On November 12, 2019 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported its financial results for the third quarter ended September 30, 2019 and provides a corporate update (Press release, Hookipa Biotech, NOV 12, 2019, View Source [SID1234550964]).

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"In the third quarter, HOOKIPA has made significant progress toward achieving its corporate objectives. Highlights include demonstrating the potential of our TheraT technology with the presentation of robust preclinical data at the CICON cancer immunotherapy conference in Paris. We also welcome two new individuals to our Executive Team who have very strong manufacturing expertise and business development capabilities," commented Joern Aldag, HOOKIPA’s Chief Executive Officer. "Looking ahead, we reaffirm our plan to initiate our first clinical program for TheraT in oncology before the end of 2019 and hope that the preclinical results showcased at CICON translate into positive results for cancer patients."

R&D Pipeline Update and Clinical Progress

HB-101, lead product candidate in infectious diseases

HOOKIPA’s VaxWave-based prophylactic Cytomegalovirus vaccine candidate, HB-101, is currently in a Phase 2 randomized, double-blinded clinical trial in cytomegalovirus-negative patients awaiting kidney transplantation from Cytomegalovirus-positive donors. The majority of sites have been activated. HOOKIPA expects safety and immunogenicity data in the first half of 2020 from the first cohorts enrolled. Preliminary efficacy data scheduled to follow late in the second half of 2020.

HB-201 and HB-202, programs for the treatment of Human Papillomavirus-positive cancers

HB-201 and HB-202, HOOKIPA’s lead oncology product candidates, are in development for the treatment of Human Papillomavirus (HPV)-positive cancers. In July 2019, HOOKIPA announced that its Investigational New Drug (IND) Application for a Phase 1/2 clinical trial of HB-201, a TheraT-based immunotherapy for the treatment of HPV-positive cancers was accepted by the U.S. Food and Drug Administration (FDA). HOOKIPA reaffirms its plan to initiate a Phase 1/2 clinical trial of HB-201 in patients with treatment-refractory HPV16+ cancers before the end of the year. This trial will be HOOKIPA’s first clinical trial in immuno-oncology.

The HB-202 IND filing with the FDA is planned for the first half of 2020. A Phase 1/2 trial combining HB-201 and HB-202, both with and without a checkpoint inhibitor, in patients with treatment-refractory HPV16+ cancers is expected to commence in late 2020.

During the CICON Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) in September in Paris, HOOKIPA presented four posters highlighting the robust preclinical data and broad therapeutic potential for TheraT-based immunotherapies.

Strategic Collaborations

Progress under Gilead collaboration for therapeutic hepatitis B virus (HBV) and human immunodeficiency virus (HIV)

In May 2019, HOOKIPA achieved a $2.0 million research milestone for HBV by designing and delivering 10 research-grade vectors to Gilead Sciences, Inc., along with the characterization of these vectors and delivery of a data package for the HBV program. These research vectors will be subject to further pre-clinical testing in order to validate a clinical candidate for novel combination therapies for the treatment of HBV. This follows the delivery of 14 research-grade vectors for the HIV program in January 2019.

Management Update

Christine D. Baker and Roman Necina joined HOOKIPA’s Executive Team

In September 2019, HOOKIPA announced the hiring of Christine D. Baker as Chief Business Officer (CBO) and Roman Necina, PhD, as Chief Technology Officer (CTO).

Ms. Baker joined HOOKIPA from EpicentRx in August, where she was the CBO and previously Novartis Oncology, where she was a leader in oncology business development and M&A. At HOOKIPA, Ms. Baker will be responsible for the company’s business development, alliance management, and commercial planning and is based in HOOKIPA’s New York City office.

Dr. Necina joined HOOKIPA in November from Takeda, where he was a Senior Vice President for Technical Development and Chief Strategist. He is based in Vienna and will be responsible for HOOKIPA’s manufacturing operations including analytical and process development.

Third Quarter 2019 Financial Results

HOOKIPA’s net loss for the three months ended September 30, 2019 was $11.4 million, compared to a net loss of $4.0 million for the three months ended September 30, 2018. This increase was due to an increase in research and development expenses, mainly driven by the progression of HOOKIPA’s oncology programs, and an increase in general and administrative expenses following HOOKIPA’s IPO.

Revenue was $2.0 million for the three months ended September 30, 2019, compared to $1.9 million for the three months ended September 30, 2018.

HOOKIPA’s research and development expenses for the three months ended September 30, 2019, were $11.0 million, compared to $6.2 million for the three months ended September 30, 2018. The primary drivers of the increase were an increase in direct research and development expenses by $3.6 million and an increase in personnel expenses by $0.9 million. Direct research and development expenses increased primarily due to the expansion of earlier stage programs and the preparation costs of clinical trials for HOOKIPA’s HB-201 and HB-202 programs. In addition, costs related to HOOKIPA’s collaboration with Gilead contributed to the increase in direct expenses.

General and administrative expenses for the three months ended September 30, 2019 were $4.6 million, compared to $1.3 million for the three months ended September 30, 2018. The

increase was mainly due to the growth in personnel related expenses, an increase in professional and consulting fees as well as costs associated with ongoing business activities and costs to operate as a public company.

HOOKIPA’s cash, cash equivalents and restricted cash as of September 30, 2019 was $124.0 million compared to $48.6 million as of December 31, 2018. The increase was primarily attributable to $37.3 million in net proceeds received from the issuance of shares of Series D convertible preferred stock in February 2019, and $74.6 million in net proceeds received from HOOKIPA’s initial public offering in April 2019, offset by cash used in operating and investing activities. On April 23, 2019, HOOKIPA completed an initial public offering of its common stock by issuing 6.0 million shares of its common stock, at $14.00 per share.

Upcoming Investor Events

· Jefferies 2019 London Healthcare Conference, November 20 – 21, 2019

· Piper Jaffray 31st Annual Healthcare Conference NY, December 3 – 5, 2019

On November 12, 2019 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical stage biotechnology company focused on discovering and developing novel small molecule drugs for the treatment of cancer and other life-threatening diseases, reported its financial results for the third quarter ended September 30, 2019 (Press release, Calithera Biosciences, NOV 12, 2019, View Source [SID1234550980]). As of September 30, 2019, cash equivalents and investments totaled $133.6 million.

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"This was a productive quarter for Calithera, during which we achieved multiple, key milestones in our clinical and pipeline programs," said Susan Molineaux, PhD, president and chief executive officer of Calithera. "This includes completing enrollment in our registrational CANTATA trial evaluating telaglenastat for the treatment of patients with renal cell carcinoma, and presenting new data on several programs at both the ESMO (Free ESMO Whitepaper) and SITC (Free SITC Whitepaper) annual meetings. In addition, we successfully completed a Phase 1 trial in healthy volunteers of CB-280, an oral arginase inhibitor for the treatment of cystic fibrosis."

Third Quarter 2019 and Recent Highlights

Completed patient enrollment of randomized CANTATA trial of telaglenastat with cabozantinib in advanced renal cell carcinoma. The CANATA trial is a global, randomized, double-blind clinical trial of telaglenastat combined with cabozantinib, in patients with advanced or metastatic renal cell carcinoma who have received one or two prior treatments. The CANTATA trial enrolled 445 patients at multiple centers globally. The primary endpoint is progression-free survival. Calithera plans to report top-line efficacy and safety data from the trial in the second half of 2020.

Presented results of Phase 2 ENTRATA study of telaglenastat (CB-839) with everolimus in renal cell carcinoma at the ESMO (Free ESMO Whitepaper) 2019 Congress. The ENTRATA trial (NCT03163667) was a Phase 2 randomized, double-blind trial designed to evaluate the safety and efficacy of telaglenastat in combination with everolimus versus placebo with everolimus in patients with advanced clear cell RCC who have been treated with at least two prior lines of systemic therapy, including at least one prior VEGFR-targeted tyrosine kinase inhibitor. The trial enrolled 69 patients at multiple centers in the United States. The primary endpoint of ENTRATA was progression-free survival (PFS). Telaglenastat, when added to everolimus, doubled the median PFS in heavily pretreated patients with advanced RCC to 3.8 months as compared to 1.9 months for everolimus alone, and reduced the risk of disease progression or death by 36% (HR=0.64, p=0.079 one-sided). The primary endpoint of the trial was PFS per investigator assessment with a predetermined threshold of p£0.2 one-sided. The secondary endpoint of overall survival is not yet mature.

Initiated Phase 1/2 clinical trial of telaglenastat in combination with palbociclib for solid tumors. The Phase 1/2 clinical trial is evaluating telaglenastat in combination with Pfizer’s CDK4/6 inhibitor palbociclib, also known as Ibrance. The study will evaluate the safety and anti-tumor activity of telaglenastat plus palbociclib in patients with KRAS-mutated colorectal cancer (CRC) and KRAS-mutated non-small cell lung cancer (NSCLC).

Presented new INCB001158 data at the ESMO (Free ESMO Whitepaper) 2019 Congress. Calithera and Incyte are collaborating to conduct this Phase 1 study evaluating INCB001158 as monotherapy and in combination with the PD-1 inhibitor pembrolizumab in checkpoint inhibitor refractory and naïve advanced/metastatic solid tumors. Responses were observed in patients with microsatellite stable (MSS) colorectal cancer, a disease not historically sensitive to checkpoint inhibition.

Completed a Phase 1 clinical trial of CB-280 in healthy volunteers. The first-in-human Phase 1 trial evaluated the safety, tolerability and pharmacokinetic profile of oral CB-280 in healthy volunteers. A phase 1b clinical study in cystic fibrosis patients is expected to start enrollment in the first half of 2020.

Presented new preclinical data for IL4I1 and CD73 programs at the SITC (Free SITC Whitepaper) Annual Meeting. CB-708 is a selective, oral inhibitor of CD73, an enzyme that synthesizes the immunosuppressive agent adenosine and is over-expressed in multiple tumor types. By blocking adenosine production in the tumor, CB-708 is designed to enhance T-cell activation, leading to anti-tumor activity. Interleukin 4 (IL-4)-Induced Gene 1 (IL4I1) is an enzyme that is primarily expressed by tumor cells and antigen presenting cells, and produces hydrogen peroxide, an inhibitor of T-cell function. IL4I1 has a potential role in immune evasion, and inhibition may enhance an effective anti-tumor immune response. Calithera announced the IL4I1 inhibitor program this quarter.

Selected Third Quarter 2019 Financial Results

Cash, cash equivalents and investments totaled $133.6 million at September 30, 2019.

Research and development expenses were $17.2 million for the three months ended September 30, 2019, compared with $16.4 million for the same period in the prior year. The increase of $0.8 million was primarily due to a $0.7 million increase in the telaglenastat program, including for the CANTATA trial, an increase of $0.4 million in the INCB001158 program, and an increase of $0.5 million in our early-stage research programs, partially offset by a decrease of $0.8 million in our CB-280 program.

General and administrative expenses were $3.9 million for the three months ended September 30, 2019, compared with $3.1 million for the same period in the prior year. The increase of $0.8 million primarily related to higher professional services costs and personnel-related costs.

Net loss for the three months ended September 30, 2019 was $20.3 million, or $0.38 per share.

Conference Call Information

Calithera will host an update conference call today, Tuesday, November 12, at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time. The call may be accessed by dialing (855) 783-2599 (domestic) or (631) 485-4877 and referring to conference ID 6499000. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at www.calithera.com. The webcast will be recorded and available for replay on Calithera’s website for 30 days.

On November 12, 2019 KemPharm, Inc. (Nasdaq:KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported that the Company will host a conference call and live audio webcast on Thursday, November 14, 2019, at 5:30 p.m. ET, to discuss its corporate and financial results for the third quarter 2019 (Press release, KemPharm, NOV 12, 2019, View Source [SID1234550998]).

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Conference Call Information:
Interested participants and investors may access the conference call by dialing either:

(866) 395-2480 (U.S.)
(678) 509-7538 (international)
Conference ID: 8076615
An audio webcast with slide presentation will be accessible via the Investor Relations section of the Company’s website View Source An archive of the webcast and presentation will remain available for 90 days beginning at approximately 6:30 p.m. ET, on November 14, 2019.

On November 12, 2019 Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company focused on hospital acute care and gastroenterology reported third quarter 2019 financial results with net revenues of $10.4 million, up 22% over the prior year quarter (Press release, Cumberland Pharmaceuticals, NOV 12, 2019, View Source [SID1234551014]). Year-to-date net revenues were $33.9 million, up 24% from the prior year period. The Adjusted Earnings year-to-date were $3.5 million or $0.22 per diluted share. As of September 30, 2019, Cumberland had over $100 million in total assets, including just under $30 million in cash and marketable securities.

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QUARTER HIGHLIGHTS:

Announced a U.S. Food and Drug Administration (FDA) Orphan Drug Grant to advance a new Phase II clinical program for patients with Duchenne Muscular Dystrophy (DMD).
Provided the FDA with additional data in support of approval submission for a new line of methotrexate products designed for the treatment of patients with arthritis and psoriasis.
Pursued an update to the Caldolor label that includes new geriatric, shortened infusion, pediatric, and safety data.
Continued a company-wide strategic review of products, partners, and organization.
"There has been an abundance of activity here at Cumberland during the third quarter," said A.J. Kazimi, Chief Executive Officer of Cumberland Pharmaceuticals. "We had a series of positive developments that helped us continue to make progress towards our goal of building a company that offers long-term, sustainable growth."

KEY DEVELOPMENTS:

Ifetroban

In September 2019, Cumberland announced the receival of FDA Orphan Drug Grant funding for a new Phase II clinical program. The Company has now initiated the clinical development of ifetroban for the treatment of cardiomyopathy associated with Duchenne Muscular Dystrophy (DMD). Based on pre-clinical findings, the FDA has cleared Cumberland’s application to study ifetroban in DMD patients, seven years of age and older. In addition, Cumberland was awarded just over $1 million in funding from the FDA through their Orphan Drug Grant program to support this Phase II DMD clinical study. This study is the first DMD clinical study approved for FDA Orphan Product Development funding.

DMD is a rare, fatal, genetic neuromuscular disease and is characterized by the progressive loss of muscle which results in deterioration of the skeletal, heart and lung muscles. This deterioration leads to loss of movement and wheelchair dependency. Heart muscle disease is now the leading cause of death in patients with DMD. There is currently no universally effective treatment for the cardiomyopathy associated with DMD.

Additional Phase II studies of ifetroban are underway including several investigator-initiated trials. The Company is awaiting further study results before deciding on the best path for approval for ifetroban, Cumberland’s first new chemical entity.

Methotrexate

In January 2019, Cumberland received notification from the FDA that the new drug application (NDA) for its new line of methotrexate products was complete and notified the Company of their acceptance for review, setting September 2019 as the Prescription Drug User Fee (PDUFA) action date for an approval decision. Since that time, Cumberland has had a number of communications with the FDA and addressed their questions through multiple amendments that were submitted to the application.

On August 22, 2019, the FDA sent Cumberland a goal extension letter in order to provide them with additional time to review the application setting a new PDUFA action date of early December 2019.

Caldolor

In January 2019, the FDA approved the application for Cumberland’s next generation Caldolor product, featuring a new, patented formulation in a more convenient-to-use package. In April 2019, the Company began initial shipments of the product to select customers. During the third quarter of 2019, there was a growing demand for the product from these select accounts and planning for a full-scale launch of this next generation product is underway.

In addition, Cumberland completed a submission to the FDA in support of an update to a Caldolor approval that included new geriatric, shortened infusion, pediatric, and safety data. Aiming to further expand the product’s label, the Company provided important data generated from its clinical studies regarding an optimal infusion time, additional safety information, as well as geriatric and pediatric administration. The revised label will also include a class label update on the use of NSAIDs with aspirin.

In early September 2019, the FDA informed Cumberland that the submission was not accepted for review because of the number of new claims. The FDA recommended splitting up the submission into several separate submissions, each containing a single proposed labeling claim or group of related labeling claims, with additional data in support of each claim. The Company is planning a Type A meeting to discuss the FDA’s recommendations.

Cumberland has also completed a study to evaluate Caldolor in newborns up to six months of age. The Company is still gathering the data from this study and will report top-line results as they are available. Filing for an additional label expansion with this new data will then be considered.

Cumberland Strategic Review

Earlier this year, Cumberland announced a strategic review of its brands, capabilities, and international partners. This review followed an accelerated business development initiative, which resulted in a series of transactions. Because of that progress, the Company felt that it was prudent to take a fresh look at its product portfolio, partners, and organization to ensure proper focus and capabilities.

As a result, Cumberland:

Expanded international arrangements through several new agreements, including a license with WinHealth Pharma for Vibativ in China and a license with R-Pharma JSC for Vibativ for Russia and several adjacent markets.
Added personnel to its corporate, sales, and medical teams.
During the third quarter of 2019, the Company:

Completed the assignment and amendment of a Commercialization Agreement with Dr. Reddy’s Laboratories Limited ("Dr. Reddy’s") for the registration and distribution of Vibativ in India. Dr. Reddy’s is a multinational pharmaceutical company based in Hyderabad, India. The company currently markets over 190 medications through their commercial operations in over 35 countries. Combined with their extensive network of manufacturing capabilities, Dr. Reddy’s generated over $2.2 billion in sales during their 2018 – 2019 fiscal year.
Extended the arrangements with Clinigen for Ethyol and Totect. On May 13, 2019, Cumberland entered into an agreement with Clinigen Healthcare Limited to conclude the license and distribution agreement for Ethyol and Totect. In early September 2019, Clinigen and Cumberland agreed to change the transition date to late December 2019. Under the terms of the Agreement, Cumberland will no longer distribute Ethyol or Totect after the transition date and will receive $5 million in financial consideration from Clinigen, paid over a two-year period.
Agreed to conclude a co-promotion agreement with Piramal Critical Care effective November 2, 2019. Piramal had been promoting Cumberland’s Caldolor and Vaprisol in hospitals that Cumberland does not cover. A transition plan has been agreed and implemented to return those accounts from Piramal to Cumberland.
FINANCIAL RESULTS:

Net Revenue: For the three months ended September 30, 2019, net revenues were $10.4 million, up 22% from $8.5 million the prior year period.

Net revenue by product for the three months ended September 30, 2019, included $3.3 million for Ethyol and $2.9 million for Kristalose. Net revenue for the Company’s other brands included $1.5 million for Vibativ, $1.2 million for Caldolor, $0.8 million for Acetadote (including the brand and Company’s Authorized Generic), and $0.5 million for its other brands.

For the nine months ended September 30, 2019, net revenues were $33.9 million, up 24% from $27.2 million for the nine months ended September 30, 2018.

Operating Expenses: Total operating expenses for the three months ended September 30, 2019 were $12.2 million, compared to $10.3 million during the prior year period. The primary drivers of this increase were the increase in sales, new cost of goods, and amortization expenses associated with the addition of Vibativ.

Total operating expenses for the first nine months of 2019 were $36.5 million compared to $32.4 million for 2018.

Earnings: Net income (loss) for the third quarter 2019 was $(2.0) million or $(0.13) a share, compared to $(1.6) million or $(0.11) a share for the prior year period.

Adjusted Earnings for the third quarter were $0.1 million or $0.01 per diluted share, up from $(0.8) million or $(0.05) per diluted share for the prior year period.

Adjusted Earnings for the first nine months of 2019 were $3.5 million or $0.22 per diluted share. The definition and reconciliation of Adjusted Earnings to net income (loss) is provided in this release.

Balance Sheet: At September 30, 2019, Cumberland had $29.2 million in cash and marketable securities, including approximately $27.0 million in cash and equivalents. Total assets at September 30, 2019 were $103.8 million. Total liabilities were $52.1 million, including $20.0 million outstanding on the Company’s revolving line of credit, resulting in Total shareholder’s equity of $51.7 million.

Conference Call and Webcast

A conference call and live Internet webcast will be held on Tuesday, November 12, 2019 at 4:30 p.m. Eastern Time to discuss the Company’s third quarter 2019 financial results. To participate in the call, please dial 877-303-1298 (for U.S. callers) or 253-237-1032 (for international callers). A rebroadcast of the teleconference will be available for one week and can be accessed by dialing 855-859-2056 (for U.S. callers) or 404-537-3406 (for international callers). The Conference ID for the rebroadcast is 9865969. The live webcast and rebroadcast can be accessed via Cumberland’s website at View Source

On November 12, 2019 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) will webcast management presentations as follows (Press release, Regeneron, NOV 12, 2019, View Source [SID1234551029]):

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Guggenheim Healthcare Talks Idea Forum, Neuro/Immunology Day, at 11:15 a.m. EST on Monday, November 18, 2019
Piper Jaffray 31st Annual Healthcare Conference at 8:00 a.m. EST on Tuesday, December 3, 2019
The sessions may be accessed from the "Investors & Media" page of Regeneron’s website at View Source Replays of the webcasts will be archived on the Company’s website for at least 30 days.