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 13, 2019 Potentially curative cancer treatments, like chimeric antigen receptor (CAR) T-cell therapies, have revolutionized care for illnesses once thought to be a death sentence after relapse, such as pediatric acute lymphoblastic leukemia (pALL) and diffuse large B-cell lymphoma (DLBCL). While 2 approved treatments are available (tisagenlecleucel and axicabtagene ciloleucel), therapy initiation is often delayed due to payment processes—in fact, it was only this month that the Centers for Medicare and Medicaid Services released a long-awaited payment decision for Medicare beneficiaries with cancer in need of treatment with CAR T-cell therapy as well as a boost in hospital payment rates (Press release, The American Journal of Managed Care, AUG 13, 2019, View Source [SID1234538661]). In a new study in the current issue of The American Journal of Managed Care (AJMC), researchers defined the concept of the social value that these potentially curative therapies provide, but they caution that treatment delays caused by a lack of payment mechanisms and policies are limiting the potential impact of these treatments.

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The issue stems from the fact that one-time CAR T-cell therapies, and even more costly upcoming gene therapies, are a challenge to existing US payment structures; their costs accrue up front, in contrast with other cancer drugs that are given—and paid for—over an extended time period.

Using an economic framework, researchers measured the social value of CAR T-cell therapy for relapsed or refractory pALL and DLBCL. Social value analyses were used to quantify a therapy’s economic value from a societal perspective and determine the share of that value accruing to the manufacturer and patients. Expanded patient access and greater health benefits increase social value, while higher production costs to develop the therapy reduce it.

Manufacturers have stronger incentives for innovation with a rising share of social value, but when treatment is delayed, social value is lost for both patients and manufacturers: Patients lose access to health improvements and manufacturer profit is reduced.

The study found that CAR T-cell therapy generated up to $6.5 billion and $34.8 billion of social value for patients with pALL and DLBCL, respectively. However, with one, two and six months of treatment delay, patients with pALL lost 9.8 percent, 36.2 percent and 67.3 percent of social value, respectively; patients with DLBCL lost 4.2 percent, 11.5 percent and 46 percent of social value, respectively.

The degree of CAR T-cell therapy’s value depends on timely patient access, but the authors note that it is not unusual for CAR T-cell therapy reimbursement approval to take up to 90 days, which may be longer than a waiting patient may live. Given other high-cost therapies in the pipeline, lessons learned from CAR T-cell therapies can help payers, policy makers and manufacturers, the authors say.

"Our research shows that CAR T offers potentially large value for patients and society, but only if patients can access it promptly," said lead study author Julia Thornton Snider, PhD. "It will be essential for all stakeholders to work together to ensure appropriate policies are in place to secure timely access for patients."

The study was funded by Novartis, maker of tisagenlecleucel.

On August 13, 2019 4SC AG (4SC, FSE Prime Standard: VSC) reported, that it received a single digit million Euro milestone payment from its partner Guangzhou Link Health Pharma Co., Ltd (Link Health) in accordance with the licensing and development agreement for the cancer therapeutic candidate 4SC-205 (Press release, 4SC, AUG 13, 2019, View Source [SID1234538646]).

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Under the 2016 agreement, Link Health received from 4SC the exclusive licensing rights for the development, regulatory submission and marketing of 4SC-205 in China, Hong Kong, Taiwan and Macao and is responsible for performing and financing the clinical development of 4SC-205.

Yan Song, Ph.D., CEO of Link Health, says: "We initiated a Phase I, open label, dose escalation trial to assess the safety, tolerability and pharmacokinetics of LH031 (4SC-205) orally administered in adult patients with advanced solid malignancies or lymphoma. Secondary objectives include to assess the drug candidate’s potential anti-cancer activity."

Jason Loveridge, Ph.D., CEO of 4SC, comments: "We are very pleased that our Chinese partner has successfully advanced 4SC-205 into the next stage of development and initiated the first clinical trial with our drug candidate in Asia. 4SC remains focused on advancing our core drug candidates resminostat and domatinostat, but nonetheless our strategy to monetize and advance non-core assets through partnerships continues to progress well."