An­a­lysts write off Di­men­sion’s lead pro­gram af­ter see­ing weak da­ta on he­mo­phil­ia B, shares plunge

Shares of Di­men­sion Ther­a­peu­tics $DMTX were pum­meled on Tues­day, af­ter the biotech post­ed sub­par Fac­tor IX scores on its gene ther­a­py pro­gram for he­mo­phil­ia B.

By the num­bers: Pa­tients in one co­hort tak­ing DTX101 had peak Fac­tor IX (FIX) ex­pres­sion of 13%, 20% and 12% over 4 to 8 weeks, ac­cord­ing to in­ves­ti­ga­tors. At the 12-week fol­lowup, though, the scores had slipped to 5% and 8% in two pa­tients at 12-weeks fol­low-up, and 7% for the third pa­tient at 7 weeks. “For the low-dose co­hort, ex­pres­sion lev­els achieved 10-11% peak ac­tiv­i­ty, sta­bi­liz­ing be­tween 3-4% at last fol­low-up.”

Spark Ther­a­peu­tics, which has a ri­val gene ther­a­py for he­mo­phil­ia B, has al­ready boast­ed that four par­tic­i­pants ex­pe­ri­enced con­sis­tent and sus­tained fac­tor IX ac­tiv­i­ty lev­els, with a mean greater than 30% of nor­mal. But Spark al­so has had to con­tend with ev­i­dence of an im­mune re­sponse that has had to be bat­ted back with steroids.

In ad­di­tion, Di­men­sion re­port­ed el­e­va­tions in ala­nine amino­trans­ferase (ALT) — ev­i­dence of liv­er tox­i­c­i­ty — in 5 of 6 pa­tients, spurring a need to use steroids. One pa­tient in co­hort 2 ex­pe­ri­enc­ing a grade 4 ad­verse event due to an el­e­vat­ed lab­o­ra­to­ry ALT, and the biotech is hold­ing back on dos­ing co­hort 3 un­til they have a chance to re­view things with the FDA.

Gena Wang, Jef­feries

That’s not what an­a­lysts want­ed to see.

Re­port­ed Gena Wang at Jef­feries:

DMTX re­port­ed He­moB da­ta which ap­pears sub­par with 3-8% FIX ac­tiv­i­ty and steroid use in 5/6 pts. While we see lim­it­ed chance in He­moB with cur­rent da­ta, we be­lieve pun­ish­ment is over­done (trad­ing be­low cash). He­moA oppt re­mains some­what open and we see a sig­nif­i­cant­ly dif­fer­ent bar for the OTC pro­gram (urea-cy­cle dis­or­der, da­ta could be in 2017). PT low­ered to $7 to re­flect re­duced oppt in he­mo­phil­ia but main­tain Buy rat­ing and we see up­side from here.

The com­par­i­son be­tween Di­men­sion and its top ri­val proved un­ap­peal­ing to in­vestors, who slashed Di­men­sion’s shares by about half in mid-day trad­ing.

UniQure has an­oth­er he­mo­phil­ia B gene ther­a­py it’s work­ing on, which has al­so paled in com­par­i­son with Spark’s ef­fort. Bio­Marin, mean­while, is seen as well out in front with its work on he­mo­phil­ia A.

“We are en­cour­aged by the ap­par­ent ef­fi­cien­cy of gene trans­duc­tion and the ear­ly trend we are see­ing in sus­tained FIX ac­tiv­i­ty across both co­horts with our wild-type FIX AAVrh10 vec­tor in pa­tients. We con­tin­ue to ex­plore the ther­a­peu­tic win­dow for DTX101 as our da­ta ma­ture and in light of the ALT ris­es that ap­pear to be as­so­ci­at­ed with a de­cline in FIX ac­tiv­i­ty,” said Dr. An­nal­isa Jenk­ins, MBBS, FR­CP, Chief Ex­ec­u­tive Of­fi­cer of Di­men­sion.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Brian Stuglik (file photo)

Turn­ing fo­cus to clin­i­cal work, Ve­rastem ax­es 31 jobs, scales back can­cer drug pro­mo­tion af­ter dis­ap­point­ing sales

Months after taking the helm at Verastem Oncology, Brian Stuglik has a plan to take the biotech in a “new strategic direction” — but not before some layoffs.

Left out of an upbeat press release spelling out its clinical plans, and buried below news of a $100 million private placement in an SEC filing, is a planned restructuring that will claim 31 jobs. Alongside some other cost-saving measures, Verastem expects to cut expenses down by $70 million to $80 million per year.

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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