On May 14, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported its financial and operational results for the first quarter ended March 31, 2019 (Press release, Autolus, MAY 14, 2019, View Source [SID1234550817]).

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Key first quarter highlights include:

Clinical and Regulatory

In April, Autolus announced the presentation of initial data from the ongoing Phase 1/2 ALLCAR19 trial of AUTO1 in adult acute lymphoblastic B cell leukemia (ALL) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in Atlanta, Georgia. As of the data cutoff date of March 18, 2019, 13 patients were leukapheresed, and products for 12 patients were manufactured, including 7 with Autolus’ semi-automated, fully enclosed manufacturing process. Using the Lee criteria, there were no patients with severe cytokine release syndrome (CRS) (≥ Grade 3), and only 2 of 10 patients (20%) with Grade 2 CRS. Tocilizumab was used in 2 of 10 patients (20%). None of the patients were admitted to intensive care due to CRS. One patient developed delayed Grade 3 neurotoxicity following high levels of CAR T expansion, which resolved promptly following administration of steroids. Four patients died while enrolled in the trial, two due to progression of the disease and two due to sepsis, a common complication of advanced ALL. Nine patients were evaluable for response at 1 month with 9 (88%) achieving a molecular complete response. One patient died of sepsis before the one-month evaluation point. At a median follow up of 5 months (range 0.62-10.6 months), 6/10 patients are alive and continue to be in molecular remission and there continues to be evidence of ongoing B cell aplasia and CAR T persistence.

In April, Autolus announced that the United States Food and Drug Administration granted orphan drug designation to autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors targeting CD19 and CD22 (AUTO3) for the treatment of ALL.

Autolus hosted an R&D Day in New York City in March for the investment community. The event provided an update on Autolus’ current clinical programs and highlighted the company’s approach to drive molecular innovation and next-generation programed T cell products for hematological and solid tumor indications.

During the March R&D Day, Autolus provided updated data from the ongoing AMELIA Phase 1/2 study of AUTO3 in pediatric ALL which demonstrated that 6 out of 6 (100%) patients treated at the highest dose (≥3 x 106/kg) achieved minimal residual disease (MRD) negative complete responses (CR). Ongoing MRD negative CR remissions were noted in 4 out of 6 (67%) patients, with duration of up to 10 months as of February 2019, the date of latest data follow-up. There have been no reported CD19 or CD22 negative relapses in CAR T naïve patients. Data also showed that AUTO3 continues to be generally well tolerated with no ≥ Grade 3 CRS, no intensive care admission, and no pressors or critical care support for CRS required.
Manufacturing and Product Delivery

In March, manufacturing for clinical studies commenced at the Cell and Gene Therapy Catapult Manufacturing Centre in Stevenage, United Kingdom.
Corporate Highlights

In April, Autolus completed an underwritten public offering of 4,830,000 American Depositary Shares ("ADSs") representing 4,830,000 ordinary shares, at a public offering price of $24.00 per ADS, which includes an additional 630,000 ADSs issued upon the exercise in full of the underwriters’ option to purchase additional ADSs. Aggregate net proceeds to Autolus, after underwriting discounts but before estimated offering expenses, were $108.9 million. Proceeds from this public offering are not included in the March 31, 2019 financial statements.
Anticipated Milestones

Presentation of a data update from the ALEXANDER Phase 1/2 trial of AUTO3 in adult relapsed/refractory diffuse large B cell lymphoma (DLBCL) in the third quarter of 2019.

Initiation of the Phase 2 portion of the AMELIA trial of AUTO3 in pediatric ALL in the second half of 2019.

Initiation of a Phase 2/registration trial of AUTO1 in adult ALL in the second half of 2019 (pending regulatory feedback).

Presentation by the end of 2019 of data updates from the following trials: AUTO1 in adult ALL and pediatric ALL; AUTO3 in DLBCL and pALL and AUTO2 in multiple myeloma.
"In the first quarter of 2019, we made good progress in all aspects of the business. Important was the first presentation of clinical data from AUTO1 in adult patients with acute lymphoblastic leukemia, which points to a differentiated profile for AUTO1," stated Dr. Christian Itin, chairman and chief executive officer of Autolus. "For the remainder of 2019, we are placing particular focus on advancing our clinical programs, specifically AUTO3 in DLBCL and AUTO1 in adult ALL, towards registrational trials."

Financial results for first quarter 2019:

Cash and equivalents at March 31, 2019 totaled $187.7 million, compared with $217.5 million at December 31, 2018.

Net total operating expenses for the three months ended March 31, 2019 were $30.2 million, net of grant income of $2.0 million, as compared to net operating expenses of $15.5 million, net of grant income of $0.4 million, for the same period in 2018. The increase was due, in general, to the increase in clinical trial activity, which is expected to deliver on key milestones throughout the rest of 2019; increased headcount; and the cost of being a public company.

Research and development expenses increased to $22.6 million for the three months ended March 31, 2019 from $11.6 million for the three months ended March 31, 2018. Cash costs, which exclude depreciation as well as share-based compensation, increased to $17.5 million from $10.6 million. The increase in research and development cash costs of $6.9 million consisted primarily of an increase of compensation-related costs of $5.6 million primarily due to an increase in headcount to support the advancement of our product candidates in clinical development, an increase of $2.7 million in facilities costs supporting the expansion of our research and translational science capability and investment in manufacturing facilities and equipment, and an increase of $0.8 million in research and development program expenses related to the activities necessary to prepare, activate, and monitor clinical trial programs, offset by a decrease of $1.9 million in professional fees primarily related to the UCL license fees expensed for the three months ended March 31, 2018, and other reductions of $0.3 million.

General and administrative expenses increased to $9.6 million for the three months ended March 31, 2019 from $4.3 million for the three months ended March 31, 2018. Cash costs, which exclude depreciation as well as share-based compensation, increased to $6.3 million from $3.5 million. The increase of $2.8 million consisted primarily of an increase in compensation-related expense of $1.2 million due to an overall increase in headcount, and an increase in legal and professional fees of $0.9 million related to insurance and patent costs.

Net loss attributable to ordinary shareholders was $27.2 million for the three months ended March 31, 2019, compared to $16.7 million for the same period in 2018.

The basic and diluted net loss per ordinary share for the three months ended March 31, 2019 totaled $(0.69) compared to a basic and diluted net loss per ordinary share of $(0.58) for the three months ended March 31, 2018.

Autolus anticipates that cash on hand provides a runway into the second half of 2021.
Conference Call and Presentation Information

Autolus management will host a conference call today, May 14, at 8:30 a.m. EDT/1:30 p.m. BST, to discuss the company’s financial results and operational update.

To listen to the webcast and view the accompanying slide presentation, please go to: View Source

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 7358198. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 7358198.

On May 14, 2019 Bio-Techne Corporation (NASDAQ:TECH) reported that Chuck Kummeth, President and Chief Executive Officer, will present at the UBS Global Healthcare Conference on Wednesday, May 22nd, 2019, at 9:00 a.m. EDT (Press release, Bio-Techne, MAY 14, 2019, View Sourcenews/detail/138/bio-techne-to-present-at-the-ubs-global-healthcare-conference" target="_blank" title="View Sourcenews/detail/138/bio-techne-to-present-at-the-ubs-global-healthcare-conference" rel="nofollow">View Source [SID1234536925]). The conference will be held at the Grand Hyatt Hotel in New York City.

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A live webcast of the presentation can be accessed via Bio-Techne’s Investor Relations website at View Source or through the following link https://protect-us.mimecast.com/s/H5PbCR6MPEIgkMNru9JEaM?domain=cc.talkpoint.com.

On May 14, 2019 Alder BioPharmaceuticals, Inc. (NASDAQ:ALDR), a biopharmaceutical company focused on developing novel therapeutic antibodies for the treatment of migraine, reported that members of the management team will provide a business overview and update at the 2019 RBC Capital Markets Global Healthcare Conference at 9:30 am ET on Tuesday, May 21, 2019 and at the UBS Global Healthcare Conference at 10:00 am ET on Wednesday, May 22, 2019, both in New York, NY (Press release, Alder Biopharmaceuticals, MAY 14, 2019, View Source [SID1234536343]).

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The presentations will be webcast live on the Events & Presentations page of the Investors section of Alder’s website at View Source, or by following the links below in your web browser. An archived replay of each webcast will be available on Alder’s website for at least 30 days after each live event concludes.

RBC:http://www.veracast.com/webcasts/rbc/healthcare2019/15314267548.cfm

UBS: https://cc.talkpoint.com/ubsx001/052019a_as/?entity=49_QLELLE8

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.