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.