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Merrimack Reports First Quarter 2020 Financial Results

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.

Kadmon Provides Business Update and Reports First Quarter 2020 Financial Results

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]).

"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.

Savara Reports First Quarter 2020 Financial Results and Provides Business Update

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]).

"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.

Ascentage Pharma’s Core Drug Candidate HQP1351 Granted Fast Track Designation by the US FDA, Marking Another Milestone in Its Development

On May 7, 2020 Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, reported that the US Food and Drug Administration (FDA) has granted HQP1351, the Company’s core drug candidate, a Fast Track Designation (FTD) for the treatment of patients with chronic myeloid leukemia (CML) with certain genetic mutations who have failed to respond to treatments with existing tyrosine kinase inhibitors (TKIs) (Press release, Ascentage Pharma, MAY 7, 2020, View Source [SID1234557407]). This is the first FTD obtained by Ascentage Pharma, and it marks another milestone for HQP1351 following its recent Orphan Drug Designation by FDA.

FTD is designed by FDA to expedite the development and review of drug candidates to treat serious disease/conditions that present urgent unmet clinical needs. This FTD for HQP1351 will lead to a series of benefits that could accelerate the clinical development and review for this drug candidate, including more frequent communications and meetings with FDA during the clinical development; and being allowed to enter Rolling Review, a process that allows the company to submit New Drug Applications (NDAs) by sections, rather than waiting until all required materials become available. This FTD also paves the way for HQP1351 to be qualified for Accelerated Approval and Priority Review designations in the future.

A key factor in determining FTD for a drug candidate is its ability to address an unmet medical need with additional clinical benefits to patients. To qualify for FTD, a drug candidate needs to demonstrate its potential to treat a condition with no existing therapies, offer significant clinical advantages over current treatment options, or bring clinical benefits to patients intolerant to existing therapies or with poor responses to them. This FTD represents FDA’s recognition of HQP1351’s potential in addressing some of these unmet medical needs.

CML is a rare hematologic malignancy with an annual incidence rate of approximately 1.9 cases/100,000. BCR-ABL tyrosine kinase inhibitors (TKIs) have significantly improved clinical management of CML. However, despite clinical benefits offered by the first-generation BCR-ABL inhibitor imatinib (Gleevec), and several second-generation TKIs, many patients develop drug resistance. Such acquired resistance to TKIs is a major challenge in the treatment of CML. BCR-ABL tyrosine kinase mutations represent a key mechanism of acquired drug resistance; T315I, which is the most common drug-resistant mutation, occurs in about 25% of patients with drug-resistant CML. Patients with the T315I mutation are resistant to both first- and second-generation BCR-ABL inhibitors, hence presenting an urgent unmet medical need for next-generation BCR-ABL inhibitors to more effectively target the T315I mutation. Although a third-generation TKI has already been approved in the United States, there are remaining concerns about its safety As a result, patients who failed to respond to existing TKI therapies continue to present an urgent unmet clinical need for safer and more effective therapies.

HQP1351 is a novel, orally active, potent third-generation BCR-ABL inhibitor designed to effectively target BCR-ABL mutants, including T315I, and it is being developed for the treatment of patients with CML resistant to first- and second-generation TKIs. HQP1351 is the first China-developed third-generation BCR-ABL inhibitor targeting drug-resistant CML. The drug candidate is currently being evaluated in pivotal Phase II studies in China, and Ascentage Pharma plans to submit an NDA for HQP1351 this year. In July 2019, HQP1351 was cleared by FDA to enter a Phase Ib study. Data from the Phase I clinical study of HQP1351 were selected for oral presentations at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meetings two years in a row and was nominated as "Best of ASH (Free ASH Whitepaper)" research in 2019. Early data from studies of HQP1351 have demonstrated promising efficacy as well as favorable safety and tolerability profiles.

"Drug resistance to earlier-generation TKI represents an urgent unmet clinical need globally. HQP1351 is a novel, orally active, and potent third-generation BCR-ABL inhibitor being developed by an innovative biopharmaceutical company in China. In our Phase I study with a large sample size of more than 100 patients, HQP1351 demonstrated promising efficacy and a favorable safety profile, with clinical responses in many patients with relapsed or refractory CML who had no effective treatment option," Professor Xiaojun Huang, Director of the Institute of Hematology, Peking University, and the principal investigator of HQP1351 in China. "HQP1351 has the potential of becoming a new option in the clinical management of drug-resistant CML, symbolizing the great advances in biopharmaceutical R&D in China. This FTD by FDA signifies global recognition of the clinical data from China studies, and will hopefully soon benefit patients with CML worldwide. We look forward to further progress in the clinical development of HQP1351."

"HQP1351 is a China-developed third-generation BCR-ABL inhibitor. With this Fast Track Designation, received right after the recent Orphan Drug Designation by FDA, Ascentage Pharma has reached another major milestone in the global development of HQP1351," said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. "These two designations for HQP1351 indicate the urgency for addressing the unmet clinical need in the treatment of CML, and a recognition of HQP1351’s promising efficacy and safety profile by an ex-China health authority as supported by existing data. This FTD will help strengthen our communications and collaboration with FDA in future clinical development and expedite the development and review of HQP1351 in the US. Staying committed to the mission of addressing unmet clinical needs in China and around the world, we will further accelerate the clinical development of HQP1351 to hopefully soon provide a safer and more effective treatment option to patients with CML."

Palatin Technologies, Inc. to Report Third Quarter Fiscal Year 2020 Results; Teleconference and Webcast to be held on May 12, 2020

On May 7, 2020 Palatin Technologies, Inc. (NYSE American: PTN) reported that it will announce its third quarter fiscal year 2020 operating results on Tuesday, May 12, 2020 before the open of the U.S. financial markets (Press release, Palatin Technologies, MAY 7, 2020, View Source [SID1234557406]).

Palatin will also conduct a conference call and live audio webcast hosted by its executive management team on May 12, 2020 at 11:00 a.m. ET. The conference call will include a review of the company’s operating results and an update on programs under development.

Schedule for the Operating Results Press Release, Conference Call / Audio Webcast

Q3 Fiscal Year 2020 Financial Results Press Release

5/12/2020 at 7:30 a.m. ET

Q3 Fiscal Year 2020 Conference Call-Live

5/12/2020 at 11:00 a.m. ET

US/Canada Dial-In Number:

1-888-204-4368

International Dial-In Number:

1-323-994-2082

Conference ID:

8845359

Q3 Fiscal Year 2020 Conference Call-Replay

5/12/2020-5/19/2020

US/Canada Dial-In Number:

1-888-203-1112

International Dial-In Number:

1-719-457-0820

Replay Passcode:

8845359
Audio Webcast Live and Replay Access

View Source

The audio webcast and replay can be accessed by logging on to the "Investors-Webcasts" section of Palatin’s website at View Source.