On May 14, 2019 KemPharm, Inc. (Nasdaq: KMPH), a specialty pharmaceutical company engaged in the discovery and development of proprietary prodrugs, reported its corporate and financial results for the quarter ended March 31, 2019 (Press release, KemPharm, MAY 14, 2019, View Source [SID1234536342]).

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"The first quarter of 2019 and early April were highlighted by advancements in our KP415 and APADAZ programs, serving to propel each towards what we believe will be significant value building events in the coming months," said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "For KP415, our prodrug product candidate being developed for the treatment of attention-deficit/hyperactivity disorder (ADHD), we announced the completion of the pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA). We have recently received the minutes from that meeting which confirm the previously reported results. In summary, we believe the review of the KP415 pre-NDA briefing package, which included clinical, non-clinical and human abuse potential study results, as well as regulatory elements, was consistent with our interpretation and previous interactions with the FDA, and, as a result, we believe that we are positioned to submit the KP415 NDA in late second quarter or early third quarter 2019."

"In parallel with the regulatory activities for KP415, we have entered into what we believe are the final stages of the partnering process for our ADHD franchise, which includes KP415 and KP484," continued Dr. Mickle. "Since the initiation of the partnering process, our goal has been focused on securing an agreement with a partner fully dedicated to maximizing the commercial value of KP415 and KP484. While still engaged with multiple parties, we believe that the process should be complete sometime later this quarter or early third quarter."

"With regard to APADAZ, we continue to work with our partner, KVK Tech, to advance several initiatives that we believe will contribute to a successful market introduction of the product. The commercial launch remains on track for the second half of 2019," said Dr. Mickle. "The first quarter was highlighted by three important events. In January, we received FDA approval of two additional dosage strengths of APADAZ. Following that, in February, APADAZ was added to compendia with authorized generic pricing similar to current generics. And most recently, beginning in March, we learned that the ongoing process of formulary adoption of APADAZ and its authorized generic had advanced more rapidly than we anticipated. Notably, according to Managed Markets Insights and Technology estimates, APADAZ, both the brand and authorized generic, is currently being reviewed and added to formularies in various markets including both commercial and Medicaid. Based on this progress and other indicators, we continue to believe that the replacement of current hydrocodone/acetaminophen (APAP) products with APADAZ and its authorized generic has the potential to be a meaningful market opportunity."

Q1 2019 Financial Results:
For the quarter ended March 31, 2019, KemPharm’s reported net loss was $12.3 million, or $0.46 per basic and diluted share, compared to a net loss of $26.2 million, or $1.77 per basic and diluted share for the same period in 2018. Net loss for Q1 2019 was driven primarily by an operating loss of $11.4 million and net interest expense and other items of $1.4 million, partially offset by non-cash fair value adjustment income of $0.5 million. The operating loss of $11.4 million for Q1 2019 was a decrease of $3.4 million compared to $14.8 million in Q1 2018, which was primarily due to decreases of $3.1 million in research and development expenses and $0.3 million in general and administrative expenses, respectively.

As of March 31, 2019, total cash, which is comprised of cash, cash equivalents and restricted cash, was $14.0 million, which was a decrease of $8.4 million as compared to December 31 2018. The decrease in total cash during Q1 2019 was due to a use of cash of $11.1 million, offset by proceeds of $2.7 million on the sale of approximately 1.4 million shares under the equity line of credit with Lincoln Park Capital.

"As we seek to finalize the partnering process for KP415 and KP484, we have utilized a portion of our equity line of credit with Lincoln Park Capital. This additional capital, combined with cost reductions, extends the cash runway further into Q3 2019," said Dr. Mickle. "In addition, we expect that our cash burn rate will reduce substantially following the NDA filing for KP415."

Presentation and Webcast at RBC Capital Markets Global Healthcare Conference:

KemPharm also announced today that Dr. Mickle will present at the RBC Capital Markets 2019 Global Healthcare Conference being held May 21-22, 2019, at the InterContinental New York Barclay.

Details of KemPharm’s presentation are as follows:

Event: RBC Capital Markets 2019 Global Healthcare Conference
Date: Wednesday, May 22, 2019
Time: 1:35 PM (EST)
Room: Morgan Suite
Location: InterContinental New York Barclay, 111 East 48th Street, New York, NY
The presentation will be webcast and available one hour following the live event at http://www.veracast.com/webcasts/rbc/healthcare2019/82314394157.cfm. The replay will be available for 90 days.

In addition, the presentation will be available on the Investor Relations section of the Company’s website at View Source

Recent and Q1 2019 Activities:

Announced Completion of KP415 Pre-NDA Meeting with FDA

On April 11, 2019, KemPharm announced that it concluded a pre- NDA meeting with the FDA for KP415, KemPharm’s investigational attention-deficit/hyperactivity disorder (ADHD) product candidate that contains serdexmethylphenidate (SDX, a prodrug of d-methylphenidate) and d-methylphenidate. At the pre-NDA meeting, representatives from the FDA reviewed KemPharm’s summary of the data package being prepared for the KP415 NDA submission, including clinical, non-clinical and human abuse potential studies, as well as regulatory elements. Based on the feedback from the FDA, the Company believes its regulatory data package will be sufficient for submission, with acceptance of the filing subject to the FDA’s review of the complete package.
Provided Update on APADAZ Formulary Adoption

On March 13, 2019, KemPharm provided an update on formulary adoption of APADAZ, an immediate release combination of KemPharm’s prodrug, benzhydrocodone, and APAP. APADAZ and its authorized generic are currently being reviewed and added to formularies in various markets including Medicaid. KemPharm believes the continued adoption of APADAZ by formularies is another stepping stone in the efforts to prepare APADAZ for commercial launch as soon as the second half of this year.

Announced Enhancements to U.S. and Global Intellectual Property Estate, Including IP Protection for KP415 in Canada, Japan and Korea

On January 29, 2019, KemPharm announced enhancements to its U.S. and global intellectual property estate governing its portfolio of prodrug product candidates. The United States Patent and Trademark Office issued seven (7) new patents to KemPharm during 2018 related to several of its compound families, including KP201, KP303, KP511, KP606 and KP746. In addition, KemPharm has augmented and strengthened the global patent estate for KP415 with the addition of issued patents last year in Canada, Japan and Korea.
Presented Scientific Posters at APSARD’s 2019 Annual Meeting

On January 18, 2019, KemPharm announced that research assessing the oral and intranasal human abuse potential of SDX, KemPharm’s prodrug of d-methylphenidate (d-MPH), as well as new pharmacokinetic data for KP415, were presented in four posters and one oral "data blitz session" at the 2019 Annual Meeting of the American Professional Society for ADHD and Related Disorders (APSARD).
Announced FDA Approval of sNDA for Two Additional Strengths of APADAZ (4.08 mg benzhydrocodone/325 mg APAP and 8.16 mg benzhydrocodone/325 mg APAP

On January 7, 2019, KemPharm announced that the FDA approved a Supplemental New Drug Application (sNDA) for two additional strengths of APADAZ. The approval of these new dosage strengths, 4.08 mg benzhydrocodone/325 mg APAP and 8.16 mg benzhydrocodone/325 mg APAP, follows the NDA approval on February 23, 2018 of the 6.12 mg benzhydrocodone/325 mg APAP dosage strength of APADAZ.

On May 14, 2019 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, and through our license to Santen Pharmaceutical Co. Ltd., wet age‐related macular degeneration, reported financial results for the first quarter ended March 31, 2019 (Press release, Tracon Pharmaceuticals, MAY 14, 2019, View Source [SID1234536341]).

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Recent Corporate Highlights

In April, we announced termination of further enrollment into company sponsored trials of TRC105 due to lack of efficacy in the Phase 3 TAPPAS trial evaluating TRC105 in combination with Votrient (pazopanib) in patients with advanced or metastatic angiosarcoma.
"While we were disappointed in the outcome of the TAPPAS interim analysis, we have several other active clinical programs and look forward to developing multiple bispecific antibodies through our broad and long term partnership with I-Mab Biopharma," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "We are poised to initiate first-in-human dosing of the CD73 antibody TJ004309 and look forward to 2020 when we expect to begin clinical development of the first of up to five bispecific antibodies. We also continue to evaluate companies with first-in-class or best-in-class clinical stage assets who would benefit from accessing our product development platform, which we believe offers a rapid and capital-efficient U.S. drug development solution."

Expected Upcoming Milestones

Dosing of the first patient in a Phase 1 study of TJ004309 as a single agent and in combination with Tecentriq (atezolizumab), a PD-L1 checkpoint inhibitor marketed by Roche, in patients with advanced solid tumors is expected mid-2019.

Publication of TRC253 Phase 1 data in patients with metastatic castrate resistant prostate cancer is expected in the second quarter of 2019.

Top-line data from the randomized Phase 2 AVANTE trial of DE-122 in patients with wet age-related macular degeneration (AMD) are expected in the first half of 2020.
First Quarter 2019 Financial Results

Cash, cash equivalents and short-term investments were $32.1 million at March 31, 2019, compared to $39.1 million at December 31, 2018. We expect our current cash, cash equivalents and short-term investments to fund operations into the third quarter of 2020.

Collaboration revenue was $0 for the first quarter of 2019 compared to $3.0 million for the first quarter of 2018. The decrease was due to the $3.0 million non-refundable upfront payment received in connection with our prior agreement with Ambrx, which was recorded as revenue in the first quarter of 2018.

Research and development expenses for the first quarter of 2019 were $5.2 million compared to $9.4 million for the first quarter of 2018. The decrease was primarily attributable to lower manufacturing expenses for TRC105 in the first quarter of 2019 as compared to the 2018 period.

General and administrative expenses for the first quarter of 2019 were $1.9 million compared to $1.8 million for the first quarter of 2018.

Net loss for the first quarter of 2019 was $7.2 million compared to $8.4 million for the first quarter of 2018.
Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EST / 1:30 p.m. PST to provide an update on corporate activities and to discuss the financial results of its first quarter 2019. The dial-in numbers are (855) 779‑9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 9290299. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About DE-122 (carotuximab)

DE-122, a novel ophthalmic formulation of carotuximab, is active in preclinical choroidal neovascularization (CNV) models and designed to enhance the effect of approved VEGF inhibitors used to treat wet AMD. DE-122 is being investigated in the Phase 2 randomized AVANTE trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD.

About TRC253

TRC253 is a novel, orally bioavailable small molecule that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F877L mutation. The AR F877L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR. TRC253 is currently being studied in a Phase 1/2 clinical trial in prostate cancer. For more information about the clinical trial, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in a Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.

On May 14, 2019 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH / TSX:AUP) ("Aurinia" or the "Company") reported financial results for the first quarter ended March 31, 2019 and provided an update on recent operational highlights. Amounts, unless specified otherwise, are expressed in U.S. dollars (Press release, Aurinia Pharmaceuticals, MAY 14, 2019, View Source [SID1234536340]).

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First Quarter 2019 Highlights

Fully-enrolled AURORA Phase 3 trial in lupus nephritis ("LN") continues on track with results anticipated in late 2019.
Reported results from a Phase 2a Dry Eye study with voclosporin ophthalmic solution ("VOS") that achieved statistically superior efficacy in secondary objective endpoints compared to cyclosporin ophthalmic emulsion 0.05% (Restasis), the current DES market leader. VOS did not meet the primary endpoint as both drugs were well tolerated and demonstrated less than anticipated drop discomfort

Received a Notice of Allowance from the United States Patent and Trademark Office ("USPTO") for claims which have the potential to cover voclosporin’s method of use and dosing protocol for lupus nephritis ("LN’) until December 2037.
Appointed Mr. Peter Greenleaf as Chief Executive Officer and Dr. George Milne to Chairman of the Board of Directors.
Cash, cash equivalents, and short-term investments of $144.3 million as of March 31, 2019.
Recent Director and Officer Appointments

On April 29, 2019, Aurinia appointed Peter Greenleaf as Chief Executive Officer and a Director on the Aurinia Board.

Concurrently, Dr. Richard M. Glickman, who previously announced his plans to retire on November 6, 2018, stepped down from his role as Chairman and CEO. Dr. Glickman remains an advisor to the Company for a period of 12 months.

"It is an honor to join Aurinia at this time and lead the organization through its next phase of growth to advance voclosporin toward commercialization based upon the Phase 3 AURORA results in lupus nephritis anticipated by the end of this year," commented Mr. Peter Greenleaf, Chief Executive Officer of Aurinia. "After following the Aurinia story and after conducting further due diligence, I am truly impressed with the Aurinia team, their ability to execute and advance voclosporin in a cost-efficient manner, with the ongoing goal of bringing voclosporin to help patients suffering with LN."

In conjunction with Dr. Glickman’s retirement as the Chairman, the Board elevated George M. Milne, Jr., PhD, to the position of Chairman of the Board effective April 29, 2019. In addition, the Board appointed Dr. Daniel Billen to the Board also effective April 29, 2019.

Dr. Milne stated, "With the appointment of Peter and Daniel to the board, combined with our experienced and committed employees and management team, Aurinia is strongly positioned to achieve our milestones and maximize the value of voclosporin for all of our stakeholders

VOS for Dry Eye Syndrome ("DES")

Based upon the exploratory Phase 2a results generated with VOS in a head-to-head comparison vs. the current market leader for the treatment of DES, Aurinia plans to initiate a Phase 2/3 study by late 2019. This study will encompass certain critical regulatory requirements that the FDA has traditionally required for DES product approval, these requirements include both dose-optimization requirements along with a comparison versus vehicle.

"I’m confident that the internal Aurinia team along with our key ophthalmology clinical advisors have crafted a framework of a plan that minimizes the clinical and regulatory risk for VOS and maximizes our probability of launching VOS into the multi-billion dollar DES market in due course," said Michael R. Martin, Chief Operating Officer of Aurinia.

Financial Liquidity at March 31, 2019

As at March 31, 2019, Aurinia had cash, cash equivalents and short-term investments of $144.3 million compared to $125.9 million of cash, cash equivalents and short-term investments as at December 31, 2018. Net cash used in operating activities was $13.1 million for the first quarter ended March 31, 2019 compared to $14.4 million for the first quarter ended March 31, 2018.

The Company believes, that based on its current plans that Aurinia has sufficient financial resources to fund the existing LN program, including the AURORA trial and the AURORA 2 extension trial, complete the NDA submission to the FDA, conduct the ongoing Phase 2 study for FSGS, commence additional DES studies and fund operations into mid-2020.

The increase in our cash position at March 31, 2019 was primarily the result of the following:

At-The-Market ("ATM") Facility

On November 30, 2018, Aurinia had entered into an open market sale agreement with Jefferies LLC pursuant to which the Company could from time to time sell, through ATM offerings, common shares that would have an aggregate offering amount of up to $30 million. The ATM was fully utilized in the first quarter. Aurinia received gross proceeds of $30 million and issued 4.6 million common shares. The Company incurred share issue costs of $1.2 million including a 3% commission and professional and filing fees related to the ATM offerings.

February 14, 2014 Warrant Exercises

The remaining derivative warrants outstanding from the February 14, 2014 private placement were exercised in the first quarter ended March 31, 2019. Certain holders of these warrants elected the cashless exercise option and the Company issued 687,000 common shares on the cashless exercise of 1.3 million warrants. Three holders of 464,000 warrants exercised these warrants for cash, at a price of $3.2204 per common share. The Company received cash proceeds of $1.5 million and issued 464,000 common shares.

Financial Results for the First Quarter Ended March 31, 2019

The Company reported a consolidated net loss of $12.4 million or $0.14 per common share for the first quarter ended March 31, 2019, as compared to a consolidated net loss of $15.5 million or $0.18 per common share for the first quarter ended March 31, 2018.

The loss for the first quarter ended March 31, 2019 reflected a reduction of $1.7 million in the estimated fair value of derivative warrant liabilities compared to an increase of $2.6 million in the estimated fair value of derivative warrant liabilities for the first quarter ended March 31, 2018. The derivative warrant liabilities will ultimately be eliminated on the exercise or forfeiture of the warrants and will not result in any cash outlay by the Company.

The loss before the change in estimated fair value of derivative warrant liabilities and income tax was $14.1 million for the first quarter ended March 31, 2019 compared to $12.9 million for the same period in 2018.

Research and development ("R&D") expenses increased to $10.6 million for the first quarter ended March 31, 2019 2019, compared to $8.9 million for the first quarter ended March 31, 2018. The increase in these expenses primarily reflected completion costs for the DES study and higher costs incurred for the AURORA 2 extension trial, the DDI study and the FSGS Phase 2a study as these studies had more activity in the first quarter ended March 31, 2019 compared to the same period in 2018.

Corporate, administration and business development expenses increased slightly to $3.9 million for the first quarter of 2019, compared to $3.8 million for the first quarter of 2018.

This press release should be read in conjunction with our unaudited interim condensed consolidated financial statements and the Management’s Discussion and Analysis for the first quarter ended March 31, 2019 which are accessible on Aurinia’s website at www.auriniapharma.com, on SEDAR at www.sedar.com or on EDGAR at www.sec.gov/edgar.

Aurinia will host a conference call and webcast to discuss the first quarter ended March 31, 2019 financial results today, Monday, May 13, 2019 at 4:30 p.m. ET. This event can be accessed on the investor section of the Aurinia website at www.auriniapharma.com.

On May 14, 2019 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported financial results and business highlights for the first quarter 2019 (Press release, Cyclacel, MAY 14, 2019, View Source [SID1234536297]). The Company’s net loss applicable to common shareholders for the three months ended March 31, 2019 was $1.9 million. As of March 31, 2019 cash and cash equivalents totaled $17.9 million.

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"We continue to execute on our strategy to develop innovative therapies to overcome cancer resistance mechanisms through combinations of our candidates with approved drugs," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "Two Phase 1, dose escalation studies evaluating CYC065 in combination with venetoclax in patients with relapsed/refractory CLL and CYC140 as single agent in a first-in-human trial are open for accrual and patients have been dosed on both studies. Two additional studies evaluating combinations of CYC065 and sapacitabine with venetoclax are under review by institutional review boards. The Phase 1 study of single agent CYC065 has been amended to evaluate an oral form of CYC065. We are pleased to report that the first two patients with BRCA mutant breast cancer treated in the IST evaluating sapacitabine and olaparib have achieved tumor shrinkage. During the quarter, we also extended our projected cash runway to the end of 2020 through our ATM equity sales agreement."

Key Company Highlights

·Data was presented at the 2019 AACR (Free AACR Whitepaper) Annual Meeting from the Company’s DNA damage response program with an oral, sequential regimen of sapacitabine and seliciclib from an expansion cohort in patients with BRCA mutant metastatic breast cancer. The data demonstrated that the regimen was safe and led to a clinical benefit rate of 30%. All eight PARP inhibitor naïve patients, half of the patients previously treated with platinum agents and one on previous PARP inhibitor responded. Progression on previous platinum or PARP inhibitors was associated with lack of benefit. Both sapacitabine and PARP inhibitors are more effective in cancer cells with BRCA mutations or other homologous recombination repair deficiencies.
·Based on data from the above study, the investigators are enrolling a Phase 1b/2 study with an oral, concomitant regimen of sapacitabine and olaparib in patients with BRCA mutant breast cancer. According to the investigators three patients have been dosed. The first two achieved tumor shrinkage and continue on treatment and the third has completed first cycle without dose-limiting toxicity. Dual targeting of the DNA damage response pathway with sapacitabine and olaparib may improve the current standard of care for such patients.
·Two patients have been treated in the Phase 1, dose escalation clinical trial evaluating CYC065 in combination with venetoclax, a Bcl-2 inhibitor, in patients with relapsed/refractory CLL. Preclinical data presented at the 2018 AACR (Free AACR Whitepaper) showed synergistic activity of CYC065 and venetoclax combination in CLL tumor samples, including those with 17p deletions. The combination was also active in CLL samples resistant to either agent alone, suggesting that dual targeting of Mcl-1 and Bcl-2 dependent mechanisms could overcome intrinsic resistance to each individual compound.
·Two patients have been dosed in the recently opened Phase 1, first-in-human, dose escalation study evaluating CYC140 monotherapy in patients with advanced leukemias. CYC140 is a small molecule, selective polo-like-kinase 1 (PLK1) inhibitor that has demonstrated potent and selective target inhibition and high activity in xenograft models of human cancers.
·The Company raised net proceeds of approximately $4.1 million from its Common Stock Sales Agreement with H.C. Wainwright.

x 200 Connell Drive, Suite 1500, Berkeley Heights, NJ 07922, USA Tel +1 908 517 7330 Fax +1 866 271 3466

¨ 1 James Lindsay Place, Dundee, DD1 5JJ, UK Tel +44 1382 206 062 Fax +44 1382 206 067

www.cyclacel.com – [email protected]

Key Upcoming Business Objectives

· Initiate CYC065-venetoclax Phase 1 study in patients with relapsed or refractory AML or MDS;
· Initiate sapacitabine-venetoclax Phase 1 study in patients with relapsed or refractory AML or MDS;
· Report initial data from the CYC065-venetoclax Phase 1 study in relapsed/refractory leukemias;
· Report initial data from the CYC140 Phase 1 First-in-Human study;
· Report initial data and bioavailability from the Phase 1 study of an oral formulation of CYC065;
· Report updated CYC065 Phase 1 data in patients with advanced solid cancers;
· Report data from the IST Phase 1b/2 trial of sapacitabine-olaparib combination in patients with BRCA mutant metastatic breast cancer when reported by the investigators;
· Determine regulatory pathway and submissibility of sapacitabine in elderly AML patients.

Financial Highlights

As of March 31, 2019, cash and cash equivalents totaled $17.9 million compared to $17.5 million as of December 31, 2018. The increase of $0.4 million in the three months was primarily due to net proceeds from a Common Stock Sales Agreement with H.C. Wainwright of $4.1m, offset by net cash used in operating activities of $3.7 million. The Sales Agreement was concluded in the first quarter 2019.

Research and development expenses were $1.0 million for the three months ended March 31, 2019 compared to $0.8 million for the same period in 2018.

General and administrative expenses were $1.2 million for the three months ended March 31, 2019 compared to $1.4 million for the same period in 2018.

Other income, net for the three months ended March 31, 2019 was $0.1 million compared to $0.6 million for the same period of the previous year.

The United Kingdom R&D and tax credit was $0.3 million for the three months ended March 31, 2019 compared to $0.2 million for the same period in 2018.

Net loss for the three months ended March 31, 2019 was $1.8 million compared to $1.3 million for the same period in 2018. With the projected cash-sparing benefits accruing from the MD Anderson alliance the Company believes that cash and marketable securities, which were approximately $17.9 million as of March 31, 2019, will be sufficient to finance operations through the end of 2020.

Conference call information:

US/Canada call: (877) 493-9121 / international call: (973) 582-2750

US/Canada archive: (800) 585-8367 / international archive: (404) 537-3406

Code for live and archived conference call is 9383419

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.

On May 14, 2019 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage immunotherapeutics company, reported financial results for the first quarter ended March 31, 2019 and provided a business update (Press release, Altimmune, MAY 14, 2019, View Source [SID1234536296]).

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"We made great progress in the first quarter, as we executed on our strategy and worked to build long-term shareholder value," said Vipin K. Garg, Ph.D., President and Chief Executive Officer. "We are diligently exploring opportunities to expand our pipeline via acquisition and in-licensing opportunities in both immuno-oncology and liver disease indications. We continue to advance the development of our current key assets, HepTCell and ALT-702, and anticipate achieving significant milestones in both these programs during the next 12 to 18 months. In addition, we have made important advances in both our NasoVAX and NasoShield programs."

Recent Highlights

HepTcell Phase 1 Clinical Trial Data Presented at International Liver Congress
The Company presented results of its HepTcell Phase 1 clinical trial at The International Liver CongressTM sponsored by The European Association for the Study of the Liver (EASL). Mark Thursz, MBBS, MD, FRCP, Professor of Hepatology at Imperial College, London, presented HepTcell Phase 1 data in the session entitled "Hepatitis B – Drug Development." As previously announced, the successful clinical trial met its primary endpoint of safety and showed that HepTcell treatment was associated with increased HBV-specific cellular immune responses. The Company is preparing to initiate a Phase 2 clinical study in the U.S. in 2020.

ALT-702 Preclinical Development Initiated
Recently initiated preclinical development of its immunostimulant product candidate, ALT-702. ALT-702 is based on a new synthetic peptide conjugate technology platform that allows localized immune stimulation without the safety risk of systemic inflammation. ALT-702 represents a new approach in immuno-oncology that can act alone or improve the effectiveness of immune checkpoint inhibitors, oncolytic viruses and other approaches in immuno-oncology. The Company expects to provide an update on the progress of ALT-702 later this year.

NasoVAX Phase 2 Extension Study Completed Showing Durable Immune Response
The Company presented results of its NasoVAX Phase 2 study and the recently completed extension study at the World Vaccine Congress. As previously announced, the Phase 2 data showed that NasoVAX was well-tolerated and highly immunogenic, demonstrating 100% seroprotection at two of the three dose levels studied. Data from the extension study showed that the immunogenic responses were durable with 100% of the evaluated subjects remaining seroprotected with no decrease in seroconversion rate more than one year after vaccination. Durable responses on the order of one year are not expected from current injected influenza vaccines and suggest that the immune response induced by NasoVAX could be protective for the duration of a long flu season. The Company is actively looking for a strategic partner to further develop and commercialize NasoVAX.

NasoShield Investigation Completed with Potential Path Forward in the Clinic
Completed the investigations of potential causes that may have contributed to the lower than expected immune response in the Phase I study of NasoShield funded through a contract (HHSO100201600008C) with the Biomedical Advanced Research and Development Authority ("BARDA"). The results of these investigations are very encouraging and clearly show that while the vaccine product used in the clinical study continues to meet all manufacturing standards, the immune response to NasoShield is strongly affected by the intranasal administration method. Based on these findings, the Company believes that a simple adjustment to the intranasal dosing procedure may allow NasoShield to elicit a full immune response in humans.

Completed Registered Direct Offering Raising Gross Proceeds of $14 Million
Completed a Registered Direct Offering in March 2019 that raised gross proceeds of $14 million, which brings total gross proceeds received from equity offerings since September 2018 to $56 million. These financings provide the Company with the necessary resources to further develop its programs and position itself for potential acquisitions.

Financial Results for the First Quarter Ended March 31, 2019

The Company had cash, restricted cash and cash equivalents of $44.9 million at March 31, 2019. This was an increase of $10.6 million since the prior year end due to the receipt of net proceeds of $12.7 million through the Registered Direct Offering offset by operating cash burn of $2.1 million.

Revenue in the first quarter was $2.96 million compared to $2.69 million in the prior year period. The increase was due primarily to billings under the Company’s NasoShield contract with BARDA.

Research and development expenses in the first quarter were $3.22 million compared to $5.75 million in the prior year period. The decrease was primarily attributable to lower spending on its NasoVAX and HepTcell programs.

General and administrative expenses in the first quarter were $2.07 million compared to $2.45 million in the prior year period. The decrease was due primarily to a reduction in legal and professional costs.

Net loss attributed to common stockholders for the first quarter was $2.55 million, or ($0.27) per share, compared to $5.06 million, or ($7.49) per share in the same period of 2018.

Conference Call Details

Date:

Wednesday, May 15, 2019

Time:

8:30am Eastern Time

Domestic:

877-423-9813

International:

201-689-8573

Conference ID:

13690295

Webcast:
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