call / Webcast Information

Anavex will host a conference call and webcast today at 4:30 p.m. ET.

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 939-3921 and international callers should dial 1 (678) 302-3550. Please use confirmation number 49673201, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.

On May 7, 2020 Ayala Pharmaceuticals, Inc. (NASDAQ: AYLA), 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 the pricing of its initial public offering of 3,666,667 shares of common stock at a public offering price of $15.00 per share, before underwriting discounts and commissions (Press release, Ayala Pharmaceuticals, MAY 7, 2020, View Source [SID1234558078]). All of the common stock is being offered by Ayala. The offering is expected to close on May 12, 2020, subject to customary closing conditions. In addition, Ayala has granted the underwriters a 30-day option to purchase up to an additional 550,000 shares of its common stock at the public offering price, less underwriting discounts and commissions. The gross proceeds of the offering are expected to be $55.0 million, excluding any exercise of the underwriters’ option.

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Ayala’s common stock is expected to begin trading on the NASDAQ Global Market under the ticker symbol "AYLA" on May 8, 2020.

Citigroup and Jefferies are acting as the lead book-running managers for the offering. Oppenheimer & Co. and Raymond James are acting as the co-managers for the offering.

On May 7, 2020 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) [("Merrimack" or the "Company")] reported its first quarter 2020 financial results for the period ended March 31, 2020 (Press release, Merrimack, MAY 7, 2020, View Source [SID1234557415]).

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"We are pleased to report that we have significantly reduced our operating expenses and also sold certain of our remaining intellectual property assets during the first quarter of 2020," said Gary Crocker, Chairman of Merrimack’s Board of Directors. "As a result of reduced general and administrative costs and the proceeds from the previously announced asset sale we reported a profit of $249,000 for the first quarter. We are currently focused on making further reductions to legal, accounting and management consulting expenses which we plan to realize during the second half of 2020."

First Quarter 2020 Financial Results

Merrimack reported net income of $0.25 million for the first quarter ended March 31, 2020, or $0.02 per basic and diluted share on a fully diluted basis, compared to a net loss of $10.5 million, or $0.78 per basic and diluted share on a fully diluted basis, for the same period in 2019.

Merrimack reported a gain on the sale of assets for the first quarter ended March 31, 2020 of $2.1 million, attributable to the sale of certain of our preclinical nanoliposome programs.

No research and development expenses were recognized for the first quarter ended March 31, 2020 compared to $6.4 million for the same period in 2019.

General and administrative expenses for the first quarter ended March 31, 2020 were $1.9 million, compared to $3.7 million for the same period in 2019.

As of March 31, 2020, Merrimack had cash and cash equivalents and investments of $18.0 million, compared to $16.6 million as of December 31, 2019.

As of March 31, 2020, Merrimack had 13.4 million shares of common stock outstanding.

On May 7, 2020 Kadmon Holdings, Inc. (NYSE:KDMN) reported financial and operational results for the first quarter of 2020 (Press release, Kadmon, MAY 7, 2020, View Source [SID1234557409]).

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"We made encouraging progress this past quarter, having completed our pre-NDA meeting with the FDA. Based on the feedback we have received from the Agency to date, we are confident that our proposed data package will be sufficient to support a submission of KD025 for the treatment of patients with cGVHD in the fourth quarter of this year, as previously guided. We continue to expect to announce topline results from the six-month primary analysis of our pivotal trial of KD025 in cGVHD in the second quarter of 2020, which will include an updated look at Overall Response Rate and safety, as well as initial data on duration of response," said Harlan W. Waksal, M.D., President and CEO of Kadmon.

Dr. Waksal continued, "Although our near-term goal of advancing KD025 in cGVHD remains on target, we are experiencing COVID-19-related delays in enrollment in the ongoing Phase 2 study of KD025 in systemic sclerosis as well as a delay in the initiation of the clinical trial in KD033, our immuno-oncology fusion protein. We continue to monitor the situation at hand in order to attain a better understanding of new timelines within these studies."

Dr. Waksal concluded, "We continue to prioritize the health and safety of our employees and patients as we manage the impact of COVID-19. We have implemented measures including work-from-home directives, while maintaining our operations. In these unprecedented times, the steps we are taking position the Company to continue to advance our pipeline of product candidates."

2020 Anticipated Key Clinical Milestones:

KD025

Announce topline results (updated Overall Response Rate and safety data as well as initial data on duration of response) from primary analysis of KD025-213, the pivotal trial in chronic graft-versus-host disease (cGVHD), conducted six months after completion of enrollment, in the second quarter of 2020; additional secondary endpoints, including failure-free survival and overall survival, will be submitted for presentation at an upcoming medical meeting
Submit New Drug Application (NDA) for KD025 in cGVHD to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020
Continue enrollment in ongoing Phase 2 clinical trial in systemic sclerosis (KD025-209); enrollment is delayed due to the COVID-19 pandemic
KD033

Previously guided to initiate clinical trial of KD033, Kadmon’s anti-PD-L1/IL-15 fusion protein, in the second quarter of 2020; enrollment is delayed due to the COVID-19 pandemic
KD045

Continue ongoing Investigational New Drug Application (IND)-enabling activities of KD045, Kadmon’s next-generation ROCK inhibitor for the treatment of fibrotic diseases
Financial Results

First Quarter 2020 Results

Loss from operations for the three months ended March 31, 2020 was $16.1 million, compared to $22.7 million for the same period in 2019.

The decrease in loss from operations was primarily due to $6.0 million of license revenue recognized by the Company during the three months ended March 31, 2020 related to the strategic partnership with Meiji Seika Pharma Co., Ltd.

Liquidity and Capital Resources

At March 31, 2020, the Company’s cash and cash equivalents totaled $120.0 million, compared to $139.6 million at December 31, 2019. In addition, as of March 31, 2020, the Company held approximately 2.1 million ordinary shares of MeiraGTx Holdings plc (Nasdaq: MGTX), a clinical-stage gene therapy company, with a fair value of $28.2 million.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. KD025 is being studied in an ongoing pivotal trail in cGVHD as well as an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209). The FDA has granted Breakthrough Therapy Designation to KD025 for the treatment of patients with cGVHD after failure of two or more prior lines of systemic therapy. The FDA has also granted Orphan Drug Designation to KD025 for the treatment of patients with cGVHD.

On May 7, 2020 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported financial results for the first quarter ending March 31, 2020 and provided a business update (Press release, Savara, MAY 7, 2020, View Source [SID1234557408]).

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"With the recent clarity around the IMPALA 2 study design, along with the expansion of our pipeline with the Phase 3 Apulmiq development program, 2020 has kicked off with a strong start," said Rob Neville, Chief Executive Officer, Savara. "We are now working diligently to get these two studies initiated as soon as possible."

Recent Developments

Molgradex for autoimmune pulmonary alveolar proteinosis (aPAP)

Based on discussions with the U.S. Food and Drug Administration (FDA), the Company believes the second Phase 3 study will be a randomized, double-blind, placebo-controlled study of Molgradex 300 µg administered once daily continuously compared to matching placebo over 48 weeks. The primary endpoint will be change from baseline to week 24 in diffusion capacity of the lungs (DLCO) percent predicted. Secondary endpoints will be change in baseline to week 24 in St. George’s Respiratory Questionnaire (SGRQ) Total Score, SGRQ Activity Component, and exercise capacity using a treadmill test.
Apulmiq for non-cystic fibrosis bronchiectasis (NCFB)

Obtained the global rights to develop and commercialize Apulmiq (inhaled ciprofloxacin).
The Company expects to work with the FDA to plan a confirmatory Phase 3 study that will be based on key learnings from previous studies of inhaled antibiotics for NCFB.
AeroVanc for methicillin-resistant Staphylococcus aureus (MRSA) lung infection

Due to COVID-19 concerns, the Company closed enrollment in the Phase 3 AVAIL study. Total target enrollment was 200 patients. Enrollment in the adult population completed, with 55 patients out of a target of 50. One hundred and thirty-three patients were enrolled in the primary analysis population (younger patients between 6-21 years of age) out of a target of 150. Top line results are still expected in early 2021.
Molgradex for nontuberculous mycobacterial (NTM) lung infection

Due to COVID-19 concerns, the Company closed enrollment in the Phase 2a ENCORE study. Fourteen patients out of a target of ~30 were enrolled. Despite closing enrollment early, data from the enrolled patients will provide useful information on the safety, and potential efficacy, of Molgradex in people living with cystic fibrosis who have NTM lung infection.
First Quarter Financial Results (Unaudited)

Savara’s net loss attributable to common stockholders for the three months ended March 31, 2020 was $15.4 million, or $(0.27) per share, compared with a net loss attributable to common stockholders of $12.1 million, or $(0.34) per share, for the three months ended March 31, 2019.

Research and development expenses were $13.2 million for the three months ended March 31, 2020, compared with $10.0 million for the three months ended March 31, 2019. The increase was primarily due to approximately $5.4 million equal to the aggregate of the fair value of Savara common stock to be issued and cash remunerated to the licensor under the development and commercialization licensing rights to Apulmiq. The upfront license payment expenses were offset by decreased development costs associated with the development of Molgradex and AeroVanc in the amount of $1.7 million and $0.5 million, respectively.

General and administrative expenses for the three months ended March 31, 2020 were $3.0 million, compared with $2.8 million for the three months ended March 31, 2019. The increase was primarily due to increased noncash stock-based compensation charges, personnel costs, and corporate insurance costs for the three months ended March 31, 2020.

As of March 31, 2020, Savara had debt of approximately $25.0 million and cash, cash equivalents, and short-term investments of approximately $105 million. Under the current operating plan, the Company believes this is sufficient capital to fund planned operations into 2022.

Conference Call/Webcast

Savara management will host a conference call/webcast today at 4:30 p.m. Eastern Time (ET) / 1:30 p.m. Pacific Time (PT). Shareholders and other interested parties may access the call by dialing (855) 239-3120 from the U.S., (855) 669-9657 from Canada, and (412) 542-4127 from elsewhere outside the U.S. and requesting the "Savara Inc." call. A live webcast of the call can be accessed on the Investors page of Savara’s website at View Source

Approximately one hour after the call, a telephone replay will be available and will remain available through May 14, 2020 by dialing (877) 344-7529 from the U.S., (855) 669-9658 from Canada and (412) 317-0088 from elsewhere outside the U.S. and entering the replay access code 10143130. A webcast replay will be available on the Investors page of Savara’s website and will remain available for 30 days.