On August 13, 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 June 30, 2019 (Press release, KemPharm, AUG 13, 2019, View Source [SID1234538734]).

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"Our highest priority at KemPharm has been securing a licensing agreement that maximizes the value of KP415 and KP484. This licensing process has taken longer than expected as we have had several late entrants. Yet, we believe we are in the final phase of this process and remain optimistic about reaching a successful conclusion as soon as possible," said Travis C. Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "Looking ahead, once this partnership is finalized, we will focus our full attention in conjunction with our partner to finalize the NDA for KP415, which we believe could likely be filed prior to year-end."

"Additionally, we continue to work diligently with KVK Tech to prepare for the commercial launch of APADAZ, which remains on track for the second half of 2019," added Dr. Mickle. "Together with KVK, both the commercial outreach and manufacturing processes are moving forward in parallel. We look forward to reporting our progress in the coming months."

Q2 2019 Financial Results:

For the quarter ended June 30, 2019, KemPharm’s reported net loss was $9.3 million, or $0.33 per basic and diluted share, compared to a net loss of $10.0 million, or $0.65 per basic share and $0.91 per diluted share for the same period in 2018. Net loss for Q2 2019 was driven primarily by a loss from operations of $7.8 million, and net interest expense and other items of $1.5 million. The loss from operations for Q2 2019 decreased by $6.1 million compared to a loss from operations of $13.9 million in Q2 2018, which was primarily due to decreases of $5.7 million in research and development expenses and $0.4 million in general and administrative expenses, respectively.

As of June 30, 2019, total cash, which is comprised of cash, cash equivalents and restricted cash, was $7.8 million, which was a decrease of $6.2 million as compared to March 31, 2019.

"During the quarter, we took a number of steps to reduce our operating, general and administrative expenses, including an approximate 30% reduction in workforce compared to the end of Q1 2019, with the goal of reducing our cash burn rate while maintaining our research and development capabilities to support our ongoing work with KP415 and KP484, as well as remaining positioned to support the ultimate commercial partner once that process has been completed. Additionally, as the NDA filing approaches, we expect to see the expenditures related to KP415 R&D continue to reduce substantially," concluded Dr. Mickle.

On August 13, 2019 Mirati Therapeutics presented the corporate presentation (Presentation, Mirati, AUG 13, 2019, View Source [SID1234538644]).

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On August 13, 2019 Quanterix Corporation (Nasdaq: QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, reported the closing of its previously announced underwritten public offering of 2,732,673 shares of its common stock at a public offering price of $25.25 per share, including 356,435 shares sold pursuant to the full exercise of the underwriters’ option to purchase additional shares (Press release, Quanterix, AUG 13, 2019, View Source [SID1234538660]). Gross proceeds from the sale of the shares, before deducting underwriting discounts and commissions and offering expenses, were approximately $69.0 million.

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J.P. Morgan Securities LLC and SVB Leerink LLC acted as joint book-running managers for the offering. Canaccord Genuity LLC acted as co-manager for the offering. Perella Weinberg Partners acted as independent capital markets advisor to Quanterix for the offering.

The public offering was made pursuant to a shelf registration statement on Form S-3 that was previously filed with and declared effective by the Securities and Exchange Commission ("SEC"). The final prospectus supplement and the accompanying prospectus relating to this offering has been filed with the SEC and is available on the SEC’s website located at www.sec.gov, copies of which can be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, telephone: (866) 803-9204; or SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA, 02110, by telephone at (800) 808-7525, ext. 6132 or by e-mail at [email protected].

This press release shall not constitute an offer to sell, or a solicitation of an offer to buy, nor will there be any sale of these securities in any state or other jurisdiction in which such an offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On August 12, 2019 Zai Lab Limited (NASDAQ: ZLAB), a Shanghai-based innovative commercial stage biopharmaceutical company, and Novocure (NASDAQ: NVCR), a global oncology company with a proprietary platform technology called Tumor Treating Fields, reported that the China National Medical Products Administration (NMPA) granted Innovative Medical Device Designation for Optune, a Tumor Treating Fields delivery system that uses electric fields tuned to specific frequencies to disrupt cancer cell division, inhibiting tumor growth and causing affected cancer cells to die (Press release, Zai Laboratory, AUG 12, 2019, View Source [SID1234538601]).

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"We are excited that Optune has been granted the Innovative Device Designation as it will allow our team to accelerate dialogue with the NMPA and bring us closer to commercializing Optune in China," said Dr. Samantha Du, Founder and CEO of Zai Lab. "Our launch in Hong Kong has provided valuable insight into the impact that this device can have on patients with GBM, which is an area of high unmet clinical need in China and globally. We look forward to working closely with the NMPA as Optune advances through the regulatory process in China."

The Innovative Device Designation allows Zai Lab to take advantage of an expedited approval procedure for Optune that offers opportunities for pre-consultation with and input from the NMPA throughout the approval process. Novocure granted Zai Lab an exclusive license for Tumor Treating Fields, including the brand name Optune, in Greater China in September 2018 and Zai Lab successfully launched the product in Hong Kong for the treatment of glioblastoma (GBM) late last year. Novocure markets Optune in the United States, European Union, Japan and certain other countries for the treatment of GBM and the NovoTTF-100L System, another Tumor Treating Fields delivery system, in the U.S. for the treatment of malignant pleural mesothelioma. Tumor Treating Fields is in late stage clinical development for four solid tumor indications including non-small cell lung cancer, brain metastases, pancreatic and ovarian cancers. Tumor Treating Fields was included and recommended with Level 1 evidence as a treatment for GBM in China’s Glioma Treatment Guideline published in 2018.

"We are committed to bringing Optune to as many patients who may benefit as possible," said Novocure’s Executive Chairman Bill Doyle. "Zai Lab has been an excellent partner as we strive to extend survival in some of the most aggressive forms of cancer by developing and commercializing Tumor Treating Fields therapy. We are pleased that together with our partners at Zai Lab we are one step closer to commercializing Optune in China."

About Tumor Treating Fields

Tumor Treating Fields is a cancer therapy that uses electric fields tuned to specific frequencies to disrupt cell division, inhibiting tumor growth and causing affected cancer cells to die. Tumor Treating Fields does not stimulate or heat tissue and targets dividing cancer cells of a specific size. Tumor Treating Fields causes minimal damage to healthy cells. Mild to moderate skin irritation is the most common side effect reported. Tumor Treating Fields is approved in certain countries for the treatment of adults with glioblastoma and mesothelioma, two of the most difficult cancer types to treat. The therapy shows promise in multiple solid tumor types – including some of the most aggressive forms of cancer.

On August 12, 2019 Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing novel drug candidates in indications of high unmet medical need, reported financial results for the second quarter ended June 30, 2019 (Press release, Applied Therapeutics, AUG 12, 2019, View Source [SID1234538620]).

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"During the second quarter, we continued to execute on advancing our robust pipeline of novel drug candidates through the clinic," said Shoshana Shendelman, Founder, Chief Executive Officer and Chair of the Board of Applied Therapeutics. "We initiated our Phase 1/2 trial of AT-007 in Galactosemia in June and reported favorable Single Ascending Dose data from healthy volunteers last week. We look forward to advancing AT-007 through the next portion of the trial, which includes treatment of adults with Galactosemia. In addition, we are preparing for the dosing of the first patient in our registrational Phase 3 trial for our lead asset, AT-001, in Diabetic Cardiomyopathy (DbCM), which we expect to occur in the third quarter."

Recent Highlights

Reported Single Ascending Dose Data from Healthy Volunteer Portion of Phase 1/2 ACTION-Galactosemia Trial Evaluating AT-007. In August 2019, we announced the completion of the Single Ascending Dose (SAD) healthy volunteer portion of the Phase 1/2 study of AT-007 in Galactosemia. AT-007 was well tolerated, with no drug-related adverse events or dose-limiting toxicities reported. The study, referred to as ACTION-Galactosemia, was initiated in June 2019 and is designed to investigate the safety and pharmacokinetics (PK) of AT-007, a central nervous system (CNS) penetrant Aldose Reductase (AR) inhibitor in healthy volunteers, and biomarker effects in adult subjects with Galactosemia. Data from the adult Galactosemia patient portion of the trial is expected in the fourth quarter of 2019. We plan to employ recent FDA guidance permitting biomarker-based development in low prevalence, slowly progressing rare metabolic diseases, such as Galactosemia.

·Presented Phase 1/2 Data Highlighting Safety and Efficacy for AT-001 in DbCM at the American Diabetes Association (ADA) 79th Annual Scientific Sessions in San Francisco. In June 2019, we presented Phase 1/2 Data Highlighting Safety and Efficacy for AT-001 in DbCM at the ADA Annual Scientific Sessions. The data, presented as part of the Late Breaking session, demonstrated that AT-001 was well tolerated at all dose levels, and target engagement was confirmed by potent AR inhibition as evidenced by significant reductions in sorbitol, a pharmacodynamic biomarker of AR activity. AT-001 also improved selectivity and affinity for AR and resulted in potent AR inhibition.

·Received FDA Orphan Drug Designation for AT-007 in Galactosemia. In May 2019, we received orphan drug designation for AT-007 in Galactosemia. The designation allows Applied Therapeutics to qualify for a number of incentives, including: seven years of market exclusivity upon regulatory approval, if received; exemption from FDA application fees for Galactosemia; and tax credits for qualified clinical trials.

·Presented Phase 1/2 Data Highlighting Safety and Proof of Biological Activity for AT-001 in DbCM at The European Society for Cardiology (ESC) 6th World Congress in Athens, Greece. In May 2019, we presented two posters at ESC, the first of which was presented in the Late Breaking session and highlighted key data from a recently completed Phase 1/2 study in approximately 120 type 2 diabetic patients describing the safety, pharmacokinetics and proof of biological activity for AT-001 in DbCM. Supporting preclinical data from an animal model of DbCM was also presented, demonstrating that AT-001 prevents or reduces cardiac damage in a relevant disease model.

·Completed Initial Public Offering. In May 2019, we completed our IPO, generating approximately $34.6 million in net proceeds, after deducting underwriter discounts and commissions and offering expenses payable by us.

Financial Results

·Cash and cash equivalents totaled $41.1 million as of June 30, 2019, compared with $18.8 million at December 31, 2018.

·Research and development expenses for the three months ended June 30, 2019 were $4.3 million, compared to $1.9 million for the three months ended June 30, 2018. The increase of approximately $2.4 million was primarily related to costs associated with progressing our clinical trials, including an increase in clinical and pre-clinical expenses of $1.6 million and personnel expenses of $2.1 million due to the hiring of research and development personnel, including the Chief Medical Officer in August 2018. These increases are offset by a decrease in drug manufacturing and formulation expenses of $1.3 million.

·General and administrative expenses were $4.2 million for the three months ended June 30, 2019, compared to $0.4 million for the three months ended June 30, 2018. The increase of approximately $3.8 million was primarily related to personnel expenses of $2.2 million due to the increase in headcount, including the hiring of the interim Chief Financial Officer and the Controller, professional fees of $0.9 million due to increased legal and consulting fees, and other expenses of $0.7 million, primarily due to public relations efforts, travel expenses and recruiting efforts.

·Net loss for the second quarter of 2019 was $8.4 million, or $0.60 per basic and diluted common share, compared to a net loss of $3.2 million, or $0.58 per basic and diluted common share, for the second quarter of 2018.