Investors & Media Press Releases
Axovant Announces Third Fiscal Quarter 2018 Financial Results and Corporate Updates
- Expanded pipeline with two investigational gene therapy programs licensed from the
University of Massachusetts Medical Schoolfor the treatment of GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases
- First two patients dosed in SUNRISE-PD phase 2 trial of AXO-Lenti-PD for Parkinson's disease and initial patient dosed with AXO-AAV-GM2; data from both programs expected in
March 2019 Axovant Sciences Ltd.intends to change its name to Axovant Gene Therapies Ltd.and ticker symbol to “AXGT” to reflect its exclusive focus on gene therapies
“With the addition of new investigational gene therapies for the treatment of GM1 gangliosidosis, Tay-Sachs, and Sandhoff diseases, we are excited about having built a deep pipeline of potentially transformative gene therapies addressing serious conditions. Over the last year, Axovant has become leaner and more cutting-edge in its scientific approach, but we are no less ambitious in our drive to make a difference for patients,” said Pavan Cheruvu, M.D., Chief Executive Officer of Axovant. “We look forward to continuing this momentum with a rich set of clinical development milestones this quarter, including the first data readouts in our AXO-Lenti-PD and AXO-AAV-GM2 programs next month, and additional milestones across our pipeline throughout 2019.”
Key Highlights and Development Updates
- In-licensed two programs, AXO-AAV-GM1 and AXO-AAV-GM2, for GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases from the
University of Massachusetts (UMass) Medical Schoolin December 2018. The programs aim to restore enzymes that are deficient in these diseases by introducing a functional copy of the defective genes.
- First patient dosed with AXO-AAV-GM2 in an investigator-initiated study with initial data expected in
March 2019. Plan to dose first patient with AXO-AAV-GM1 in the first half of calendar year 2019.
- Two patients were dosed with AXO-Lenti-PD in the SUNRISE-PD trial for patients with Parkinson’s disease. Initial data is expected in
March 2019. FDAconfirmed that studies previously conducted using first generation ProSavin® may be considered part of a single development program with AXO-Lenti-PD, including the preclinical data and over 10 years of clinical data of ProSavin. The FDAalso provided feedback on the design of the SUNRISE-PD phase 2 study of AXO-Lenti-PD and agreed that current manufacturing and quality control plans were adequate for the clinical program.
- Hired five new senior leaders with decades of collective gene therapy expertise in key areas such as clinical development, vector optimization and delivery, regulatory affairs, manufacturing and commercialization. Assembled a
Scientific Advisory Boardof pre-eminent leaders in gene therapy to provide strategic guidance across all development programs.
- Shankar Ramaswamy, MD, was named Chief Business Officer and is responsible for the identification, evaluation, and negotiation of transactions for new gene therapy pipeline assets and other business development opportunities. He was most recently Senior Vice President of Business Development and Operations.
Axovant Sciencesplans to change its name to Axovant Gene Therapies and its stock symbol to “AXGT” to reflect its exclusive focus on the development and commercialization of innovative gene therapies. The company’s name change is expected to take effect in March 2019. Common stock will begin trading under a new ticker symbol “AXGT” at the opening of trading on February 14, 2019. The former ticker symbol “AXON” will remain effective through market close on February 13, 2019.
$30 millionequity financing in December 2018led by Deerfield Management Company, Sphera Funds Management and Roivant Sciences.
Third-Quarter Financial Summary
For the third fiscal quarter ended December 31, 2018, research and development expenses were
Nine-Months Financial Summary
For the nine months ended December 31, 2018, research and development expenses were
As of December 31, 2018, the Company had
This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” “continue,” or “well-positioned” and other similar expressions are intended to identify forward-looking statements. For example, all statements Axovant makes regarding the initiation, timing, progress, and reporting of results of its preclinical programs, clinical trials, and research and development programs; its ability to advance its gene therapy product candidates into and successfully initiate, enroll, and complete clinical trials; the potential clinical utility of its product candidates; its ability to continue to develop its gene therapy platforms; its ability to develop and manufacture its products and successfully transition manufacturing processes; its ability to perform under existing collaborations with, among others,
Media and Investors
Condensed Consolidated Statements of Operations
(Unaudited, in thousands, except share and per share amounts)
|Three Months Ended
|Nine Months Ended
|Research and development expenses(1)|
|(includes total share-based compensation expense of $1,910 and $2,453 for the three months ended December 31, 2018 and 2017 and $3,299 and $14,625 for the nine months ended December 31, 2018 and 2017, respectively)||$||21,483||$||37,346||$||80,403||$||119,613|
|General and administrative expenses(2)|
|(includes share-based compensation expense of $2,648 and $8,186 for the three months ended December 31, 2018 and 2017 and $9,575 and $26,954 for the nine months ended December 31, 2018 and 2017, respectively)||10,933||18,032||33,309||69,662|
|Total operating expenses||32,416||55,378||113,712||189,275|
|Other expense (income)||(78||)||550||275||324|
|Loss before income tax expense||(34,244||)||(57,878||)||(119,795||)||(195,301||)|
|Income tax expense||52||24||224||953|
|Net loss per common share — basic and diluted||$||(0.27||)||$||(0.54||)||$||(1.01||)||$||(1.83||)|
|Weighted average common shares outstanding — basic and diluted||128,771,900||107,719,476||119,183,117||107,241,043|
(1) Includes total costs allocated from
(2) Includes total costs allocated from RSL, RSI and RSG of
Condensed Consolidated Balance Sheets
|December 31, 2018||March 31, 2018|
|Cash and cash equivalents||$||84,939||$||154,337|
|Prepaid expenses and other current assets||4,883||2,174|
|Income tax receivable||1,580||1,751|
|Total current assets||91,402||158,262|
|Other non-current assets||3,449||—|
|Property and equipment, net||1,365||2,524|
|Liabilities and Shareholders’ Equity|
|Due to RSL, RSI and RSG||693||1,011|
|Current portion of long-term debt||20,583||9,753|
|Total current liabilities||45,205||46,575|
|Common shares, par value $0.00001 per share, 1,000,000,000 shares authorized, 155,527,771 and 107,788,074 issued and outstanding at December 31, 2018 and March 31, 2018, respectively||2||1|
|Additional paid-in capital||699,064||628,110|
|Accumulated other comprehensive income||664||126|
|Total shareholders’ equity||22,760||71,286|
|Total liabilities and shareholders’ equity||$||96,216||$||160,786|
Source: Axovant Sciences Ltd.