Analysts expect CSL shares to soar above $300 after FDA approves world’s first gene therapy for haemophilia

Chief executive Paul Perreault says the FDA’s approval for the company’s new drug Hemgenix – the world’s first gene therapy treatment for haemophilia – is ‘historic’.

Australia’s biggest health company will launch the first gene therapy for haemophilia B after the US Food and Drug Administration approved its next blockbuster drug Hemgenix – a move that is expected to send its shares soaring well above $300 again.

The drug, known as EtranaDez during its development, will stop spontaneous bleeds in patients by allowing them to produce their own factor IX – a protein that helps form clots.

Wilsons analysts expect Hemgenix, as well as other drugs CSL’s research and development pipeline, will add another $30 to the company’s market value in the next year, sending its share price surging to $327.

CSL shares last traded around that level in early 2020 when it briefly became the biggest company on the ASX, overtaking BHP Billiton, excluding the miner’s then listed London shares, and Commonwealth Bank.

CSL chief executive Paul Perreault described the FDA’s decision as “historic approval”, with Hemgenix becoming the first and only one-time gene therapy for adults with haemophilia B.

“As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with haemophilia B,” Mr Perreault said.

Haemophilia is a genetic blood disorder that plagued royalty in the 19th and early 20th centuries. It continues to affect scores of people today, with haemophilia B occurring in about one in every 40,000 males.

It is a lifelong disorder that stems from a single gene defect that results in insufficient production of factor IX. Before Hemgenix, treatments included prophylactic infusions of the clotting protein, which needed to be administered regularly.

Despite having a strict lifelong schedule of infusions, some patients still experienced spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease.

Peter Marks, director of FDA’s Centre for Biologics Evaluation and Research said the approval comes after 20 years of research.

“Gene therapy for haemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of haemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” Dr Marks said.

“Today’s approval provides a new treatment option for patients with Haemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of haemophilia.”

Most people who have haemophilia B and experience symptoms are men, but many women carriers of the disease have no symptoms. Queen Victoria is believed to have been a carrier, passing on the condition to three of her nine children, including her son Leopold who died of a haemorrhage after a fall when he was 30.

Queen Victoria is believed to have been a haemophilia B carrier.

Kim Phelan, chief operating officer of The Coalition for Haemophilia B, said she was “thrilled” at the launch of Hemgenix, which she said was a “milestone”.

“Over the years we have seen a variety of advancements for the haemophilia community, but gene therapy is the first treatment option to offer those living with haemophilia B — and caregivers — the possibility of freedom from the need for regular, ongoing infusions,” she said.

An ongoing HOPE-B trial found 94 per cent – 51 out of 54 – of patients treated with Hemgenix did not need ongoing prophylaxis therapy. The multi-year clinical development program for Hemgenix was led by uniQure – a gene therapy company listed on the Nasdaq – and sponsorship of the clinical trials transitioned to CSL after it acquired global rights to commercialise the treatment.

Other regulatory agencies are now assessing the drug.

CSL chief medical officer Bill Mezzanotte said Hemgenix gave haemophilia patients more “choice in treatments and better and more durable control over their disease”.

Wilsons analyst Shane Storey said: “Hemgenix will open up the haemophilia B market to a younger cohort of patients that exhibit suboptimal adherence to prophylactic therapy due to the onerous nature of delivery”.

Haemophilia is a lifelong condition.

“The potential to replace ≥10 years of regular prophylactic management for these patients with a single shot of Hemgenix is a powerful driver of sector dominance, which brings with it margin expansion and sales leverage opportunities within the CSL Behring recombinant haemophilia (rHaem) portfolio. We anticipate commercial launch FY24,” Dr Storey said in a note to investors.

CSL shares firmed 0.2 per cent to $297.70 on Wednesday. Dr Storey has a $327 12-month share price target for CSL.

Dr Storeybroke down the valuation of $265 a share in relation to forecast earnings for CSL’s Behring, Seqirus and Vifor divisions. This implied a multiple of 23.3 times earnings.

He then added $62 a share to reflect CSL’s research and development pipeline, which includes Hemgenix and other potential blockbuster drugs.